2015 annual report - center for international blood and marrow … · 2016-02-01 · suite c5500 ....
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CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program®/Be The Match® and Medical College of Wisconsin
Sharing Knowledge. Sharing Hope.
2015 Annual Report
2015 Annual Report
January – December
Milwaukee Campus Medical College of Wisconsin 9200 W Wisconsin Ave, Suite C5500 Milwaukee, WI 53226 (414) 805-0700
Minneapolis Campus National Marrow Donor Program/ Be The Match 500 N 5th St Minneapolis, MN 55401 (612) 627-5800
cibmtr.org
CIBMTR 2015 Annual Report TABLE OF CONTENTS
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TABLE OF CONTENTS
1.0 WHO WE ARE ........................................................................................................................................ 1
1.1 Mission .............................................................................................................................................. 1
1.2 Value to the HCT Community ............................................................................................................ 1
1.3 Organizational Structure ................................................................................................................... 1
1.3.1 Scientific Working Committees .................................................................................................. 6
2.0 WHAT WE DO ........................................................................................................................................ 8
2.1 Clinical Outcomes Research Program ............................................................................................. 11
2.1.1 Scientific Working Committees ................................................................................................ 11
2.1.2 Cellular Therapies and Non-Transplant Therapy Research Initiatives ..................................... 15
2.2 Immunobiology Research Program ................................................................................................. 16
2.3 Clinical Trials Support Program ....................................................................................................... 18
2.3.1 Blood and Marrow Transplant Clinical Trials Network ............................................................ 18
2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation ............................ 20
2.4 Health Services Research Program.................................................................................................. 21
2.5 Bioinformatics Research Program ................................................................................................... 23
2.6 Statistical Methodology Research Program .................................................................................... 24
2.7 Stem Cell Therapeutic Outcomes Database (SCTOD) ..................................................................... 25
2.8 Corporate Program .......................................................................................................................... 27
3.0 HOW WE SHARE KNOWLEDGE ........................................................................................................... 28
3.1 Information Request Service ........................................................................................................... 33
3.2 Internet Presence ............................................................................................................................ 34
3.2.1 CIBMTR Public Website ............................................................................................................ 34
3.2.2 CIBMTR Collaborative Site ........................................................................................................ 35
3.2.3 CIBMTR Portal Site .................................................................................................................... 36
3.2.4 Be The Match Public Website................................................................................................... 37
3.2.5 Be The Match Clinical Website ................................................................................................. 37
3.2.6 HRSA Blood Cell Transplant Website........................................................................................ 37
3.2.7 Other Applications and Data Exchange Standards ................................................................... 38
3.3 BMT Tandem Meetings ................................................................................................................... 39
3.4 Data Management Training ............................................................................................................ 40
4.0 HOW WE COLLECT AND MANAGE DATA ............................................................................................ 41
4.1 Research Data Life Cycle ................................................................................................................. 41
4.2 Collecting and Storing Data ............................................................................................................. 42
4.2.1 FormsNet .................................................................................................................................. 42
4.2.2 Research Database ................................................................................................................... 42
4.3 Ensuring Data Quality ...................................................................................................................... 42
CIBMTR 2015 Annual Report TABLE OF CONTENTS
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4.3.1 Continuous Process Improvement ........................................................................................... 42
4.3.2 Verification and Validation ....................................................................................................... 43
4.3.3 On-Site Data Audit Program ..................................................................................................... 44
4.4 Protecting Patients and Data .......................................................................................................... 45
4.4.1 Human Subjects / HIPAA Compliance ...................................................................................... 45
4.4.2 Information Security and Data Privacy ..................................................................................... 45
5.0 WHAT WE WILL DO NEXT.................................................................................................................... 46
2015 KEY ACCOMPLISHMENTS ................................................................................................................. 49
APPENDIX A: TRANSPLANT CENTERS ........................................................................................................ 53
Appendix A1: US Centers....................................................................................................................... 54
Appendix A2: International Centers ...................................................................................................... 65
APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP ..................... 74
Appendix B1: Organizational Structure – Milwaukee Campus ............................................................. 75
Appendix B2: Organizational Structure – Minneapolis Campus ........................................................... 76
Appendix B3: Coordinating Center Leadership ..................................................................................... 78
APPENDIX C: COMMITTEE MEMBERSHIP ................................................................................................. 87
Appendix C1: Advisory Committee Membership .................................................................................. 87
Appendix C2: Executive Committee Membership ................................................................................ 90
Appendix C3: Consumer Advocacy Committee Membership ............................................................... 92
Appendix C4: Nominating Committee Membership ............................................................................. 93
Appendix C5: Scientific Working Committee Leadership ..................................................................... 94
Appendix C6: Immunobiology Steering Committee Membership ........................................................ 98
Appendix C7: Clinical Trials Advisory Committee Membership ............................................................ 99
APPENDIX D: PUBLICATIONS ................................................................................................................... 100
Appendix D1: Scientific Working Committee Publications ................................................................. 100
Appendix D2: BMT CTN Publications .................................................................................................. 112
Appendix D3: Health Services Research Program Publications .......................................................... 115
Appendix D4: Bioinformatics Research Program Publications ........................................................... 116
Appendix D5: Statistical Methodology Research Program Publications ............................................ 119
Appendix D6: Coordinating Center Publications ................................................................................. 120
APPENDIX E: PRESENTATIONS ................................................................................................................ 124
APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ..................................................... 132
APPENDIX G: CLINICAL TRIALS ................................................................................................................ 135
Appendix G1: BMT CTN Clinical Trials Open for Enrollment ............................................................... 135
Appendix G2: RCI BMT Clinical Trials .................................................................................................. 137
APPENDIX H: FORMS SUBMISSION PROCESS ......................................................................................... 140
APPENDIX I: WEBSITES ............................................................................................................................ 141
APPENDIX J: GLOSSARY ........................................................................................................................... 142
CIBMTR 2015 Annual Report TABLE OF CONTENTS
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FIGURES AND TABLES Figure 1.1. Scientific Organizational Structure ........................................................................................... 2
Figure 1.2. Functional Organizational Structure with Scientific Oversight ................................................. 3
Table 1.3. Committee Structure ................................................................................................................. 4
Figure and Table 2.1. Distribution of Patients in the CIBMTR Research Database by Graft Source .......... 8
Figure and Table 2.2. Distribution of Patients in the CIBMTR Research Database by Disease .................. 9
Figure 2.3. Continued Growth in the Number of Patients Registered with the CIBMTR ......................... 10
Table 2.4. 2015 Working Committee Studies ........................................................................................... 12
Figure 2.5. 2015 Publications by Program ................................................................................................ 13
Figure 2.6. Working Committee Study Proposal Review Process............................................................. 14
Figure 3.1. How to Access CIBMTR Knowledge ........................................................................................ 28
Table 3.2. How to Access CIBMTR Information ........................................................................................ 29
Table 3.3 How to Access CIBMTR Data ..................................................................................................... 29
Table 3.4 How to Access CIBMTR Tools .................................................................................................... 30
Table 3.5 How to Access CIBMTR Biospecimens ...................................................................................... 30
Table 3.6. Standard Reports Published by the CIBMTR ............................................................................ 31
Table 3.7. Data Requests Addressed by the CIBMTR in 2015................................................................... 33
Figure 4.1. Research Data Life Cycle ......................................................................................................... 41
Figure 4.2. Audit Process .......................................................................................................................... 44
Figure 5.1. Plans for 2016 ......................................................................................................................... 46
Table 5.2. Plans to Enhance Data .............................................................................................................. 47
Table 5.3. Plans to Expand Knowledge Sharing ........................................................................................ 47
Table 5.4. Plans to Increase Impact .......................................................................................................... 48
Figure A.1. Location of Transplant Centers that Submit Data to the CIBMTR ......................................... 53
CIBMTR 2015 Annual Report 1.0 WHO WE ARE
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1.0 WHO WE ARE
The CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW).
1.1 MISSION The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide to increase survival and enrich quality of life for patients. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical outcomes database.
CIBMTR Programs Clinical Outcomes Research
Immunobiology Research
Clinical Trials Support
• Blood and Marrow Transplant Clinical Trials Network (BMT CTN)
• Resource for Clinical Investigations in Blood and Marrow Transplantation (RCI BMT)
Health Services Research
Bioinformatics Research
Statistical Methodology Research
1.2 VALUE TO THE HCT COMMUNITY The CIBMTR has been collecting HCT outcomes data worldwide for >40 years, resulting in a Research Database with information on >425,000 patients. These data are available to investigators with interest in HCT and treatments for cancer and other life-threatening diseases. The CIBMTR has become a respected leader in HCT research by providing a unique resource of information and expertise to the medical and scientific communities.
1.3 ORGANIZATIONAL STRUCTURE The CIBMTR (Figures 1.1 and 1.2) represents a large network of approximately 400 participating transplant centers (Appendix A) that submit transplant-related data for patients. Centers submit data at two levels: a Transplant Essential Data (TED) level, which captures basic data, and a Comprehensive Report Form (CRF) level, which captures more detail. The CIBMTR Coordinating Center, staffed by almost 200 employees (Appendix B), provides data acquisition, management, and statistical support for analyses of these data.
The Chief Scientific Director is responsible for all administrative and scientific operations. The Associate and Senior Scientific Directors assist in overseeing operational aspects of the Coordinating Center. CIBMTR committees (Table 1.3) provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of the scientific and medical communities.
CIBMTR 2015 Annual Report 1.0 WHO WE ARE
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Figure 1.1. Scientific Organizational Structure
Joint Affiliation CommitteeExecutive Director
J Chell, MD
National Marrow Donor Program/Be The MatchTransplant Centers
CIBMTR Assembly
Advisory Committee
Executive Committee
Medical College of Wisconsin
Senior Research AdvisorD Weisdorf, MD
Senior Scientific Director for Research Operations
M Eapen, MBBS, MS
Senior Scientific Director for SCTOD
JD Rizzo, MD, MS
Associate Scientific Director for CIBMTR
MinneapolisD Confer, MD
Chief Scientific DirectorM Horowitz, MD, MS
Scientific Working CommitteesSteering Committees
Consumer Advocacy Committee
Chief Statistical Director
MJ Zhang, PhD
Statistical Methodology
Research ProgramMJ Zhang, PhD
Clinical Outcomes Research Program
M Eapen, MBBS, MS
Health Services Research Program
L Burns, MD
Immunobiology Research Program
S Lee, MD, MPH
Clinical Trials Support Program
D Confer, MDL Burns, MD
M Pasquini, MD, MSB Shaw, MD, PhD
Blood and Marrow Transplant Clinical Trials Network
(BMT CTN)M Pasquini, MD, MS
Resource for Clinical Investigations in Blood and Marrow Transplantation
(RCI BMT)D Confer, MDL Burns, MD
B Shaw, MD, PhD
Advisory role Senior Scientific Director for Data OperationsB Shaw, MD, PhD
Advisory role
Bioinformatics Research Program
M. Maiers, MS
CIBMTR 2015 Annual Report 1.0 WHO WE ARE
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Figure 1.2. Functional Organizational Structure with Scientific Oversight
Research & AdministrationM Horowitz & D Confer
CIBMTR Administration
P Steinert & R King
Finance, Grants & Contracts
MCW & NMDP/Be The Match
Faculty
M Horowitz & D Confer
InformationTechnology
E Bergman & M PrestegaardMKE & MPLS
(M Eapen)(D Rizzo)(B Shaw)
Corporate
S FisherMKE
(M Eapen)
Advancement
S Fisher &T HousemanMKE & MPLS
(M Horowitz)
BMT CTN
A Foley MPLS
(M Pasquini)
Data Operations
J Brunner & M Matlack
MKE & MPLS
(B Shaw)
Clinical Studies Support
R Drexler
MPLS
(D Confer)(L Burns)(B Shaw)
Quality Assurance
TBDMKE
(M Horowitz)
Human Research Protection Program
R KingMPLS
(**B Lindberg)
Auditing & Monitoring
D Christianson
MPLS
(B Shaw)
Statistics & Observational
Research
W Perez &S Spellman
MKE & MPLS
(M Eapen)
Immunobiology Research
S SpellmanMPLS
(S Lee)
Statistics & Clinical
Outcomes
W Perez & S Spellman
MKE & MPLS
(MJ Zhang)(M Eapen)
SurveyResearch
Group
D MattilaMPLS
(D Confer)
RCI BMT
R DrexlerMPLS
(D Confer)(L Burns)(B Shaw)
Database Administrator
& Data Architect
B Liu &
TBDMKE & MPLS
Data Solutions
R RennerMPLS
Project Management& Analysis
T Moerke& K Gee
MKE & MPLS
Applications
T Hongyu& E Chan
MKE & MPLS
Recipient Data
Management
S Meiers & K Gardner
MKE & MPLS
(B Shaw)
Donor Data Management
A HauckMPLS
(D Confer)
Data Entry & Imaging
B LevesqueMPLS
(B Shaw)
Training
M MatlackMPLS
(B Shaw)
Data Management
Special Projects
J Brunner & M Matlack
MKE & MPLS
(B Shaw)
MDs
PhDs
Data Support
M MatlackMPLS
(B Shaw)
TechnicalServices
T MoerkeMKE
Health Services
Research*
E DenzenMPLS
(L Burns)
Key: MKE = Milwaukee, MPS = Minneapolis, ( ) indicates scientific oversight, *staffed through NMDP/Be The Match Patient and Health Professional Services, ** institutional official oversight
Meetings
T HousemanMPLS
(M Horowitz)
Communications
S FisherMKE
(M Horowitz)
Bioinfomatics
M MaiersMPLS
Business Office
C Gonzalez &P Vespalec
MKE
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Table 1.3. Committee Structure
Committee Function Meetings Roster
Joint Affiliation Board • Reviews and approves the CIBMTR budget and research plan • Amends the terms of the affiliation agreement, as necessary • Reviews and approves data access and confidentiality policies
• Annually
Assembly • Includes representatives from each transplant center that submits CRF-level data
• Elects members of the Advisory, Nominating, and Clinical Trials Advisory Committees
• Annually during the BMT Tandem Meetings
Advisory Committee • Oversees CIBMTR policies and scientific agenda • Partners with the Working Committees to prioritize scientific
studies
• In person annually at the BMT Tandem Meetings
• By teleconference quarterly and as needed
Appendix C1
Executive Committee (subcommittee of Advisory Committee)
• Provides scientific and policy advice to the Chief Scientific Director and Coordinating Center
• Reviews audit results and makes recommendations for improvement
• Four times annually by teleconference
Appendix C2
Consumer Advocacy Committee (subcommittee of Advisory Committee)
• Provides patient and donor perspectives during the development of the CIBMTR research agenda
• Communicates CIBMTR research results and data to the non-medical community
• In person annually at the BMT Tandem Meetings
• By teleconference periodically
Appendix C3
Nominating Committee
• Prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees
• Makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments
• At least once annually each Fall by teleconference
Appendix C4
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Committee Function Meetings Roster
Scientific Working Committees (Section 1.3.1)
• Design and conduct relevant studies using CIBMTR data, statistical resources, networks, and / or centers
• Set priorities for clinical outcomes studies • Assess and revise CIBMTR data collection forms, as needed • Plan and conduct workshops at CIBMTR meetings
• In person annually at the BMT Tandem Meetings
• Leadership - by teleconference every 4-8 weeks
Leadership - Appendix C5
Immunobiology Steering Committee / NMDP/Be The Match Histocompatibility Advisory Group
• Reviews and approves the use of donor-recipient specimens from the Research Repository in CIBMTR studies
• In person twice annually, in summer and at the BMT Tandem Meetings
Appendix C6
Clinical Trials Advisory Committee
• Assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT
• In person annually at the BMT Tandem Meetings
• By teleconference as needed
Appendix C7
CIBMTR 2015 Annual Report 1.0 WHO WE ARE
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1.3.1 Scientific Working Committees
To ensure broad input into the research process and efficient use of resources, the CIBMTR facilitates 15 Scientific Working Committees focused on specific research areas.
Scientific Working Committees Acute Leukemia
Autoimmune Diseases and Cellular Therapies
Chronic Leukemia
Donor Health and Safety
Graft Sources and Manipulation
Graft-versus-Host Disease
Health Services and International Studies
Immunobiology
Infection and Immune Reconstitution
Late Effects and Quality of Life
Lymphoma
Pediatric Cancer
Plasma Cell Disorders and Adult Solid Tumors
Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders
Regimen-Related Toxicity and Supportive Care
Total Working Committee membership exceeds 2,300 researchers. Membership is open to any researcher willing to take an active role in developing and conducting studies that use CIBMTR data and / or resources. While most of these individuals are HCT clinicians, statisticians and basic scientists also participate. PhD-level statistical faculty and Master’s-level statisticians from the CIBMTR Coordinating Center provide their unique expertise in data analysis. Basic scientists investigating human leukocyte antigen (HLA), immunogenetics,
pharmacogenetics, stem cell biology, and other areas related to HCT provide essential expertise in their respective research areas. The Working Committee structure encourages a collaborative but rigorous methodological approach to all CIBMTR activities.
Working Committee Leadership Chairs (usually 3-4)
MD Scientific Director
PhD Statistical Director
MS-level Statistician
Working Committee leadership is listed in Appendix C5.
1.3.1.1 Working Committee Chairs
Working Committee Chairs are appointed by the Advisory Committee to non-renewable five-year terms. Appointments are made each fall, with terms commencing on March 1 of the following year. Terms are staggered to facilitate succession and maintain continuity. Individuals may serve as Chair more than once but not consecutively for the same committee. The Nominating Committee polls the CIBMTR membership for potential Chair candidates for open Chair positions. Current Chairs participate in the nomination process for replacement positions and give special consideration to promising junior investigators, thus promoting ongoing leadership for the work of the CIBMTR.
Working Committee Chairs provide subject matter expertise in autologous and allogeneic transplantation as well as understanding of CIBMTR organization and procedures. They must be members of CIBMTR centers that submit CRFs and that are compliant with Continuous Process Improvement (CPI) standards for data submission, unless an exception is granted by the Advisory Committee. Chairs are occasionally selected from outside these guidelines for their specific
CIBMTR 2015 Annual Report 1.0 WHO WE ARE
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scientific expertise, for example, a scientist who directs a histocompatibility laboratory, apheresis center, or donor registry, who is committed to the CIBMTR and to the field of HCT.
Chairs monitor and facilitate the progress of studies in their Working Committee’s portfolio. They communicate with Principal Investigators to address barriers and / or delays in study completion and participate in weekly CIBMTR Coordinating Center study critiques when studies in their portfolios are being discussed. In addition to chairing annual Working Committee meetings, Chairs meet by teleconference every four to six weeks with their committee’s Scientific Director and biostatisticians to review the progress of study proposals and ongoing studies. Chairs lead the annual Working Committee meeting and, using input from that meeting, prioritize studies and set the research agenda for the following year.
CIBMTR 2015 Annual Report 2.0 WHAT WE DO
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2.0 WHAT WE DO
The CIBMTR collects data for approximately 21,000 new transplant patients annually as well as a continually increasing volume of follow-up data on previously reported recipients and donors. Submission of outcomes data is mandatory for allogeneic transplants in the US and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and about 80% of US autologous transplants are reported.
The CIBMTR Research Database contains information on >425,000 patients. The distribution of patients in the database is displayed by graft type in Figure and Table 2.1 and by disease in Figure and Table 2.2.Figure 2.3 shows the continued growth in the number of patients registered with the CIBMTR.
Publications
There were 76 CIBMTR publications in 2015. As of December 31, an additional 17 manuscripts were submitted and are under review. A complete list of publications is provided in Appendix D.
Presentations
In 2015, CIBMTR study investigators presented 65 abstracts (41 oral and 24 poster) at national and international conferences, a 50% increase from last year. Presentations included 25 (17 oral and 8 poster) at the American Society for Hematology (ASH) Annual Meeting and 17 (13 oral and 4 poster) at the BMT Tandem Meetings. A complete list of presentations is provided in Appendix E.
Figure and Table 2.1. Distribution of Patients in the CIBMTR Research Database by Graft Source
Graft Source Allogeneic Autologous
TED CRF TED CRF Bone Marrow 49,509 58,935 9,885 5,887 Peripheral Blood 73,780 33,848 148,822 34,858 Cord Blood 5,408 8,721 136 8 TOTAL 128,697 101,504 158,843 40,753
CIBMTR 2015 Annual Report 2.0 WHAT WE DO
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Figure and Table 2.2. Distribution of Patients in the CIBMTR Research Database by Disease
Disease Allogeneic Autologous TED CRF TED CRF
Acute myelogenous leukemia 40,448 28,231 5,721 2,379 Acute lymphoblastic leukemia 21,096 16,616 1,089 469 Chronic myelogenous leukemia 13,914 15,163 414 282 MDS / myeloproliferative syndromes 12,805 10,635 182 86 Lymphoma 15,793 8,009 64,110 13,514 Plasma cell disorders 4,141 2,169 57,615 11,594 Other malignant diseases1 6,039 3,794 28,917 12,247 Severe aplastic anemia 5,981 6,753 14 8 Inherited erythrocyte disorders2 6,085 7,133 781 174 Immune deficiency disorders 2,395 3,001 0 0 TOTAL 128,697 101,504 158,843 40,753 1. Includes other leukemia (allogeneic, n=8,279; autologous, n=910), solid tumors
(allogeneic, n=1,554; autologous, n=40,254) 2. Includes inherited erythrocyte disorders (allogeneic, n=8,602; autologous, n=9),
inherited platelet disorder (allogeneic, n=186; autologous, n=4), inborn errors of metabolism (allogeneic, n=2,499; autologous, n=0), histiocytic disorders (allogeneic, n=1,413; autologous, n=16), autoimmune disease (allogeneic, n=125; autologous, n=555), other disorders (allogeneic, n=393; autologous, n=371)
CIBMTR 2015 Annual Report 2.0 WHAT WE DO
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Figure 2.3. Continued Growth in the Number of Patients Registered with the CIBMTR
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2.1 CLINICAL OUTCOMES RESEARCH PROGRAMClinical outcomes research using the CIBMTR Research Database is a core activity of the organization. These studies address a wide range of issues, focusing on questions that are difficult or impossible to address in single-center studies or randomized trials because diseases treated with HCT are uncommon, single centers treat few patients with a given disorder, and not all important questions are amenable to a randomized research design.
2.1.1 Scientific Working Committees
Program Activities
The 15 Scientific Working Committees oversee most of the CIBMTR’s clinical outcomes research. There are currently 176 studies in progress (Table 2.4). These studies and those recently published are detailed in the 2015 Report on Working Committee Studies. For the 2015 BMT Tandem Meetings, the Working Committees reviewed 157 new study proposals, 73 of which were presented and 35 of which were approved. The prioritization and selection process ensures that the most important issues can be addressed in a timely manner.
Publications
In 2015, Working Committee study investigators published 38 manuscripts in peer-reviewed journals, about 50% of the total number of CIBMTR publications this year (Figure 2.5). As of December 31, an additional 15 manuscripts were submitted for publication and are under review. A complete list of Working Committee publications is provided in Appendix D1.
Key Working Committee Publications this Year
Chen Y-B et al. Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous HCT. Biology of Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046-1053. Epub 2015 Feb 13. PMC4426014.
Sorror ML et al. Prospective validation of the predictive power of the HCT comorbidity index: a CIBMTR® study. Biology of Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1479-1487. Epub 2015 Apr 7. PMC4512746.
Orchard PJ et al. HCT for infantile osteopetrosis. Blood. 2015 Jul 9; 126(2):270-276. Epub 2015 May 26. PMC4497967.
Ciurea SO et al. Haploidentical transplant with post-transplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. Epub 2015 Jun 30.
Kollman C et al. The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood. Epub 2015 Nov 2.
Presentations
In 2015, Working Committee study investigators presented 38 abstracts (26 oral and 12 poster), including 19 (11 oral and 8 poster) at the ASH Annual Meeting and 10 (9 oral and 1 poster) at the BMT Tandem Meetings. A complete list of CIBMTR presentations is provided in Appendix E.
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Table 2.4. 2015 Working Committee Studies
Working Committee Studies in Progress Publications Presentations
Acute Leukemia 13 3 4
Autoimmune Diseases and Cellular Therapies 6 0 0
Chronic Leukemia 12 0 3
Donor Health and Safety 11 2 6
Graft Sources and Manipulation 5 3 0
Graft-versus-Host Disease 10 2 2
Health Services and International Studies 12 2 4
Immunobiology 43 6 5
Infection and Immune Reconstitution 9 1 0
Late Effects and Quality of Life 11 2 2
Lymphoma 6 6 7
Pediatric Cancer 4 2 0
Plasma Cell Disorders and Adult Solid Tumors 12 2 2
Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders
10 3 0
Regimen-Related Toxicity and Supportive Care 12 6 3
TOTAL 176 38* 38
*Two studies are joint studies between two committees.
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Funding
Support for the Working Committees is primarily provided by the National Institutes of Health (NIH) grant # U24CA076518 from the National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); and National Institute for Allergy and Infectious Disease (NIAID).
How to Get Involved
Working Committees are collaborative in nature, and all interested individuals are encouraged to participate. Please feel free to attend annual in-person meetings of the Working Committees at the BMT Tandem Meetings in February. Additionally, anyone willing to follow the study development and management process (Appendix F) is eligible to propose a study to the Working Committees (Figure 2.6).
Successful Working Committee Study Proposals are
Feasible. Utilize data available in the CIBMTR Research Database.
Unique. Fill a gap not addressed by current studies or publications.
Important. Impact the field by improving transplant procedures or results.
See the CIBMTR How to Propose a Study webpage for additional guidelines and advice as well as the Study Proposal Outline.
Figure 2.5. 2015 Publications by Program
Clinical Outcomes -
Working Committees
(38)
BMT CTN (8)
Coordinating Center
(14)
Bio- informatics
(9)
Statistical Methodology
(5)
Health Services (4)
Other (9)
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Figure 2.6. Working Committee Study Proposal Review Process
Submission
• By November 15, study investigator submits proposal to the CIBMTR Coordinating Center for consideration at the next BMT Tandem Meetings.
Initial Review
• Working Committee Leaderships reviews for feasibility with CIBMTR data, potential conflict with active studies, scientific merit, and ability to complete the study in a timely fashion. Researchers with similar concepts may be advised to combine their proposals.
Preliminary Assessment
• If Working Committee Leadership clears the proposal to move forward, the MS-level Statistician contacts the study investigator and prepares a table of characteristics of patient data based on the population defined in the proposal.
Presentation
• Study investigator presents the proposal at the Working Committee meeting at the February BMT Tandem meetings.
Voting
• Working Committee members vote for each proposal, assigning a scientific impact score to each.
Final Approval
• Working Committee Leadership utilizes member feedback in determining which proposals to pursue. Advisory Committee approves the CIBMTR research agenda.
Notification
• Working Committee Leadership contacts study investigator to notify of study approval / rejection by the end of April.
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2.1.2 Cellular Therapies and Non-Transplant Therapy Research Initiatives
With the consensus and support of its Advisory Committee, the CIBMTR is committed to collecting data on cellular therapies and non-transplanted patient populations. The CIBMTR amended its registration of cases to accommodate data collection for cellular and other therapies that may not involve transplantation. Additionally, the CIBMTR is working collaboratively with the Primary Immune Deficiency Disease Consortium to add transplant outcomes data to data on non-transplant therapy collected by the Consortium.
Cellular Therapies
In 2015, the CIBMTR initiated a Cellular Therapies Task Force charged with determining appropriate content and structure for cellular therapies data collection. The CIBMTR also hosted a Cellular Therapies Forum in October 2015 to discuss how to further develop and provide recommendations for a cellular therapy registry. Participants included physicians, scientists, manufacturers, and government representatives.
Cellular Therapies Forum Discussion Topics
Cell therapy for treatment of viral infection
CAR-T cells for malignancies
Manufacturing models
Regulatory framework and long-term outcomes reporting
NCI Cancer Immunotherapy Trials Network
Proposed CIBMTR Cellular Therapy Registry
Myelodysplastic Syndrome
The CIBMTR collects data on patients receiving non-HCT therapy for myelodysplastic syndrome (MDS) to compare with an ongoing study of HCT outcomes. By combining its resources with those of the BMT CTN (Section 2.3.1), the CIBMTR is able to do this in a cost-effective manner. Through November 2015, 34 centers have been activated and 141 patients enrolled on this non-HCT therapy study.
The HCT outcomes study was launched in 2010 because many elderly patients with MDS were denied access to HCT therapy in the US due to lack of Medicare insurance coverage by the Centers for Medicare and Medicaid Services (CMS). To help secure Medicare coverage for these patients, the CIBMTR, NMDP/Be The Match, American Society for Blood and Marrow Transplantation (ASBMT), and other organizations partnered with CMS to develop a Coverage with Evidence Development (CED) study, using data in the CIBMTR Research Database that are collected to fulfill SCTOD requirements. The CED approach allows CMS to provide coverage for procedures and to advocate for clinical studies that inform policy decisions. The CMS-approved study now has >2,300 patients enrolled.
Assessment of HCT in Medicare Beneficiaries with MDS
127 centers
1,294 patients ≥65 years old
815 patients 55-64 years old
213 patients <54 years old
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2.2 IMMUNOBIOLOGY RESEARCH PROGRAMThe CIBMTR maintains a Research Repository of paired tissue samples from donors and recipients, both unrelated and related. The Immunobiology Research group manages the Research Repository inventory and immunogenetic testing programs that add critical HLA and killer-cell immunoglobulin-like receptors (KIR) data for use in CIBMTR clinical outcomes studies.
The CIBMTR leverages the NMDP/Be The Match’s investment in the Unrelated Donor Research Repository with the NIH’s investment in the CIBMTR Research Database. Linking outcomes data to immunologic data available in the Research Repository supports studies that include genetic and immunobiologic data and clinical phenotype data.
The Related Donor Research Repository, supported by the Health Resources and Services Administration (HRSA), is a unique opportunity to enhance immunobiologic research. Related donor and recipient samples are better matched than unrelated recipients for HLA, a measure of immunological compatibility, thus reducing the confounding effects of HLA disparity in clinical research.
The combination of the Unrelated Donor and Related Donor Research Repositories facilitates an organized approach to studying transplant biology across the full spectrum of allogeneic HCT.
Program Activities
In 2015, 175 centers (128 transplant centers, 32 donor centers, and 15 cord blood banks) provided samples to the Research Repository. The Immunobiology Research group enhanced the Research Repository inventory and Immunogenetic Database this year by completing high resolution HLA and presence / absence KIR typing on 226 related and 2,772 unrelated HCT donor / cord and recipient
Research Repository
2,043,345 aliquots
18,901 cell lines
59,375 samples from unrelated donors and 5,128 from related donors
57,018 samples from unrelated recipients and 5,377 from related recipients
10,566 samples from unrelated cord blood units
Samples from complete pairs:
33,919 from complete unrelated adult donor-recipient pairs
4,545 from complete related donor-recipient pairs
3,579 from unrelated cord-recipient pairs
pairs, bringing the total to >17,500 unrelated donor / cord and recipient pairs that have been retrospectively high resolution typed for HLA-A, -B, -C, -DRB1 and -DQB1; >70% include -DPB1, and >10,000 include KIR.
The Immunobiology Research group consolidated all collections (unrelated, related, and clinical trial) under one inventory management system in 2015 and incorporated their information into the Integrated Data Warehouse to facilitate linkage of samples to clinical data. Additionally, the group distributed 8,770 research samples in support of Working Committee studies this year.
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Publications
In 2015, Immunobiology Working Committee study investigators published 6 manuscripts, 4 of which utilized research samples and immunobiology clinical outcomes data. The Immunobiology Research group also supports other investigators’ publications by providing research samples. 9 manuscripts published this year by investigators from other Working Committees and the BMT CTN utilized samples from the Research Repository. A complete list of Working Committee publications is provided in Appendix D1.
Presentations
In 2015, 5 Immunobiology Working Committee study investigators presented at national and international conferences, including 2 oral abstracts at the European Group for Blood and Marrow Transplantation (EBMT) Annual Meeting and 2 posters at the ASH Annual Meeting. A complete list of CIBMTR presentations is provided in Appendix E.
Funding
Support for the Immunobiology Research Program is primarily provided by the Office of Naval Research grant # N00014-14-1-0848, NIH grant # U24CA076518, and HRSA contract # HHSH250201200016C.
The Immunobiology Research Program offers limited research funds supporting immunobiology research studies. The grants are intended to subsidize lab tests, sample collection, or costs associated with the use of research samples. These grants are available to approved CIBMTR studies that support organizational research priorities. For additional information, visit the CIBMTR Grants for Immunobiology Research webpage.
Key Immunobiology Publications this Year
Gadalla SM et al. Association between donor leukocyte telomere length and survival after unrelated allogeneic HCT for severe aplastic anemia. Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. PMC4388056.
Verneris MR et al. HLA mismatch is associated with worse outcomes after unrelated donor reduced-intensity conditioning HCT: an analysis from the CIBMTR. Biology of Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1783-1789. Epub 2015 Jun 6. PMC4568127.
Petersdorf EW et al. High HLA-DP expression and graft-versus-host disease. New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. PMC4560117.
How to Get Involved
All interested parties may attend the annual in-person meeting of the Immunobiology Working Committee at the BMT Tandem Meetings in February. Additionally, the Immunobiology Working Committee encourages highly translational, hypothesis-driven proposals through the Working Committee Study Proposal Review Process (Figure 2.6).
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2.3 CLINICAL TRIALS SUPPORT PROGRAMUsing data from observational studies to support decisions regarding design of prospective clinical trials and / or amendments of such trials, the CIBMTR Research Database provides an important resource to the Clinical Trials Support Program. This program consists of the BMT CTN and RCI BMT, which conduct multicenter clinical trials. Data from the Research Database are used to design, monitor, and analyze these trials.
CIBMTR Coordinating Center Support of Clinical Trials
Trial Planning. Determine patient populations available for trials as well as appropriate inclusion criteria and outcome estimates, and identify transplant centers likely to accrue.
Data Collection. Share data to reduce reporting for transplant centers, and share data collection forms and database structures developed by transplant experts.
Statistical Consultation. Provide expert statistical review of protocols.
Real-Time Accrual Assessment. Review data to evaluate center and patient characteristics of enrolled and non-enrolled patients to address accrual barriers.
Trial Interpretation. Evaluate results of clinical trials, including through the provision of matched controls.
Long-Term Follow-Up Data. Capture follow-up data for long-term or secondary analyses, resulting in considerable cost-savings.
2.3.1 Blood and Marrow Transplant Clinical Trials Network
The BMT CTN, sponsored by NHLBI and NCI, is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation, a contract research organization based in Rockville, MD.
Program Activities
The BMT CTN has launched 37 trials (3 this year), and completed accrual for 28 of these trials (2 this year). The Network has accrued >8,500 patients to its trials from >100 centers, including >1,100 this year. Among trials currently open for enrollment, the BMT CTN achieved an overall accrual rate in 2015 that is approximately 125% of projections. The Network has established a Research Sample Repository that currently includes >350,000 biospecimens. Additionally, the BMT CTN has conducted 48 ancillary and correlative studies.
More detail regarding Network activities and protocols is provided in the annual progress report on the BMT CTN website. A list of Network trials open for enrollment is provided in Appendix G1.
Publications
In 2015, BMT CTN study investigators published 8 manuscripts, 2 of which were primary results papers. These bring the total number of Network publications to 57, including 16 primary results papers. A complete list of 2015 BMT CTN publications is provided in Appendix D2.
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Key BMT CTN Publications this Year
MacMillan ML et al. A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biology of Blood and Marrow Transplantation. 2015 Apr 1; 21(4):761-767. Epub 2015 Jan 10. PMC4359643.
Khera N et al. Comparison of characteristics and outcomes of trial participants and nonparticipants: example of BMT CTN 0201 trial. Biology of Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1815-1822. Epub 2015 Jun 11. PMC4568172.
Anderlini P et al. Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study. Lancet Haematology. 2015 Sep 1; 2(9):367-375. Epub 2015 Sep 8.
Devine SM et al. Phase II study of allogeneic transplantation for older patients with acute myeloid leukemia in first complete remission using a reduced-intensity conditioning regimen: results from CALGB 100103 / BMT CTN 0502. Journal of Clinical Oncology. Epub 2015 Nov 2.
Presentations
BMT CTN study investigators presented 11 abstracts (8 oral and 3 poster) at national and international conferences in 2015, two of which were highlighted at the ASH Annual Meeting, one as a Late Breaking Abstract and the other as a 2016 Highlight of ASH. These bring the total number of Network presentations to 59. A complete list of 2015 CIBMTR presentations is provided in Appendix E.
Funding
Support for the BMT CTN Data and Coordinating Center is provided by the NIH grant # U10HL069294 from the NHLBI and NCI.
How to Get Involved
The Network is committed to widespread participation in its trials. If you would like to serve as an Affiliate Center, visit the BMT CTN website for more information. Additionally, you may act as a Center Principal Investigator or champion a trial to increase patient accrual at your Center, serve on a Protocol Team or an Endpoint Review Committee, or act as a Medical Monitor. You may also propose an ancillary study, which uses data, biospecimens, and / or analyses outside the specific objectives of a primary BMT CTN study.
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2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation
The RCI BMT provides researchers in the field of HCT with infrastructure and expertise in HCT clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting with groups such as the BMT CTN or the national cancer cooperative groups.
Program Activities
The RCI BMT has launched 15 trials, including 3 this year. In 2015, the RCI BMT accrued just under 3,000 patients, bringing the total number of accrued patients to approximately 28,000, of which >21,000 were enrolled in a cohort study examining long-term outcomes of unrelated donors.
The RCI BMT is currently managing 2 Food and Drug Administration (FDA) investigational new drug (IND) protocols for NMDP/Be The Match Operations. Peripheral Blood Stem Cell (PBSC) Procurement accrued >2,100 patients this year, and Cord Blood Access accrued >500. Additionally, the RCI BMT supported 5 studies involving unrelated donor data or sample collection for investigators.
In 2015, the RCI BMT launched two new interconnected software systems to enhance data collection and study management. These systems will increase efficiency, allowing the RCI BMT to effectively support a wide array of clinical trials and research studies.
A complete list of RCI BMT trials is provided in Appendix G2.
Presentations
In 2015, RCI BMT study investigators presented 4 oral abstracts at national and international conferences. A complete list of CIBMTR presentations is provided in Appendix E.
Survey Research Group
The Survey Research Group is a team within the RCI BMT created to assist HCT researchers in developing and conducting research involving questionnaires, direct subject interviews, and patient reported outcomes. The group is responsible for collecting high quality, scientifically valid data from donors, patients, and their families. The Survey Research Group utilizes standardized and semi-structured telephone interviews as well as self-administered questionnaires. In 2015, the Survey Research Group supported 8 active studies and participated in the development of 1 upcoming study.
Funding
Support for the RCI BMT is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID; NMDP/Be The Match; and corporate and private sponsors of specific studies.
The RCI BMT team can work with study investigators to seek funding from a variety of sources, including government agencies, foundations, pharmaceutical companies, and private corporations.
How to Get Involved
Study investigators may solicit clinical trials services from the RCI BMT, including assistance with funding proposals; protocol development and approvals; management of study conduct; data auditing, management, and analysis; and financial administration. Study investigators may also contract for specific services as needed, such as support with surveys, site selection and management, sample management, and more. For additional information, visit the RCI BMT webpage.
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2.4 HEALTH SERVICES RESEARCH PROGRAMHealth services research is the multi-disciplinary field of scientific investigation that studies how social factors, financial systems, organizational structures and processes, technology, and behavior affect treatment outcomes, quality, and cost. The CIBMTR conducts research through the Health Services Research Program in collaboration with NMDP/Be The Match’s Patient and Health Professional Services. Research activities complement those of the CIBMTR’s Health Services and International Studies Working Committee.
Health Services Focus Areas Research
Health Policy
System Capacity Initiative
Program Evaluation
Clinical Trial Support
Training, Education, and Consultation
Program Activities
Research. The Health Services Research Program currently has 9 studies in progress. In 2015, the program completed analysis for 3 research studies.
Health Policy. In collaboration with NMDP/Be The Match’s Payer Policy department, the Health Services Research Program conducted a study, Financial barriers to HCT: the transplant center perspective, to examine the impact of the Patient Protection and Affordable Care Act on access to HCT and treatment decision-making.
Select Health Services Research Studies in Progress
Individualized care plans for HCT survivors
Cost-effectiveness of HCT vs. alternative therapy for acute myeloid leukemia (AML) in patients aged 60-70 years
Analysis of reimbursement for HCT in older patients
HCT multidisciplinary care teams: burnout, moral distress, and career satisfaction
Payer-partnered approach to community-based referral for HCT
Easy-to-read informed consent forms for HCT clinical trials (BMT CTN 1205)
System Capacity Initiative. The goal of the System Capacity Initiative is to determine how the US healthcare system can accommodate the growing number of patients in need of HCT. In 2015, the Health Services Research Program continued to support initiative-related efforts, including the study on HCT workforce burnout.
Program Evaluation. The Health Services Research Program conducted >30 evaluations for the HRSA Office of Patient Advocacy / Single Point of Contact contract and NMDP/Be The Match programs in 2015, including a needs assessment for limited English proficiency patients.
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Clinical Trial Support. A collaboration between the Health Services Research Program and BMT CTN, Easy-to-read informed consent forms for HCT clinical trials evaluates the effectiveness of a novel consent form and describes barriers to implementation. The program also provided technical writing support and developed 3 easy-to-read consent / assent forms and 6 patient information sheets for BMT CTN protocols. Training, Education, and Consultation. In 2015, the Health Services Research Program established the HCT Health Economics Interest Group, which held its first meeting at the 2015 BMT Tandem Meetings. More detail regarding program activities is provided in the Health Services Research Annual Report.
Publications
In 2015, Health Services Research study investigators published 4 manuscripts in peer-reviewed journals. A complete list of program publications is provided in Appendix D3.
Presentations
Through the HRSA Office of Patient Advocacy / Single Point of Contact contract, the Health Services Research Program is charged with disseminating findings to all stakeholder groups. In 2015, program investigators presented 3 oral abstracts at the ASH Annual Meeting. A complete list of research-focused CIBMTR presentations is provided in Appendix E.
How to Get Involved
For more information about the Health Services Research Program and / or the HCT Health Economics Interest Group, contact Ellen Denzen, MS, Senior Manager, at [email protected] or 612.884.8562.
Health Services Research Publications this Year
Besse KL et al. Estimating demand and unmet need for allogeneic HCT in the US using geographic information systems. Journal of Oncology Practice. 11(2):e120-e130. Epub 2015 Mar 1. PMC4371120.
Majhail NS et al. National survey of HCT center personnel, infrastructure and models of care delivery. Biology of Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1308-1314. Epub 2015 Mar 31. PMC4466059.
Clauser SB et al. Patient centeredness and engagement in quality-of-care oncology research. Journal of Oncology Practice. 2015 May 1; 11(3):176-179. Epub 2015 Apr 7.
Preussler JM et al. Patient housing barriers to HCT: results from a mixed-methods study of transplant center social workers. Supportive Care in Cancer. Epub 2015 Aug 15.
Funding
Support for the Health Services Research Program is provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID. Individual studies are funded via a variety of mechanisms. Individualized care plans for HCT survivors is supported by the Patient Centered Outcomes Research Institute award # CD-12-12-4062; Easy-to-read informed consent forms for HCT clinical trials is supported by the NHLBI grant # U10HL069294-12S1, and A payer-partnered approach to community-based referral for HCT is supported by the grant # 11762021 from the National Comprehensive Care Network / Pfizer.
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2.5 BIOINFORMATICS RESEARCH PROGRAMThe Bioinformatics Research Program provides expertise in, and conducts research on, translational and operational bioinformatics.
Program Activities
Current Bioinformatics Research Goals
Develop pipelines to analyze Next Generation Sequencing typing data, including full-gene HLA, KIR, and genome-wide sequencing, to refine our understanding of genetic matching
Investigate the role of genetic ancestry in transplantation, including the best way to match individuals with multiple races in their family tree
Develop data standards and tools for making immunogenetic data portable for research and clinical use
Investigate HLA data from other countries to better understand global frequencies and improve matching
Develop methods for HLA association studies
Publications
In 2015, Bioinformatics study investigators published 9 manuscripts in peer-reviewed journals. A complete list of program publications is provided in Appendix D4.
Presentations
Bioinformatics study investigators presented 11 abstracts (5 oral and 6 poster) at national and international conferences in 2015. A complete list of CIBMTR presentations is provided in Appendix E.
Key Bioinformatics Publications this Year
Slater N et al. Power laws for heavy-tailed distributions: modeling allele and haplotype diversity for the NMDP. PLoS Computational Biology. 11(4):e1004204. Epub 2015 Apr 22. PMC4406525.
Besse K et al. On modeling HLA-identical sibling match probability for alloHCT: estimating the need for an unrelated donor source. Biology of Blood and Marrow Transplantation. Epub 2015 Sep 5.
Buck K et al. 7/8 and 9/10 or better high resolution match rate for the Be The Match® Unrelated Donor Registry. Biology of Blood and Marrow Transplantation. Epub 2015 Dec 24.
Funding
Support for the Bioinformatics Research Program is primarily provided by the grant # N00014-14-1-0848 from the Office of Naval Research as well as several grants from the NIH.
How to Get Involved
For more information about the Bioinformatics Research Program, visit the NMDP/Be The Match Clinical Bioinformatics webpage or contact Martin Maiers, Director, at [email protected] or 612.627.5892.
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2.6 STATISTICAL METHODOLOGY RESEARCH PROGRAMThe CIBMTR has enjoyed a positive, collaborative association with the Division of Biostatistics in the MCW Institute for Health and Society since 1980, an association that is a distinctive asset and crucial to the success of CIBMTR research. This long-standing relationship has many benefits. Biostatisticians ensure the statistical integrity of CIBMTR scientific activities, contribute to results in articles on HCT-related statistical issues for clinical audiences, and support Working Committee study investigators in developing scientific study protocols using CIBMTR data. CIBMTR biostatisticians have pioneered novel methodologic approaches to analyzing HCT data.
Program Activities
HCT is a complex process with multiple competing risks and dramatic changes in the risks of specific events over time. The CIBMTR has developed and evaluated the statistical models used in HCT research and helped guide the research community in appropriate application and interpretation of these sophisticated models.
Statistical Methodology Research Goals
Develop new statistical models
Compare new statistical models with existing solutions using the CIBMTR Research Database
Publications
In 2015, PhD-level biostatisticians in the Statistical Methodology Research Program published 4 peer-reviewed manuscripts and 1 editorial. A complete list of program publications is provided in Appendix D5.
Key Statistical Methodology Publications this Year
Wang T et al. A re-formulation of generalized linear mixed models to fit family data in genetic association studies. Frontiers in Genetics. 6(120):1-10. Epub 2015 Mar 31. PMC4379931.
Eriksson F et al. The proportional odds cumulative incidence model for competing risks. Biometrics. 2015 Sep 1; 71(3)687-695. Epub 2015 May 26. PMC4608382.
Brazauskas R, Logan BR. Observational studies: matching or regression? Biology of Blood and Marrow Transplantation. Epub 2015 Dec 19.
Funding
Support for the Statistical Methodology Research Program is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID and the HRSA contract # HHSH250201200016C.
How to Get Involved
During the BMT Tandem Meetings in February, PhD-level biostatisticians plan and present educational sessions related to statistical design and analysis, and they provide 1:1 statistical consultation to researchers writing proposals or developing protocols for CIBMTR studies. Any interested individual may participate in these sessions. Additionally, the MCW Division of Biostatistics presents a lecture series and a seminar series throughout the year in Milwaukee.
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2.7 STEM CELL THERAPEUTIC OUTCOMES DATABASE (SCTOD)The CIBMTR administers the SCTOD for the HRSA-sponsored C.W. Bill Young Cell Transplantation Program, established by the Stem Cell Therapeutic and Research Act of 2005. Continued support for the SCTOD is provided through the Stem Cell Therapeutic and Research Reauthorization Act of 2010.
C.W. Bill Young Cell Transplantation Program Goals Fulfilled by the SCTOD
Collect, analyze, and report outcomes data for all allogeneic transplants and other therapeutic uses of blood stem cells
Publicize information about HCT to patients, families, health care professionals, and the public
Define better processes for identifying unrelated matched marrow donors, PBSC donors, and cord blood units through one electronic system
Increase availability of unrelated adult volunteer donors and cord blood units
Expand research to improve patient outcomes
Program Activities
Annually, the CIBMTR publishes HCT volumes and performance data by transplant center and provides public access to this information via the HRSA Blood Cell Transplant website.
Center-Specific Volumes and Survival Analysis. As part of the contract to operate the SCTOD, the CIBMTR provides the annual volume of transplants performed at each center and performs a center-specific survival analysis comparing the one-year survival rates among US centers. The report assesses transplants from both related and unrelated
SCTOD Contract Requirements
Collect HCT outcomes data for:
• All allogeneic HCTs performed in the US using related or unrelated donors
• All allogeneic HCTs worldwide that use grafts procured through the C.W. Bill Young Cell Transplantation Program
• Clinical applications of hematopoietic stem cells other than hematopoietic cell recovery
Use the data collected for the SCTOD to evaluate the performance of transplant centers
Provide specific SCTOD data to the public
Collect a basic set of data for analyses of program use, center-specific outcomes, donor registry, cord blood inventory size, and patient access to HCT
Establish a Related Donor-Recipient Research Sample Repository (Section 2.2)
donors. The most recent report was finalized in September 2015 and contains information on all first allogeneic transplants performed in US centers from January 1, 2011, through December 31, 2013.
Center Outcomes Forums. The CIBMTR has conducted 4 Center Outcomes Forums to engage relevant stakeholders in the center-specific outcomes reporting process. The most recent meeting was held in June 2014 and generated recommendations to improve risk adjustment, enhance future collection of relevant data elements, and develop tools to enhance centers’ quality improvement efforts.
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Quality of Life Assessments. In 2011, the CIBMTR launched a pilot program at 5 adult and 3 pediatric transplant centers. The principal goal was to test center-based recruitment of patients to a quality-of-life data collection program, followed by communication between the CIBMTR and the patient to collect longitudinal quality-of-life information. This pilot program is unique in that the CIBMTR collects data directly from patients after HCT. Accrual closed in 2013 with 390 patients enrolled. Data analysis is in progress.
Cellular Therapies for Regenerative Medicine (CTRM). The CTRM data repository tracks novel uses of blood stem cells. The SCTOD contract mandates data collection on uses of cells found in bone marrow, peripheral blood, and umbilical cord blood for alternative therapeutic applications, including regenerative medicine.
The CTRM data repository captures uses of cells for the treatment of diseases without the intention of replacing the recipient’s hematopoietic function. These therapies include, but are not limited to, treatment of malignancies as well as infectious, cardiovascular, rheumatologic, neurologic, musculoskeletal, and endocrinologic diseases with the intent to improve organ function. As of November 30, the CTRM data repository includes data for 983 patients.
The CIBMTR anticipates the expansion of the cellular therapy field with the use of not only hematopoietic derived cells but also cells from other tissues; therefore, it is expanding the capability and flexibility of data collection in this rapidly changing area. In October, the CIBMTR held a Cellular Therapy Forum (Section 2.1.2) to discuss how to further develop its cellular therapy registry.
Study Summaries for Patients. In conjunction with Be the Match’s Patient and Health Professional Services and the Consumer Advocacy Committee, the CIBMTR publishes lay summaries of CIBMTR publications for patients and their loved ones. In 2015, the CIBMTR published 12 patient-friendly research summaries.
Funding
Support for the SCTOD is provided by the HRSA contract # HHSH250201200016C.
How to Get Involved
All US centers performing allogeneic HCTs provide data to the CIBMTR for the SCTOD. These data are used to generate reports, which are distributed to transplant center medical directors and posted on the HRSA Blood Cell Transplant website.
Publicly Available Reports developed by the CIBMTR for the SCTOD
Transplant Outcomes
• US Patient Survival Report
• US Transplant Data by Center Report
• US Transplant Data by Disease Report
Transplant Activity Report
These reports are available on the HRSA Blood Cell Transplant website
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2.8 CORPORATE PROGRAMThe CIBMTR Corporate Program provides opportunities for industry collaborators to access CIBMTR data and statistical support to address questions specific to their business needs through Corporate Membership as well as Corporate Studies and Projects.
Corporate Membership. The CIBMTR Corporate Membership program provides a variety of resource materials to corporations needing access to the most current and comprehensive data on HCT. These materials are useful for Marketing Managers, Medical Directors, Research Directors, Product Managers, Case Managers, and Transplant Coordinators. There are four Corporate Membership levels available, each described on the CIBMTR Corporate Membership Program webpage.
Corporate Membership Benefits
CIBMTR Report on Survival Statistics for BMT
Center Volumes Dataset
Worldwide CIBMTR Directory of BMT Physicians
Reduced registration rates at CIBMTR meetings and educational forums, including the BMT Tandem Meetings
Access to CIBMTR data and resources
Corporate Studies and Projects. Corporate partners may contract with the CIBMTR to conduct a study, support a project involving more complex analyses, or license a specified data set. Organizations interested in funding a study, such as one comparing HCT with one or more non-HCT therapies, or using historical controls, can negotiate with the CIBMTR for data and / or expert statistical analyses.
Program Activities
The CIBMTR engaged in 8 studies with corporate partners in 2015. Currently 14 organizations participate in the CIBMTR Corporate Membership program, including 9 that joined this year.
How to Get Involved
If you would like to learn more about the CIBMTR Corporate Program, visit the CIBMTR Corporate Membership webpage or contact Sherry Fisher, Director of Advancement, at [email protected] or 414.805.0687. If you are a Corporate Member requesting analyses, please complete the Corporate Member Information Request Form.
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3.0 HOW WE SHARE KNOWLEDGE
The CIBMTR is committed to sharing the data we collect as well as the information and knowledge produced from our data and our extensive collaborations with investigators in the HCT field.
The CIBMTR shares its knowledge in different ways. To determine the best way to access specific types of CIBMTR knowledge, review Figure 3.1 and Tables 3.2-3.5.
Figure 3.1. How to Access CIBMTR Knowledge
Details provided in Tables 3.2-3.5.
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Table 3.2. How to Access CIBMTR Information*
Information
GENERAL In addition to reviewing this report, access the Summary of Accomplishments on the Administrative and Progress Reports webpage. Read editions of the quarterly newsletter on the Newsletters webpage, and email cibmtr-news@mcw. edu to be added to the electronic distribution list.
ACTIVITIES Review Section 2 or visit the What We Do webpage. Visit the Studies webpage, SCTOD webpage, or Corporate Support webpage to learn more about CIBMTR research programs, the SCTOD, or Corporate Program, respectively.
PUBLICATIONS Review the CIBMTR’s >1,000 publications on the Publication List webpage. For lay summaries of selected CIBMTR publications, written specifically for patients and the lay public, visit the Study Summaries for Patients webpage.
MEETINGS Visit the BMT Tandem Meetings webpage to view agendas, register, reserve housing, and submit abstracts.
TRAINING Review Section 3.4 or visit the Training and Reference webpage to access the Center Reference Guide, Forms Instruction Manual, FormsNet and AGNIS trainings, and online courses.
OTHER Email [email protected].
Table 3.3 How to Access CIBMTR Data*
Data
TYPES Review the baseline and follow-up data available for recipients and donors on the Types of Data Available for Research or Request webpage.
STANDARD REPORTS Access the Summary Slides, BMT Survival Statistics Report, Center Transplant Activity Report, Patient Transplant Outcomes Reports, and Center-Specific Survival Reports via Table 3.6 or on the Slides and Reports webpage.
ELECTRONIC RETURN OF CENTER Utilize the Data Back to Centers application on the Portal site to download TED-level variables that have been validated and processed in the CIBMTR Research Database.
RESEARCH STUDY Propose a study as explained on the How to Propose a Study webpage, or participate in one of the existing studies listed on the Working Committee Study Lists webpage.
CORPORATE ANALYSIS Complete the Corporate Member Information Request Form.
CUSTOM ANALYSIS Complete the Custom Information Request Form.
OTHER Email [email protected].
*If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).
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Table 3.4 How to Access CIBMTR Tools*
Tools
DISEASE RISK INDEX ASSIGNMENT TOOL
Access this tool on the DRI Assignment Tool webpage to categorize patients undergoing allogeneic HCT for hematologic malignancy by disease risk.
PATIENT ONE-YEAR SURVIVAL CALCULATOR FOR ALLOGENEIC TRANSPLANTS
Transplant Center Medical Directors may access this tool on the Portal site to predict one year survival for individual allogeneic HCT recipients.
Table 3.5 How to Access CIBMTR Biospecimens*
Biospecimens
SAMPLES TYPES AND INVENTORY Determine the >2 million samples available in the Research Repository via the Sample Types and Inventory Summary webpage.
REQUESTING SAMPLES For studies that include recipient clinical outcome data, propose a study as explained on the How to Propose a Study webpage. For studies that do not include clinical outcome data, review the How to Request Samples from the Research Sample Repository webpage.
OTHER Email [email protected].
*If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).
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Table 3.6. Standard Reports Published by the CIBMTR
Report Title Description Month(s) Released Format Accessibility*
CIBMTR Summary Slides
Charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT; developed in conjunction with the BMT Tandem Meetings
February PPT Published on the CIBMTR Summary Slides webpage
CIBMTR Progress Report
Information on the CIBMTR's goals and achievements as well as operational details on how the CIBMTR is funded, supported, promoted, and maintained
February PDF Published on the CIBMTR Administrative and Progress Reports webpage
CIBMTR Report of Survival Statistics for BMT
Highly detailed report on survival statistics that describes use and outcome of autologous and allogeneic HCT in the >500 centers that have participated in the CIBMTR
September Word Via Corporate Membership Program (Section 2.8) or by request from physicians for making treatment decisions or clinical investigators planning clinical studies to [email protected]
CIBMTR Summary of Accomplishments
High level summary of CIBMTR fiscal year accomplishments and high impact publications
September PDF Published on the CIBMTR Administrative and Progress Reports webpage
US Centers Annual Transplant Activity Report
Dataset containing center-specific pre-transplant patient-, disease-, and transplant-related characteristics data for nearly all allogeneic and a majority of autologous HCTs performed in the US annually since 2008
September PDF Published on the HRSA Blood Cell Transplant website
US Patient Center-Specific Survival Report
Comparison of observed to expected one-year survival rates among centers in the C.W. Bill Young Cell Transplantation Program network; evaluates outcomes for transplants using both related and unrelated donors
December Web Published on the Be The Match Transplant Center Directory webpage; available as a Word document upon request to [email protected]
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Report Title Description Month(s) Released Format Accessibility*
US Patient Transplant Outcomes
Disseminated in 3 different reports: • US Patient Survival Report: 100-day, 1-
year, and 3-year survival rate estimates for US HCT recipients by disease and donor type
• US Transplant Data by Center Report: Number of bone marrow and cord blood transplants performed at a specific transplant center
• US Transplant Data by Disease Report: Number of bone marrow and cord blood transplants for a specific disease
December Web Published on the HRSA Blood Cell Transplant website
US Allogeneic Transplant Activity Report
Report containing patient, disease, donor HLA match, donor age, and gender match information for allogeneic transplant activity in the US since 2010
January April July
October
PDF Via Corporate Membership Program (Section 2.8)
CIBMTR Newsletter Articles regarding Working Committees, the SCTOD, data management and collection, and noteworthy events in the HCT community
February May
August November
Web Published on the CIBMTR Newsletters webpage and distributed via email; contact [email protected] to be added to the distribution list
Study Summaries for Patients
Summaries of CIBMTR research publications written for patients and others in the lay public
Ongoing PDF Published on the CIBMTR Study Summaries webpage
*If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).
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3.1 INFORMATION REQUEST SERVICEThe CIBMTR Information Request Service provides timely access to data on transplantation to patients, physicians, hospitals, pharmaceutical companies, insurance companies, and others involved in healthcare. Requests range from simple queries of patient, disease, and transplant frequencies to those with greater complexity involving specific data combinations and / or statistical analysis of outcomes.
Potential Reasons for Information Requests
Self-education and decision making
Patient counseling or clinical decision making
Presentation support
Transplant center assessments
Clinical trial planning
Market assessments
Coordinating Center staff members fulfill requests related to clinical decision making within three days and most other requests within three weeks. If a request will take more than an estimated four weeks to fulfill, a Coordinating Center staff member will contact the requestor to discuss an appropriate timeline.
Accomplishments
In 2015, the CIBMTR fulfilled 469 requests for information and data (Table 3.7).
Table 3.7. Data Requests Addressed by the CIBMTR in 2015
Requestor Number of Requests
Physician / Researcher 352
Patient or Relative 41
Pharmaceutical / Biotech Company 38
Market Research Firm 22
Student 6
Patient Advocacy Group 4
News Media 4
Federal Government Agency 1
Cord Blood Bank 1
TOTAL 469
How to Access
For more information about requesting data from the Research Database, visit the CIBMTR How to Request Data webpage. If you would like a one-time, custom analysis, complete the Custom Information Request Form. If you have questions about requesting CIBMTR data, please contact [email protected].
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3.2 INTERNET PRESENCEThe CIBMTR Internet presence provides the scientific community and the public with access to HCT information. Current websites include general information about HCT and CIBMTR activities; training and support; a shared communications and collaborative environment for member centers; and secure web access to CIBMTR data for Working Committees, transplant centers, scientific investigators, and CIBMTR staff.
3.2.1 CIBMTR Public Website
The CIBMTR public website (cibmtr.org) is unrestricted and provides information about the CIBMTR and its research. It supports the Working Committees and BMT CTN with information regarding proposal submission, access to a listing and summaries of all studies in process, and access to a summary of all CIBMTR publications. The website facilitates data and information requests, and it provides access to all current and past data collections forms, training manuals, and videos as well as other materials for both investigators and data professionals. The website information is, in part, supported by DISCO (Data and Information for Statistical Center Operations), an application which maintains data on >750 studies, >1,000 publications, and >1,800 authors and their institutions at time of publication. In 2015, the CIBMTR public website had approximately 418,000 unique page views.
About CIBMTR
Administrative and Progress Reports (663 unique page views in 2015) Provides access to the CIBMTR’s annual Progress Report, annual Summary of Accomplishments, and Manual of Operations.
Studies
Working Committee Studies Lists (4,835 unique page views in 2015) A summary of the planned, in-progress, and recently published clinical outcomes studies for each Working Committee.
Meetings
Annual BMT Tandem Meetings Materials (34,073 unique page views in 2015) Provides access to agendas, handouts, and educational materials from specific meetings at the BMT Tandem Meetings: Working Committee Meetings and Clinical Research Professionals / Data Management Conferences.
Reference Center
Summary Slides (10,654 unique page views in 2015) Includes charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT.
Web-based US Transplant Reports (31,964 unique page views in 2015) Directs users to the Be The Match US Center Listing Report and customizable reports of patient survival and transplant available through the HRSA Blood Cell Transplant website.
Publication List (5,836 unique page views in 2015) Searchable descriptive list of >1,000 publications resulting from the use of CIBMTR data and statistical resources.
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Newsletter (3,199 unique page views in 2015) Published 3-4 times per year. Articles feature updates on Working Committees, the SCTOD, data management and collection, and noteworthy events in the HCT community.
Patient Resources (4,590 unique page views in 2015) Includes lay summaries of CIBMTR research articles as well as post-transplant care recommendations for adult and pediatric autologous and allogeneic HCT recipients to help patients and clinicians understand and plan for the specialized care of transplant recipients. 12 lay summaries were published in 2015.
Statistical Resources (4,183 unique page views in 2015) Provides access to resources offered through the unique partnership between the CIBMTR and MCW Division of Biostatistics, including biostatistical publications, a series of statistical lectures targeted at basic and clinical investigators, and research tools, such as the Disease Risk Index Assignment Tool.
Data Management
Data Management Manual (38,071 unique page views in 2015) A comprehensive reference document for completing CIBMTR data collection forms. The manual also details reporting requirements, describes protocols and the consent process, and includes downloadable versions of report forms.
Data Back to Centers Application (628 unique page views) Links to the Data Back to Centers (DBtC) application that provides CIBMTR member centers the ability to retrieve all the TED-level data their center has reported to the CIBMTR through FormsNet or AGNIS.
Data Collection Forms (47,303 unique page views in 2015) Provides access to current and retired versions of the forms used by the CIBMTR to collect standard data elements for all transplant recipients.
Training and Reference (68,004 unique page views in 2015) Provides access to a wide variety of CIBMTR data management training and reference materials.
3.2.2 CIBMTR Collaborative Site
The CIBMTR Collaborative site (collaborate.cibmtr.org) uses the SharePoint Enterprise Collaboration platform to promote cooperative work among CIBMTR staff members and provides a communication platform for specific studies and initiatives. This site is secured by username and password, and user-specific security credentials are assigned centrally.
The CIBMTR uses the site for storing and sharing protocol and consent documents, donor / recipient tracking tools, confidential committee information, data, manuscript drafts, and other relevant information. While only two Working Committees currently use the Collaborate site on a regular basis, it is available to all Working Committees for sharing information.
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3.2.3 CIBMTR Portal Site
The CIBMTR Portal site (portal.cibmtr.org) delivers applications and data to CIBMTR transplant centers and other partners. In 2015, unique, external visitors viewed 2,675 portal pages. Four applications are currently hosted on this site:
Data Back to Centers
The current DBtC application provides authorized users the ability to download CIBMTR TED-level data variables for their centers. The data have been validated and processed in the CIBMTR Research Database and are reviewed and refreshed quarterly. Legacy International Bone Marrow Transplant Registry (IBMTR) data from as far back as 1964 and some legacy NMDP/Be The Match data from as far back as 1987 are available. In 2015, 628 unique, non-CIBMTR visitors viewed 914 DBtC pages and downloaded data 418 times. In Spring 2016, the CIBMTR plans to release an enhanced DBtC (eDBtC) application that will give centers access to both TED- and CRF-level source data as well as a variety of analytic tools via a user-friendly application called Qlikview.
Center Volumes Portal
The Center Volumes Portal allows centers to preview; correct, if necessary; and approve center volume data published annually to the HRSA Blood Cell Transplant website. The CIBMTR gives centers access to display and download the previous six years (2009-2014) of volume data as well as the current year under review (2014). In 2015, 188 unique, external visitors viewed 706 Center Volumes Portal pages.
Patient One-Year Survival Calculator – Allogeneic Transplants
Accessible by medical directors, the Patient One-Year Survival Calculator for Allogeneic Transplants provides centers with a tool to predict one year survival for individual allogeneic HCT recipients. The calculator data are updated annually to reflect new information contained in the center outcomes analysis. In 2015, 448 unique, non-CIBMTR visitors viewed 1,357 survival calculator pages.
Qlikview
In 2015, the CIBMTR selected Qlikview, a third party business intelligence tool, to provide transplant centers with self-service access to their data and information, including descriptive statistics, outcomes, and trends. Using Qlikview, centers will be able to more readily visualize their data in predefined dashboards, interact with their data through ad hoc analytics, and export their data to files that will support further analysis. The CIBMTR will introduce two Qlikview applications at the 2016 BMT Tandem Meetings: eDBtC, which will provide transplant centers with access to their TED- and CRF-level data, and Center-Specific Analysis Tool, which will provide transplant centers with access to their SCTOD database data.
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3.2.4 Be The Match Public Website
The Be The Match Public website (bethematch.org) is designed for patients and families, donors, and supporters. It incorporates detailed information about transplantation and donation written for the public. The website provides scientific information in lay terms for donors related to the donation process and for patients related to specific diseases, various treatment options, the process of transplantation, and life after transplant. It also addresses concerns related to specific populations, including children and caregivers. The CIBMTR collaborates with NMDP/Be The Match to provide content for several areas of this website, including data for the US Center Listing Report.
US Center Listing Report
Transplant Center Directory Provides transplant center specific information about facilities, personnel, diseases treated, cost, and transplant experience, including the number of transplants performed and survival rates by age, disease type, and disease stage.
3.2.5 Be The Match Clinical Website
The Be The Match Clinical website (bethematchclinical.org) is designed for clinicians, network participants, payors, and bioinformatics professionals. For clinicians, the website provides access to evidence-based tools, clinical guidelines, outcomes data, and education courses on HCT. The website also provides information specific to types of network participants: transplant centers, donor centers, apheresis and collection centers, and cord blood banks. For payors, the website offers information to help individuals understand BMT, determine coverage, and answer employer and patient questions. Related to bioinformatics, the website provides resources for immunogenetic-focused research and operational bioinformatics as well as frequently used HLA tools.
3.2.6 HRSA Blood Cell Transplant Website
The HRSA Blood Cell Transplant website (bloodcell.transplant.hrsa.gov) provides information for the public, physicians, and other constituents. It incorporates transplant resources, donor information, and cord blood information as well as research, data, and outcomes. CIBMTR data and research findings are incorporated in numerous ways, including through CIBMTR-created reports:
Transplant Outcomes and Data
US Patient Survival Report Provides disease-specific post-HCT survival estimates by the length of time after transplant: 100 days, 1 year, and 3 years. Survival estimates are also available by patient age, patient gender, patient race, or cell source.
Transplant Data by US Center Report Displays the number of adult donor and cord blood transplants performed at a specific transplant center.
Transplant Data by Disease Report Displays the number of adult donor and cord blood transplants reported for a specific disease. Totals are also available by patient age, patient gender, patient race, cell source, and the year the transplant was performed.
Transplant Activity Report Displays the number of transplants performed at US transplant centers, including autologous as well as related and unrelated allogeneic. Numbers are also available by patient age, patient gender, patient race, cell source, disease, transplant center location by state, and year in which the transplant was performed.
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3.2.7 Other Applications and Data Exchange Standards
The CIBMTR has 7 methods for sharing data. The 4 hosted on the CIBMTR Portal site (DBtC, Center Volumes Portal, Patient One-Year Survival Calculator, and Qlikview) were described in Section 3.2.3. The other 3 methods for sharing data are:
AGNIS
AGNIS (A Growable Network Information System) allows participating centers to electronically collect and share data with the CIBMTR as well as others who link to AGNIS. Data are entered once and then distributed and synchronized among databases. In 2015, a total of 12,873 forms for 1,840 patients were submitted through AGNIS by 20 US transplant centers and by EBMT for 47 of their affiliated centers.
BRIDG
The BRIDG (Biomedical Research Integrated Domain Group) Model is an information model, representing a shared view of the concepts of basic, pre-clinical, clinical, and translational research. Common data elements for certain standard CIBMTR forms have been extracted and associated in the BRIDG model to one of three contexts: recipient, donor, or stem cell product. Future expansion of the BRIDG model will add HCT content as the basis for a physical database model, which will help remove barriers that transplant centers experience in electronic transfer of HCT data to the SCTOD. It will provide a foundation upon which centers and vendors can develop their own in-house data systems and, eventually, develop electronic medical record integration engines to submit data to the CIBMTR Research Database.
Disease Risk Index Assignment Tool
In March 2015, the CIBMTR launched a Disease Risk Index Assignment Tool developed by investigators at the Dana Farber Cancer Institute and validated in a large CIBMTR study. It is intended for use by clinical researchers. The tool was developed for the primary outcome of overall survival after HCT and, at present, only applies to adult patients with hematologic malignancies. It is NOT intended to give an accurate prognosis for individual patients. In 2015, 1,370 unique, non-CIBMTR visitors viewed the Disease Risk Index Assignment Tool 3,764 times.
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3.3 BMT TANDEM MEETINGSThe BMT Tandem Meetings are co-sponsored with the ASBMT and are held annually in February. They include 5 days of plenary sessions, concurrent scientific sessions, and other meetings. Reports on recent progress and updates in basic science, translational research, and clinical studies are targeted to worldwide physicians, scientists, and other health professionals with an interest in HCT.
2015 BMT Tandem Meetings
With 3,132 attendees from 47 countries, the 2015 BMT Tandem Meetings included 5 plenary sessions, 11 concurrent sessions, 108 oral abstracts, 2 poster sessions, 6 corporate-supported symposia, and 3 product theaters. Continuing Medical Education (CME) and Continuing Education credits were issued through MCW to physicians and allied health professionals. In addition to the extensive scientific agenda, many educational opportunities focused on young investigators and other allied health professionals.
Clinical Research Professionals / Data Management Conference With almost 200 attendees, this conference provided forms training, which increases the accuracy with which CIBMTR forms are completed.
BMT CTN Coordinators and Investigators Meetings With approximately 100 and 300 attendees, respectively, these meetings focused on treatment options; study management, such as promoting studies and reporting adverse events; processes, such as endpoint review; and specific clinical trials.
IT Forum With almost 100 attendees, this forum discussed the Research Data Life Cycle and various ways to share data, including from electronic medical records via BRIDG.
BMT Center Administrators Conference With approximately 165 attendees, this conference focused on many topics related to quality and outcomes, including benchmarking and key metrics, such as readmission rates, as well as Medicare coverage and workforce capacity.
BMT Pharmacists Conference With almost 200 attendees, this conference presented the latest research and best practices with a focus on specific diseases and preparative regimens.
Transplant Nursing Conference With almost 500 attendees, this conference presented the latest research as well as disease-specific information and communication techniques.
BMT Clinical Education Conference This conference is designed for Nurse Practitioners, Physician Assistants, Fellows, and Junior Faculty. With almost 200 attendees, the conference focused not only on the latest clinical research but also informed consent and mental health.
2016 BMT Tandem Meetings
The 2016 BMT Tandem Meetings will include 5 plenary sessions, 9 concurrent sessions, 133 oral abstracts, 2 poster sessions, 5 corporate-supported symposia, and 5 product theaters. They are expected to attract approximately 3,200 attendees.
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3.4 DATA MANAGEMENT TRAININGThe CIBMTR has developed comprehensive, secure and efficient applications to allow you to electronically submit data to the CIBMTR. Visit the CIBMTR Data Management Training and Reference webpage to access resources.
Center Reference Guide Learn about participation in CIBMTR research, center membership, access to FormsNet, data manager education, mentor program, forms submission process and many useful tips and links.
Manuals Find the answers to your data submission questions by accessing the Forms Instructions Manual, which includes general instructions and instructions for each form type.
FormsNet
Learn how to submit data to the CIBMTR via the FormsNet application, a secure clinical research management system, which is in compliance with SCTOD requirements.
Conference Materials Access meeting materials as well as audio and visual presentations on the form submission process presented at Clinical Research Professionals / Data Management Conferences.
AGNIS Learn how to retrieve and transmit form data, extracted directly from your own institution’s database, directly to the FormsNet application using AGNIS, a secure, standards-based system.
Legacy Data Review retired data manuals, forms, and other archived documents for reference purposes and to assist in making changes to legacy data.
Adverse Events Learn how to report adverse events and product issues through FormsNet.
Newsletters and eBlasts Read archived issues of the Data Matters Training Newsletter and eBlasts.
Tip Sheets Access sheets providing tips and instructions for various CIBMTR forms.
Online Training Review educational modules developed for new and seasoned data managers through the NMDP/Be The Match Learning Center.
Learning Center Courses
Data Back to Centers Overview
HLA SERIES
Introduction to HLA
HLA Reporting (Form 2005)
Basic Biology of HLA
Advanced Biology of HLA
ESSENTIAL FORM SERIES
Baseline Form 2000
CRID 2804
Form 2400 Pre-TED
Indication for CRID Assignment (Form 2814)
Infusion Data (Form 2006) Reporting Overview
Reporting Preparative Regimen on Pre-TED (Form 2400) and Baseline (Form 2000)
DISEASE SPECIFIC SERIES
Form 2016 / 2116 Plasma Cell Disorders
Form 2018 / 2118 Lymphoma
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4.0 HOW WE COLLECT AND MANAGE DATA
4.1 RESEARCH DATA LIFE CYCLEThe Research Data Life Cycle (Figure 4.1) describes the path of data from the point of capture to its ultimate use in analysis, reporting, and publication.
The process begins with data collection. Most centers enter data in FormsNet, a web application now in its third generation. Centers that have implemented local or third party systems can also capture and submit data electronically using AGNIS. An overriding goal of these applications is to ease the data capture burden on the centers.
Following collection, data undergo quality assessment and validation and are extracted 4 times per month and loaded into the CIBMTR Research Database.
Data Sharing completes the cycle, providing data for analysis that have been collected and curated to ensure research value. These data are extracted from the Research Database in monthly and quarterly retrievals to serve a range of research and stakeholder needs. The data retrievals provide the basis for research study data files, reports, and externally-requested datasets. TED-level data are also directly available to centers through use of the DBtC application (Section 3.2.3).
Figure 4.1. Research Data Life Cycle
Abbreviations: AGNIS = A Growable Network Information System, CRID = CIBMTR Recipient Identification Number, CRF = Comprehensive Report Form, FN = FormsNet, IDW = Integrated Data Warehouse, RDB = Research Database
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4.2 COLLECTING AND STORING DATA
4.2.1 FormsNet
More than 95% of data collected by the CIBMTR is submitted electronically via FormsNet, a comprehensive electronic data submission system containing >240 forms related to capturing HCT outcomes for donors and recipients. The application was updated in 2015 to provide key enhancements supporting operational efficiencies and enhanced data collection. For instance, a more flexible CIBMTR Recipient ID (CRID) Assignment Form establishes a patient ID independent of treatment, allowing the CIBMTR to expand data collection to patients who receive cellular therapy as well as non-HCT treatments. In addition, the CIBMTR released several forms to support data collection needs of the Radiation Injury Treatment Network (RITN), which will collect incident, medical history, and follow-up data on casualties exposed to or contaminated by radioactive material in the event of a national radiation event.
4.2.2 Research Database
The CIBMTR Research Database now contains information on >425,000 patients. Submission of outcomes data is mandatory for allogeneic HCTs in the US and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and about 80% of US autologous HCTs are reported.
4.3 ENSURING DATA QUALITY
4.3.1 Continuous Process Improvement
Robust data collection is critical to the success of the CIBMTR. The CPI program ensures timeliness and completeness of data forms submissions (Appendix H).
Recipient Forms
Transplant centers receive CPI reports 3 times per year (January, May, and September), listing the number of follow-up forms that were due in the previous trimester and the number and percentage of each submitted within the trimester. A form is not officially submitted until all errors are resolved and all applicable information is submitted and approved. To be compliant, centers must submit ≥90% of forms due for the trimester, for all unrelated donor transplants and for related donor and autologous transplants that have occurred since December 3, 2007.
Donor Forms
The Donor Data Management Team oversees submission of transplant donor forms from NMDP/Be The Match donor, collection, and apheresis centers. Donor CPI reports are generated 4 times per year (January, April, July, and October). To be compliant, centers must submit 100% of the forms required for that CPI period.
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4.3.2 Verification and Validation
FormsNet
When data are entered into FormsNet, a series of entry level validation checks takes place to ensure data consistency. This process flags certain errors at the time of entry and allows the CIBMTR to contact the center data manager so errors can be corrected immediately while source documents are readily available. If a data field does not pass the FormsNet validation checks, an error comment is generated, and the data manager is navigated to an error review page to review, resolve, or override the unresolved errors. Lastly, an error report is generated that lists any unresolved errors as well as errors that have been overridden.
Entry Level Validation Checks
Mandatory field validation
Range validation
Cross form consistency
Within form consistency
Core field validations
Research Database
Data extracted from FormsNet and loaded to the Research Database each month undergoes comprehensive validation and verification. These data are rigorously validated for consistency, completeness, and uniqueness using business rules implemented in custom logic for the categories provided below. Finally, the Data Quality Team reviews errors and works with transplant centers to correct data.
Extraction Validation Checks
Fields that cannot be null
Cross form consistency
Longitudinal consistency
Logical relationships
Rules across these categories were updated and tested as part of the recent Forms Revision. The CIBMTR continues to work toward reducing the number of data entry errors. Currently, the rate of form rejection due to inconsistently reported data is <2%, which is attributed to recent enhancements in transplant center education as well as to enhanced validations built in at the point of entry in FormsNet.
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4.3.3 On-Site Data Audit Program
On-going data audits are performed at all CIBMTR participating transplant centers. The audit compares data in source documents maintained at the transplant center with data contained in the CIBMTR Research Database. Clinical Research Associates perform the on-site transplant center audits, spending 3-4 days at each center reviewing original source documents. The overall audit process is displayed in Figure 4.2.
The Foundation for the Accreditation of Cellular Therapy (FACT) and the CIBMTR agreed in 2015 to consolidate transplant center data audits. Therefore, beginning in 2016, FACT auditors will use the results of the CIBMTR data audit and discontinue use of the current independent FACT data audit. Consolidating audits of transplant center data
collection and reporting eases the reporting and compliance burdens of transplant centers and demonstrates the value of the CIBMTR’s data collection and management to an international accrediting agency.
In 2015, 62 centers were scheduled for audit (48 domestic, 16 international). As of December 31, 57 centers were notified of their final audit results, including requested corrective action follow-up. Of the centers sent reports, 80% passed with ≤3% critical field errors. Of the 11 centers that did not pass the audit, 7 completed all required corrective action; the remaining 4 centers are in the process of completing requested corrective action.
Figure 4.2. Audit Process
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4.4 PROTECTING PATIENTS AND DATA
4.4.1 Human Subjects / HIPAA Compliance
The CIBMTR is committed to the ethical conduct of research. All Coordinating Center personnel maintain Collaborative IRB Training Initiative (CITI) certification. The NMDP/Be The Match IRB, which is fully accredited by the Association for the Accreditation of Human Research Protection Programs, reviews all human subject research conducted by the CIBMTR. The CIBMTR maintains compliance with the Health Insurance Portability and Accountability Act (HIPAA) Privacy Rule, as applicable. CIBMTR rules requiring the registration of all consecutive HCT recipients ensure the inclusion of women, minorities, and children, so the Research Database population includes women and minorities in the same proportion as found in the general HCT population. Children are included in most CIBMTR studies; their inclusion is dependent on the study focus.
4.4.2 Information Security and Data Privacy
The CIBMTR protects the data and information received from transplant centers and patients. The SCTOD contract requires specific protections through minimum security controls, policies, and standards. The CIBMTR’s data systems are maintained in accordance with the Federal Information Systems Management Act of 2002, with information security guidance provided by the National Institute of Standards and Technology. In accordance with National Institute of Standards and Technology Special Publication 800-18, and supervised by HRSA’s Office of Information Technology, the CIBMTR maintains a System Security Plan that outlines management, operational, and technical controls.
Since 2008, the CIBMTR holds an Authority to Operate from the Chief Information Officer of HRSA. The certification was renewed in June 2015, and security audits are performed annually, most recently in September 2015. The NMDP/Be The Match also holds an Authority to Operate from HRSA, ensuring similar standards of information security are applied to all CIBMTR and NMDP/Be The Match systems. These controls, maintained by the CIBMTR and NMDP/Be The Match, protect the data and information in these systems in ways beyond those required by HIPAA.
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5.0 WHAT WE WILL DO NEXT
In 2016, the CIBMTR will continue to conduct high quality research through a collaborative process.
Specific plans are listed in Figure 5.1 and Tables 5.2-5.4.
Figure 5.1. Plans for 2016
Details provided in Tables 5.2-5.4.
Enhance the quality and scope of CIBMTR data
Increase research
productivity and scientific
impact
Expand data and knowledge
sharing
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Table 5.2. Plans to Enhance Data
Enhance the quality and scope of CIBMTR data
Further develop a cellular therapy registry
Expand data collection to patients who receive non-HCT treatments
Continually review data collection forms for scientific content updates
Implement key data validations in FormsNet, close to the point of data capture
Implement consolidated transplant center data audits with FACT to enhance data monitoring while decreasing reporting and compliance burdens on transplant centers
Work toward an Integrated Data Warehouse to strengthen flexibility of data use and ease of data access
Provide onsite and online data management training support for transplant centers
Table 5.3. Plans to Expand Knowledge Sharing
Expand data and knowledge sharing
Implement two data sharing products using Qlikview software applications to enhance transplant centers’ self-service access to their data and information
• eDBtC will provide centers with access to a wide range of TED- and CRF-level data
• Performance data tool will provide centers with comparison data from the SCTOD annual center-specific analysis of posttransplant survival in US centers
Publish easy to read summaries of key HCT publications
Strengthen collaborations with international partners to facilitate data sharing
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Table 5.4. Plans to Increase Impact
Increase research productivity and scientific impact
Focus on studies with highest scientific impact and important clinical and policy implications; limit accepted studies to a number that can be completed in a timely manner
Support Working Committee Chairs to be active leaders in their committees
Regularly share metrics as a Working Committee management resource
Expand use of outcomes data to assist in the design, implementation, and long-term follow-up of clinical trials
Develop new statistical methodologies
Support Health Services Research initiatives
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2015 KEY ACCOMPLISHMENTS
CIBMTR by the Numbers
RESEARCH DATABASE ~400 participating centers >425,000 patients ~21,000 new patients annually
PUBLICATIONS >1,000 publications since inception
76 publications in 2015 38 from Working Committees 8 from BMT CTN 4 from Health Services Research
Program 9 from Bioinformatics Research
Program 5 from Statistical Methodology
Research Program 14 from Coordinating Center (2 cross-listed between programs)
PRESENTATIONS 65 presentations in 2015 (41 oral and
24 poster) 25 abstracts (17 oral and 8 poster)
presented at the 2015 ASH Annual Meeting
17 abstracts (13 oral and 4 poster) presented at the 2015 BMT Tandem Meetings
6 abstracts (4 oral and 2 poster) presented at the 2015 EBMT Annual Meeting
17 abstracts (7 oral and 10 poster) presented at other national and international conferences
Clinical Outcomes Research Program
WORKING COMMITTEES Administered 15 committees in which 2,300 worldwide researchers participate
Collaborated with the 54 global experts in the HCT field who voluntarily chair the committees
Conducted 176 ongoing studies
Reviewed 193 new study proposals for presentation at the 2016 BMT Tandem Meetings, >75% of which were submitted by new investigators
Presented 38 abstracts (26 oral and 12 poster) at national and international conferences
Supported approximately 400 scientific authors at 200 institutions worldwide to publish research findings
Published 38 manuscripts in peer-reviewed journals
CELLULAR THERAPIES AND NON-TRANSPLANT THERAPY
Initiated a Cellular Therapies Task Force charged with determining the type and process for cellular therapies data collection
Hosted a Cellular Therapies Forum to develop recommendations for a cellular therapy registry
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Immunobiology Research Program
Collected samples from 175 centers (128 transplant centers, 32 donor centers, and 15 cord blood banks)
Curated 12,656 samples in the Research Repository (137,464 overall)
Completed high resolution HLA and presence / absence KIR typing on 226 related and 2,772 unrelated HCT donor / cord and recipient pairs
Distributed 8,770 samples to investigators for various studies
Presented 5 abstracts (2 oral and 3 poster) at national and international conferences
Published 6 manuscripts in peer-reviewed journals
RESEARCH REPOSITORY Added 4,392 unrelated recipient samples (57,018 overall)
Added 1,264 related recipient samples (5,377 overall)
Added 4,819 adult unrelated donor samples (59,375 overall)
Added 1,258 related donor samples (5,128 overall)
Added 923 unrelated cord blood samples (10,566 overall)
Clinical Trials Support Program
BMT CTN Opened 3 new trials to accrual, bringing the total number of launched trials to 37
Accrued >1,100 patients to trials, increasing the total number of accrued patients to >8,500
Managed 12 open protocols with overall accrual for open studies at about 125% of projections
Presented 11 abstracts (8 oral and 3 poster) at national and international conferences
Published 8 manuscripts in peer-reviewed journals
RCI BMT Opened 3 new trials to accrual, bringing the total number of launched trials to 15
Accrued just under 3,000 patients, bringing the total number of accrued patients to approximately 28,000
Presented 4 oral abstracts at national and international conferences
Survey Research Group Supported 8 active studies, and participated in the development of one upcoming study
CIBMTR 2015 Annual Report 2015 KEY ACCOMPLISHMENTS
Page | 51
Health Services Research Program
Established the HCT Health Economics Interest Group, which held its first meeting at the 2015 BMT Tandem Meetings
Opened a multi-center randomized controlled study to evaluate the effectiveness of an individualized survivorship care plan template; 17 centers will participate in this study
Conducted a national survey of community hematologists / oncologists on referral practices, perceptions of HCT, and education preferences to inform an educational intervention on timing of community-based referral for HCT
Completed two studies: Identifying HCT patient housing and caregiver challenges and potential interventions and The financial impact of allogeneic HCT on patient and family: a pilot study
Conducted >30 evaluations for the HRSA Office of Patient Advocacy / Single Point of Contact contract and NMDP/Be The Match programs
Provided technical plain language writing consultation; developed 3 easy-to-read consent forms and 6 patient information sheets for BMT CTN protocols
Presented 3 oral abstracts at a national conference
Published 4 manuscripts in peer-reviewed journals
Bioinformatics Research Program
Developed guidelines for reporting HLA and KIR genotyping via Next Generation Sequencing
Investigated the role of genetic ancestry in transplantation
Developed methods for HLA association studies
Investigated HLA data from other countries
Presented 11 abstracts (5 oral and 6 poster) at national and international conferences
Published 9 manuscripts in peer-reviewed journals
Statistical Methodology Research Program
Developed new statistical models
Ensured the statistical integrity of CIBMTR scientific activities
Contributed to results in articles on HCT-related statistical issues for clinical audiences
Supported Working Committee study investigators in developing scientific study protocols using CIBMTR data
Published 4 peer-reviewed manuscripts and 1 editorial
CIBMTR 2015 Annual Report 2015 KEY ACCOMPLISHMENTS
Page | 52
SCTOD
Published annual Center-Specific Survival Report, Transplant Center Volumes Data for 2009-2013, and a Transplant Activity Report for transplants performed 2009-2013
Began implementing recommended changes to tools to enhance transplant centers’ quality improvement efforts in response to recommendations from the 2014 Center Outcomes Forum
Continued analysis of a quality of life project, which is unique in that the CIBMTR collected data directly from patients after HCT
In conjunction with Be The Match’s Patient and Health Professional Services, published 12 lay summaries of CIBMTR publications
CIBMTR 2015 Annual Report APPENDIX A: TRANSPLANT CENTERS
Page | 53
APPENDIX A: TRANSPLANT CENTERS
Figure A.1. Location of Transplant Centers that Submit Data to the CIBMTR
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
Page | 54
APPENDIX A1: US CENTERS
The following table lists the US-based transplant centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF.
Participating Center City State MUD RELATED AUTO
University of Alabama Birmingham Birmingham AL CRF CRF CRF
Providence Hospital HPC Transplant Center Mobile AL N/A N/A CRF
University of Arkansas for Medical Sciences Little Rock AR CRF TED N/A
Banner MD Anderson Cancer Center Gilbert AZ CRF CRF CRF
Banner Blood and Marrow Transplant Program Phoenix AZ CRF CRF CRF
Mayo Clinic Arizona and Phoenix Children's Hospital Phoenix AZ CRF CRF CRF
Cancer Transplant Institute at Virginia G. Piper Cancer Center Scottsdale AZ CRF CRF CRF
University Medical Center - Tucson Tucson AZ CRF TED TED
City of Hope National Medical Center Duarte CA CRF TED TED
Scripps Blood & Marrow Transplant Program La Jolla CA CRF CRF CRF
University of California, San Diego Medical Center La Jolla CA CRF CRF CRF
Loma Linda University Cancer Center Loma Linda CA CRF CRF CRF
Cedars Sinai Medical Center Los Angeles CA CRF TED N/A
Children's Hospital of Los Angeles Los Angeles CA CRF TED TED
UCLA Center for Health Sciences Los Angeles CA CRF CRF TED
USC BMT Program Los Angeles CA CRF CRF CRF
Children's Hospital of Oakland Oakland CA CRF CRF CRF
Children's Hospital of Orange County Orange CA CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
St Joseph’s Hospital Irvine Orange CA N/A N/A CRF
University of California Irvine Medical Center Orange CA CRF CRF CRF
Lucile Packard Children’s Hospital Palo Alto CA CRF CRF CRF
Sutter Cancer Center Sacramento CA TED TED TED
University of California - Davis Cancer Center Sacramento CA CRF TED TED
Rady Children's Hospital San Diego San Diego CA CRF CRF CRF
University of California - San Francisco - Adults San Francisco CA CRF CRF CRF
University of California - San Francisco - Pediatrics San Francisco CA CRF CRF CRF
Stanford Health Care Stanford CA CRF CRF TED
Stanford University Medical Center Stanford CA CRF CRF TED
The Children's Hospital of Denver Aurora CO CRF CRF CRF
University of Colorado Hospital Aurora CO CRF TED TED
Colorado Blood Cancer Institute Denver CO CRF TED TED
University of Colorado Medical School Denver CO N/A N/A N/A
University of Connecticut Farmington CT N/A N/A N/A
Yale New Haven Hospital New Haven CT CRF CRF CRF
Children's National Medical Center Washington DC CRF TED TED
Medstar Georgetown University Hospital Washington DC CRF N/A CRF
Christiana Care Newark DE CRF CRF CRF
Alfred I. duPont Hospital for Children Wilmington DE CRF CRF CRF
Shands HealthCare & University of Florida Gainesville FL CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
BMT Program of Mayo Clinic / Nemours and Wolfson Children's Hospital Jacksonville FL CRF CRF CRF
Mayo Clinic Florida - Jacksonville Jacksonville FL CRF CRF CRF
Miami Children's Hospital Miami FL CRF TED TED
University of Miami - Adults Miami FL CRF CRF CRF
University of Miami / Jackson Memorial Hospital Miami FL CRF TED TED
Blood & Marrow Transplant Center, Florida Hospital Medical Group Orlando FL CRF CRF CRF
Memorial Cancer Institute Pembroke Pines FL TED TED TED
All Children's Hospital St. Petersburg FL CRF CRF CRF
H Lee Moffitt Cancer Center Tampa FL CRF TED TED
Children's Healthcare of Atlanta at Egleston Atlanta GA CRF CRF CRF
Emory University Atlanta GA CRF CRF CRF
The Blood and Marrow Transplant Program at Northside Hospital Atlanta GA CRF CRF CRF
Georgia Regents University Cancer Center Augusta GA CRF CRF CRF
Hawaii Medical Center Honolulu HI N/A N/A N/A
Kapi'olani Medical Center for Women and Children Honolulu HI CRF CRF CRF
University of Iowa Hospital & Clinics Iowa City IA CRF CRF CRF
St. Luke's Mountain States Tumor Institute Boise ID N/A N/A CRF
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago IL CRF CRF CRF
Northwestern Memorial Hospital Chicago IL CRF CRF TED
Northwestern Memorial Hospital - Department of Immunotherapy Chicago IL CRF TED N/A
The Coleman Foundation Blood and Marrow Transplant Center, Rush University Chicago IL CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
University of Chicago Medical Center Chicago IL CRF CRF CRF
University of Illinois at Chicago Medical Center Chicago IL CRF CRF CRF
Loyola University Medical Center Maywood IL CRF CRF CRF
Advocate Lutheran General Hospital Park Ridge IL N/A TED TED
Cancer Treatment Centers of America - Midwest Zion IL CRF CRF CRF
Indiana Blood & Marrow Transplantation Beech Grove IN CRF CRF CRF
Indiana University Hospital / Riley Hospital for Children Indianapolis IN CRF CRF TED
St Vincent Hospital Indianapolis Indianapolis IN N/A CRF CRF
University of Kansas Kansas City KS CRF CRF CRF
Via Christi Hospitals Wichita Wichita KS N/A CRF CRF
University of Kentucky Medical Center Lexington KY CRF TED TED
University of Louisville Hospital / James Brown Cancer Center Louisville KY CRF TED TED
Louisiana State University Children's Hospital New Orleans LA CRF CRF CRF
Ochsner Medical Center New Orleans LA N/A CRF TED
Tulane University Medical Center New Orleans LA CRF CRF CRF
Louisiana State University Health Sciences Center - Shreveport Shreveport LA CRF CRF CRF
Beth Israel Deaconess Medical Center Boston MA CRF TED TED
Boston Medical Center Boston MA N/A N/A TED
Dana Farber Cancer Institute at Brigham and Women's Hospital - Adults Boston MA CRF CRF TED
Dana Farber Cancer Institute at Brigham and Women's Hospital - Pediatrics Boston MA CRF TED TED
Massachusetts General Hospital Boston MA CRF TED TED
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
Tufts New England Medical Center Boston MA CRF TED TED
Lahey Clinic Medical Center Burlington MA N/A N/A TED
UMass Memorial Medical Center Worcester MA CRF CRF CRF
Johns Hopkins Oncology Center Baltimore MD TED TED TED
University of Maryland School of Medicine Baltimore MD CRF CRF CRF
National Heart Lung & Blood Institute Bethesda MD N/A TED N/A
National Institutes of Allergy & Infectious Disease Bethesda MD N/A TED N/A
National Institutes of Health Bethesda MD N/A TED N/A
NIH - NCI Experimental Transplantation and Immunology Branch (Related Donor Program)
Bethesda MD N/A CRF CRF
NIH - NCI Matched Unrelated Donor Program Bethesda MD CRF N/A N/A
The University of Michigan Ann Arbor MI CRF TED TED
Children's Hospital of Michigan Detroit MI CRF CRF CRF
Henry Ford Hospital Bone Marrow Transplant Program Detroit MI CRF CRF CRF
Karmanos Cancer Institute Detroit MI CRF CRF CRF
Helen DeVos Children's Hospital Grand Rapids MI CRF CRF CRF
Spectrum Health Grand Rapids MI CRF CRF CRF
Abbott Northwest Hospital Minneapolis MN N/A N/A TED
Masonic Cancer Center University of Minnesota Minneapolis MN CRF CRF CRF
Mayo Clinic Rochester Rochester MN CRF CRF CRF
Saint Luke’s Blood & Marrow Transplant Program Kansas City MO CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
The Children's Mercy Hospitals and Clinics Kansas City MO CRF CRF CRF
Cardinal Glennon Children's Hospital St. Louis MO CRF CRF CRF
SSM Health Saint Louis University Hospital St. Louis MO CRF TED TED
Washington University School of Medicine St. Louis MO CRF CRF TED
Washington University / St Louis Children's Hospital St. Louis MO CRF CRF CRF
University of Mississippi Medical Center - Jackson Jackson MS CRF TED TED
Billings Clinic Cancer Center Billings MT N/A N/A TED
University of North Carolina Hospitals - Chapel Hill Chapel Hill NC CRF CRF CRF
BMT Program at Levine Children's Hospital / Carolinas Medical Center Charlotte NC CRF CRF CRF
Levine Cancer Institute Charlotte NC N/A CRF CRF
Duke University - Adults Durham NC CRF TED TED
Duke University Medical Center, Pediatric BMT Durham NC CRF CRF CRF
Duke University, Immunology / BMT, Pediatrics Durham NC N/A TED N/A
Novant Health Oncology Specialists Winston-Salem NC N/A N/A N/A
Wake Forest Baptist Health Winston-Salem NC CRF TED TED
CHI Health Bergan Mercy Omaha NE N/A N/A TED
CHI Health Immanuel Omaha NE N/A N/A TED
Nebraska Methodist Hospital Omaha NE N/A N/A TED
University of Nebraska Medical Center Omaha NE CRF CRF CRF
Dartmouth-Hitchcock Medical Center Lebanon NH CRF CRF CRF
Hackensack University Medical Center Hackensack NJ CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
Cancer Institute of New Jersey New Brunswick NJ CRF CRF CRF
University of New Mexico - Pediatrics Albuquerque NM N/A N/A CRF
Childrens Hospital at Montefiore Bronx NY CRF TED TED
Montefiore Medical Center Bronx NY CRF CRF TED
Roswell Park Cancer Institute Buffalo NY CRF CRF CRF
Westchester Medical Center Hawthorne NY CRF CRF CRF
North Shore University Hospital Manhasset NY CRF TED TED
Cohen Children's Medical Center of New York
New Hyde Park NY CRF CRF CRF
Memorial Sloan Kettering Cancer Center - Adults New York NY CRF TED TED
Memorial Sloan Kettering Cancer Center - Pediatrics New York NY CRF TED TED
Morgan Stanley Children's Hospital of New York New York NY CRF CRF CRF
Mount Sinai Medical Center - New York New York NY CRF CRF TED
New York Presbyterian Hospital New York NY CRF CRF CRF
New York Presbyterian Hospital / Columbia University Medical Center New York NY CRF CRF CRF
New York University Medical Center New York NY TED TED TED
St Vincent's Hospital Manhattan New York NY N/A TED TED
University of Rochester Medical Center Rochester NY CRF CRF CRF
Stony Brook University Medical Center Stony Brook NY TED TED CRF
State University of NY Upstate Medical University Syracuse NY N/A CRF CRF
Akron Children's Hospital Akron OH CRF CRF CRF
Cincinnati Children's Hospital Cincinnati OH CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
Jewish Hospital Blood and Marrow Transplant Center Cincinnati OH CRF CRF CRF
University of Cincinnati Medical Center Cincinnati OH CRF CRF CRF
Cleveland Clinic Cleveland OH CRF CRF CRF
Seidman Cancer Center - University Hospitals Case Medical Center Cleveland OH CRF CRF CRF
Nationwide Children's Hospital Columbus OH CRF CRF CRF
The Ohio State University Medical Center Columbus OH CRF CRF CRF
Miami Valley Hospital BMT Program Dayton OH N/A N/A CRF
Oklahoma University Medical Center Oklahoma City OK CRF CRF CRF
Cancer Treatment Centers of America Tulsa OK CRF CRF CRF
Saint Francis Hospital - Oklahoma Tulsa OK N/A TED TED
Legacy Good Samaritan Hospital and Medical Center Portland OR N/A N/A CRF
Oregon Health and Science University Portland OR CRF CRF CRF
Pediatric BMT Program, Doernbecher Children's Hospital (OHSU) Portland OR CRF CRF CRF
Providence Portland Medical Center Portland OR N/A N/A CRF
Geisinger Medical Center Danville PA CRF TED TED
Penn State Hershey Medical Center Hershey PA CRF CRF CRF
Abramson Cancer Center University - Pennsylvania Medical Center Philadelphia PA CRF CRF CRF
Eastern Regional Medical Center Philadelphia PA N/A CRF CRF
Hahnemann University Hospitals Philadelphia PA CRF TED TED
Philadelphia Children's Hospital Philadelphia PA TED TED N/A
St Christopher's Hospital for Children Philadelphia PA TED TED TED
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
Temple Bone Marrow Transplant Program Philadelphia PA CRF TED TED
Thomas Jefferson University Philadelphia PA TED TED TED
Children's Hospital of Pittsburgh of UPMC Pittsburgh PA CRF CRF CRF
University of Pittsburgh Medical Center Pittsburgh PA CRF CRF CRF
West Penn Hospital Pittsburgh PA CRF CRF CRF
Roger Williams Medical Center Providence RI CRF TED TED
Charleston Hematology Oncology Charleston SC N/A CRF CRF
Medical University of South Carolina Charleston SC CRF TED TED
Richland Memorial Hospital Columbia SC N/A N/A N/A
Cancer Centers of the Carolinas Greenville SC CRF CRF CRF
Saint Francis Hospital - Greenville Greenville SC N/A CRF CRF
Avera Mckennan Transplant Institute Sioux Falls SD CRF CRF CRF
Thompson Cancer Survival Center Knoxville TN N/A N/A TED
Baptist Blood and Marrow Transplant Memphis TN CRF CRF CRF
Baptist Centers for Cancer Care Memphis TN TED TED TED
St Jude Children's Research Hospital Memphis TN CRF TED N/A
The West Clinic / Methodist Healthcare Blood and Marrow Transplant Center Memphis TN N/A N/A TED
University of Tennessee Memphis TN CRF CRF CRF
Sarah Cannon BMT Center at Centennial Medical Center Nashville TN CRF TED TED
Vanderbilt University Nashville TN CRF TED TED
Vanderbilt University Veterans Center Nashville TN TED TED N/A
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
Texas Oncology Amarillo TX CRF TED TED
Southwest Regional Cancer Center Austin TX N/A N/A N/A
Baylor University Medical Center Dallas TX CRF CRF CRF
Children's Medical Center - Dallas Dallas TX CRF CRF CRF
Medical City Dallas Hospital Dallas TX CRF CRF CRF
UT Southwestern Medical Center - BMT Program Dallas TX CRF CRF CRF
Cook Children's Medical Center Fort Worth TX CRF CRF CRF
Baylor College of Medicine Houston TX CRF CRF CRF
MD Anderson Cancer Center Houston TX CRF CRF N/A
Wilford Hall Medical Center Lackland AFB TX TED TED TED
Covenant Health System Hematopoietic Transplant Program Lubbock TX N/A TED TED
Texas Tech University Medical Center Lubbock TX CRF CRF CRF
South Texas Veterans Health Care System San Antonio TX N/A CRF CRF
Texas Transplant Institute San Antonio TX CRF CRF CRF
University of Texas Health Science Center San Antonio TX N/A CRF CRF
Scott and White Memorial Hospital Temple TX N/A N/A CRF
Latter Day Saints Hospital Salt Lake City UT CRF CRF CRF
University of Utah Medical Center Salt Lake City UT CRF CRF CRF
Utah Blood and Marrow Transplant Program - Adults Salt Lake City UT CRF CRF CRF
Utah Blood and Marrow Transplant Program - Pediatrics Salt Lake City UT CRF CRF CRF
University of Virginia Health System Charlottesville VA CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS
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Participating Center City State MUD RELATED AUTO
Fairfax - Northern Virginia Hospital Fairfax VA CRF CRF CRF
Virginia Oncology Associates Norfolk VA N/A N/A TED
Virginia Commonwealth University Massey Cancer Center BMT Program Richmond VA CRF CRF CRF
University of Vermont Cancer Center Burlington VT N/A N/A TED
Fred Hutchinson Cancer Center Seattle WA CRF CRF CRF
VA Puget Sound Healthcare System Seattle WA TED TED N/A
University of Wisconsin Hospital and Clinics Madison WI CRF CRF CRF
Marshfield Clinic Marshfield WI N/A N/A CRF
Aurora St Luke's Medical Center Milwaukee WI N/A N/A CRF
Children's Hospital of Wisconsin Milwaukee WI CRF CRF CRF
Froedtert & Medical College of Wisconsin Milwaukee WI CRF CRF TED
Osborn Hematopoietic Malignancy & Transplantation Program Morgantown WV CRF CRF CRF
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Page | 65
APPENDIX A2: INTERNATIONAL CENTERS
The following table lists the international transplant centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF.
Participating Center City Country MUD RELATED AUTO
Fundaleu-Angelica Ocampo Buenos Aires Argentina CRF CRF CRF
Hospital Universitario Austral Buenos Aires Argentina CRF CRF CRF
Hospital Privado de Cordoba Cordoba Argentina CRF CRF CRF
Royal Adelaide Hospital / SA Pathology Adelaide Australia CRF CRF N/A
Royal Prince Alfred Hospital Camperdown Australia CRF CRF TED
St Vincent's Hospital Darlinghurst Australia TED TED N/A
Royal Brisbane & Women's Hospital Herston Australia CRF CRF CRF
Alfred Hospital Melbourne Australia TED TED TED
Royal Children's Hospital Parkville Australia CRF CRF N/A
Fiona Stanley Hospital Perth Australia CRF CRF N/A
Princess Margaret Hospital for Children Perth Australia CRF CRF TED
Royal Perth Hospital Perth Australia CRF CRF N/A
Sydney Children's Hospital Randwick Australia CRF CRF N/A
Lady Cilento Children’s Hospital South Brisbane Australia N/A CRF CRF
Royal Melbourne Hospital Victoria Australia CRF CRF N/A
Calvary Mater Newcastle Hospital Waratah Australia N/A N/A CRF
Children's Hospital at Westmead Westmead Australia CRF CRF TED
Westmead Hospital Westmead Australia CRF CRF N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
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Participating Center City Country MUD RELATED AUTO
St Anna Children's Hospital Vienna Austria TED TED N/A
Children's University Hospital Bruxelles Belgium TED TED TED
University Hospital Antwerp Edegem Belgium TED TED N/A
University Hospital Gasthuisberg Leuven Belgium TED TED N/A
University Estadual de Campinas Campinas Brazil CRF CRF CRF
Hospital de Clinicas Curitiba Curitiba Brazil CRF CRF CRF
Hospital Amaral Carvalho Jau Brazil CRF CRF CRF
Hospital de Porto Alegre Porto Alegre Brazil TED TED TED
Instituto Nacional de Cancer Rio de Janeiro Brazil CRF CRF CRF
Albert Einstein Hospital Sao Paolo Brazil TED TED TED
Hospital Sirio Libanes Sao Paolo Brazil CRF CRF CRF
Instituto de Oncologia Pediatrica Sao Paolo Brazil TED TED TED
Alberta Children's Hospital Calgary Canada CRF CRF CRF
Tom Baker Cancer Centre Calgary Canada TED TED TED
Queen Elizabeth II Health Sciences Center Halifax Canada TED TED TED
Hamilton Health Sciences Hamilton Canada CRF CRF CRF
Kingston General Hospital Kingston Canada N/A N/A TED
Centre Hospitalier Montreal Canada CRF CRF CRF
Maisonneuve - Rosemont Hospital Montreal Canada TED TED N/A
McGill University Health Centre - Royal Victoria Hospital Montreal Canada TED TED N/A
Montreal Children's Hospital Montreal Canada TED TED TED
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
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Participating Center City Country MUD RELATED AUTO
Hotel-Dieu de Quebec Quebec Canada TED TED TED
CHA-Enfant-Jesus Hospital Quebec City Canada TED TED TED
Saint John Regional Hospital Saint John Canada N/A N/A CRF
St John's Health Sciences Center St. John's Canada N/A N/A TED
Health Sciences North Sudbury Canada N/A N/A TED
Princess Margaret Hospital (alloHCT) Toronto Canada CRF CRF N/A
Princess Margaret Hospital (autoHCT) Toronto Canada N/A N/A CRF
British Columbia Children's Hospital Vancouver Canada CRF CRF CRF
Vancouver General Hospital Vancouver Canada N/A N/A N/A
CancerCare Manitoba / University of Manitoba Winnipeg Canada CRF CRF CRF
Instituto de Trasplante de Medula Osea de la Costa Caribe Barranquilla Colombia TED TED TED
Clinica de Marly Bogota Colombia CRF CRF CRF
Charles University Hospital - Pilsen Pilsen Czech Republic CRF CRF CRF
Institute of Hem-Blood Transfusion Praha Czech Republic TED TED N/A
University Hospital, Rigshospitalet Copenhagen Denmark CRF CRF N/A
Children's Cancer Hospital - Egypt Cairo Egypt N/A TED TED
NCI Cairo University Cairo Egypt TED TED N/A
Helsinki University Central Hospital Helsinki Finland CRF CRF N/A
Turku University Turku Finland TED TED N/A
Centre Hospitalier Regional University D'Angers Angers France TED TED N/A
Hopital Jean Minjoz Besançon France CRF CRF N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
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Participating Center City Country MUD RELATED AUTO
Hopital Claude Huriez, Lille Lille France MED-A MED-A N/A
Hospital Edouard Herriot Lyon France MED-A MED-A N/A
Institute Paoli Calmettes Marseille France CRF CRF N/A
Hospital Saint Louis Paris France CRF CRF N/A
Hospital Jean Bernard Poitiers France CRF CRF N/A
University Hospital of Essen Essen Germany CRF CRF CRF
University Children’s Hospital Frankfurt Frankfurt Germany CRF CRF CRF
Albert-Ludwig University - Freiburg Freiburg Germany CRF CRF N/A
UKE Hamburg, Klinik und Poliklinik für Stammzelltransplantation Hamburg Germany CRF CRF N/A
University of Heidelberg Heidelberg Germany CRF CRF N/A
Christian Albrechts University Kiel Germany CRF CRF CRF
University Leipzig, BMT Center Leipzig Germany CRF N/A N/A
University Hospital Mainz Mainz Germany TED TED TED
University of Munich Munich Germany CRF CRF N/A
Klinikum der Universitӓt Regensburg Regensburg Germany MED-A MED-A N/A
Universitӓts Klinikum Tubingen Tübingen Germany CRF TED N/A
Universitӓts Kinderklinik Tubingen Tübingen Germany CRF TED N/A
Universitӓt Ulm - Adults Ulm Germany CRF CRF TED
Deutsche Klinik für Diagnostik - Wiesbaden Wiesbaden Germany CRF CRF N/A
University Hospital of Patras, Patras University Medical Center Rio Patras Greece TED TED TED
Chinese University of Hong Kong Shatin Hong Kong CRF CRF N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
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Participating Center City Country MUD RELATED AUTO
Gujrat Cancer & Research Institute Ahmedabad India CRF CRF CRF
Sir Ganga Ram Hospital Delhi India CRF CRF CRF
Apollo Hospital International Ltd Gandhinagar India N/A TED TED
Fortis Memorial Research Institute Gurgaon India N/A CRF CRF
Bone Marrow Transplant Unit, Christian Medical College, Ludhiana Ludhiana India TED TED TED
Rajiv Gandhi Cancer Institute and Research Centre New Delhi India CRF CRF CRF
Jawaharlal Institute of Postgraduate Medical Education and Research Puducherry India N/A TED TED
Sahyadri Speciality Hospital Pune India CRF CRF CRF
Christian Medical College Hospital Vellore India CRF CRF N/A
Rambam Medical Center Haifa Israel CRF CRF CRF
Haddasah University Hospital Jerusalem Israel CRF CRF N/A
Davidoff Cancer Center, Rabin Medical Center, Beilinson Hospital Petah Tikva Israel CRF CRF CRF
Tel-Aviv Sourasky Medical Center Tel-Aviv Israel CRF CRF CRF
Chaim Sheba Medical Center - Pediatrics
Tel-Hashomer Israel CRF CRF CRF
Sheba Medical Center Tel-Hashomer Israel CRF CRF N/A
Instituto di Ematologia e Oncologia Medica Seragnoli Bologna Italy MED-A MED-A N/A
University Bologna - Pediatrics Bologna Italy TED TED TED
St Eugenio Hospital Rome Italy CRF CRF N/A
Ospedale Molinette Torino Italy MED-A MED-A N/A
University Torino Torino Italy TED TED N/A
Osaka City University Osaka Japan CRF CRF N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
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Participating Center City Country MUD RELATED AUTO
Hospital Universitario Monterrey Mexico CRF CRF CRF
Centro de Hematologia y Medicina Interna Clinica RUIZ de Puebla Puebla Mexico TED TED TED
Academic Medical Center Amsterdam Netherlands CRF TED TED
VU Medical Center - Amsterdam Amsterdam Netherlands CRF TED TED
University Medical Center Groningen Groningen Netherlands CRF N/A N/A
Leiden University Medical Center Leiden Netherlands CRF TED N/A
Academic Hospital Maastricht Maastricht Netherlands CRF TED N/A
University Hospital of Nijmegen Nijmegen Netherlands CRF TED N/A
Erasmus MC - Daniel den Hoed Cancer Center Rotterdam Netherlands CRF TED N/A
University Hospital Utrecht Utrecht Netherlands CRF N/A N/A
University Medical Center Utrecht - Pediatrics Utrecht Netherlands CRF N/A N/A
Auckland City Hospital Auckland New Zealand CRF CRF N/A
Starship Children's Hospital Auckland New Zealand CRF CRF N/A
Christchurch Hospital Christchurch New Zealand CRF CRF TED
Wellington Blood and Cancer Centre Wellington New Zealand CRF CRF N/A
Rikshospitalet - The National Hospital Oslo Norway CRF N/A N/A
Hill Park Hospital - National Institute of Blood Diseases and Bone Marrow Transplantation
Karachi Pakistan CRF CRF CRF
Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan N/A CRF CRF
Instituto Nacional Salud Del Niño San Borja San Borja Peru N/A TED N/A
Silesian Medical Academy Katowice Poland CRF CRF N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
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Participating Center City Country MUD RELATED AUTO
Poznan University of Medical Sciences Poznan Poland MED-A MED-A N/A
Lower-Silesian Center for Cellular Transplantation and National Bone Marrow Donor Registry
Wroclaw Poland CRF CRF N/A
Institute Portugues de Oncologia - Lisbon Lisbon Portugal CRF CRF N/A
Institute Portugues de Oncologia - Porto Porto Portugal N/A N/A N/A
St Petersburg State Medical University St. Petersburg
Russian Federation TED TED TED
King Faisal Specialist Hospital - Pediatrics Riyadh Saudi Arabia CRF CRF N/A
King Faisal Specialist Hospital & Research Center - Adults Riyadh Saudi Arabia CRF CRF N/A
National University Health System - Adults Singapore Singapore CRF CRF CRF
National University Health System - Pediatrics Singapore Singapore CRF CRF CRF
Parkway Cancer Centre Singapore Singapore CRF CRF CRF
Singapore General Hospital Singapore Singapore CRF CRF N/A
Slovak Medical University Bratislava Slovak Republic TED TED N/A
Constantiaberg Medi-Clinic Cape Town South Africa CRF CRF CRF
University of Witwatersrand Parktown South Africa CRF CRF CRF
Asan Medical Center Seoul South Korea CRF CRF N/A
Samsung Medical Center Seoul South Korea CRF CRF CRF
Seoul St. Mary's Hospital Catholic BMT Center Seoul South Korea CRF CRF CRF
University Barcelona Barcelona Spain TED TED N/A
Gregorio Maranon University General Hospital Madrid Spain CRF CRF CRF
Hospital Puerta Hierro Madrid Spain MED-A MED-A N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Page | 72
Participating Center City Country MUD RELATED AUTO
Hospital Universitario Marques de Valdecilla Santander Spain TED TED TED
Hospital Universitario La Fe Valencia Spain CRF CRF N/A
Sahlgrenska University Hospital Gothenborg Sweden CRF CRF N/A
University Hospital of Lund Lund Sweden MED-A MED-A N/A
Karolinska University Hospital, Centre for Allogeneic Stem Cell Transplantation
Stockholm Sweden CRF CRF N/A
University Hospital - Uppsala Uppsala Sweden CRF CRF N/A
Basel Kantonsspital Basel Switzerland CRF CRF N/A
University Hospital-Zurich Zurich Switzerland MED-A MED-A N/A
Chang Gung Children's Hospital Taoyuan Taiwan CRF CRF CRF
King Chulalongkorn Memorial Hospital Bangkok Thailand TED TED TED
Ankara University Faculty of Medicine Ankara Turkey TED TED N/A
Gulhane Military Medical Academy Ankara Turkey CRF CRF CRF
Medical Park Hospital - Antalya Istanbul Turkey CRF CRF CRF
Birmingham Children's Hospital Birmingham United Kingdom TED TED N/A
Queen Elizabeth Hospital - Birmingham Birmingham United
Kingdom CRF CRF N/A
Bristol Children's Hospital Bristol United Kingdom CRF CRF N/A
Addenbrooke's NHS Trust Cambridge United Kingdom CRF CRF N/A
Beatson West of Scotland Cancer Centre Glasgow United
Kingdom MED-A MED-A N/A
Royal Hospital for Sick Children Glasgow United Kingdom CRF CRF N/A
Imperial College / Hammersmith Hospital London United
Kingdom CRF CRF N/A
CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Page | 73
Participating Center City Country MUD RELATED AUTO
Imperial College - St Mary's Hospital London United Kingdom CRF CRF N/A
The Royal Free Hampstead NHS Trust London United Kingdom MED-A MED-A N/A
Royal Marsden Hospital Sutton United Kingdom N/A N/A N/A
British Hospital Montevideo Uruguay CRF CRF CRF
Centro de Trasplante del Servicio Medico Integral (SMI) Montevideo Uruguay CRF CRF CRF
Hospital Maciel Montevideo Uruguay CRF CRF CRF
Ciudad Hospitalaraia Dr Enrique Tejera Valencia Venezuela TED TED TED
APPENDIX B: COORDINATING CENTER CIBMTR 2015 Annual Report ORGANIZATIONAL STRUCTURE AND PERSONNEL
Page | 74
APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP
The CIBMTR Coordinating Center resides on two campuses: one is located at MCW in Milwaukee, WI, and the other is located at NMDP/Be The Match in Minneapolis, MN. The Coordinating Center provides administrative, statistical, data management, clinical trials, IT, and personnel support for CIBMTR activities, and it benefits from a unique, collegial partnership with the Division of Biostatistics of MCW.
CIBMTR Milwaukee has approximately 70 employees and is an academic division of the MCW Department of Medicine. The Milwaukee office receives administrative support from the MCW departments of Grants and Contracts, Development, Office of Technology Development, Public Affairs, Human Resources and the Department of Medicine Administration. CIBMTR Minneapolis has approximately 120 employees, and several NMDP/Be The Match departments provide support for CIBMTR activities, including Finance, Contracts, and Marketing & Communications.
CIBMTR 2015 Annual Report APPENDIX B: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS
Page | 75
APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS
Chief Scientific DirectorM Horowitz, MD, MS
AdministratorCIBMTR MilwaukeeP Steinert, PhD, MBA
Medical Faculty
Senior Research Advisor
D Weisdorf, MD1
Senior Scientific Director
L Burns, MD2
M Eapen, MD, MSS Lee, MD, MPH3
M Pasquini, MD, MSJD Rizzo, MD, MSB Shaw, MD, PhDScientific DirectorM Arora, MD,MS1
A D’Souza, MBBS, MDM Hamadani, MD
P Hari, MD, MSM Riches, MD, MS4
W Saber, MD, MSE Thiel, MD, MS
Program Manager, Business
OfficeP Vespalec
Program Coordinator II
R DunnAdministrative
Assistant, SrK Jackson
AdministrativeAssistant
DL CampbellJ Claas
Program Manager,
SCTODC Doleysh
Meetings Program Manager
D Waldoch Snyder
Program Director, Statistics & Clinical Outcomes Research
W Pérez, MPH
Senior Biostatistician
J CarrerasM ChenH Wang
BiostatisticianK Bo-Subait
A DiGilioM FeiN He
M HemmerK Hu
J HuangH Millard
A St. MartinX Zhu
Program Coordinator IM Geronime
Director, ITE Bergman, MBA, MS
Database Administrator
B LiuProgrammer
Analyst IVH Tian
ProgrammerAnalyst IIIP Gengler
J GierDatabase Analyst III
X ZhangProgrammer
Analyst IIT Graczkowski
Database Analyst IM Desai
Metadata AnalystA KummerowManager IS I (part time)
C Zhang
Program Director, Advancement
S Fisher
Medical WriterJ Gillis-Smith
Program Coordinator II
M BreyM Simaytis
Communications SpecialistV Vlach
Program Director, Data Operations
J Brunner-Grady, PA-C
Manager, Data
OperationsS Meiers
Program Coordinator II
K BhavsarClinical Research
Coordinator IIA Prentice
T HuntProgram
Coordinator IA Pope
Clinical Research Coordinator I
A Benoit
Study Clinical Research
CoordinatorS Kulkarni
Clinical Research
Assistant IIIC AbelClinical
Research Assistant II
M PatelData EntryA Pereles
Program/IS ManagerT Moerke
Bus Systems Analyst Assoc
TBDInformation
Security AnalystJ RineckDesktop Support
TechnicianV Buendia
Grants and Contracts Financial Manager
C Gonzalez
1University of Minnesota2NMDP/Be The Match3Fred Hutchinson Cancer Research Center4University of North Carolina
CIBMTR 2015 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS
Page | 76
APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 1)
Chief Scientific DirectorMary M Horowitz, MD, MS
Associate Scientific DirectorD Confer, MD
Vice President, CIBMTR MinneapolisR King, MPH
Director, CIBMTR IT SystemsM Prestegaard
Data Solutions ManagerR Renner
Senior Metadata Analyst
W ZhangMetadata Analyst
M NychS Sorensen
Principal Data Architect
E ZinkBusiness Architect
B Burgess
Senior Manager, Program Management
K Gee
Principal Business Systems Analyst
E NarrSenior Business Systems Analyst
S EwerBusiness Systems
AnalystD Kloker
Project ManagerS FreemanK O’Connor
A PullB Wakaruk
Project CoordinatorDM Campbell
Solutions ManagerE Chan
Senior System Administrator
C YangBusiness Intelligence Solutions Architect
TBD Principal Software
EngineerC Giddegowda
Senior Software EngineerA GomezN HoodJ Smith
Software EngineerZ AhmedC JordahlG Ogega
Software Engineer ETLN AryalS Stagg
Associate System Administrator
A Westin
Quality Assurance ManagerV Yarra
Senior Data Analyst
D McDonellK Schaper
T WirthSenior Quality
Assurance AnalystB SambaQuality
Assurance Analyst
V MurukurthyJ Oakes
Director, Immunobiologyand Observational Research
S Spellman, MBS1
Senior Biostatistician
P ChitphakdiathaiM HaagensonBiostatistician
D KieferS Rouse
Principal Immunobiology
Research ScientistA Howard
C Vierra-GreenSenior
Immunobiology Research Scientist
M BrownImmunobiology
Research Scientist C Brady
D SchellerS Waldvogel
Associate Immunobiology
Research ScientistA Spahn
Project CoordinatorH Severance
AdministrativeSpecialistA Carlson
Director, Bioinformatics M Maiers, MS1
Senior Bioinformatics
ScientistsA MadboulyE Williams
Bioinformatics Scientists
S FingersonM Halagan
D Roe
1Also serves as Scientific Director
CIBMTR 2015 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS
Page | 77
APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 2)
Chief Scientific DirectorMary M Horowitz, MD, MS
Associate Scientific DirectorD Confer, MD
Vice President, CIBMTR MinneapolisR King, MPH
Senior Manager, Data ManagementM Matlack
Supervisor, Quality Control
B Levesque
Quality ControlSpecialistK Kutzner
Data EntryCoordinator II
L HorneData Entry
Coordinator IP Lee
K XongImaging Assistant II
N GibitzT Winder
Imaging AssistantJ Lund
Supervisor, Clinical Research,
Donor Data Management
A Hauck
Clinical Research Coordinator III
A Ewer Clinical Research
Coordinator IIR Krunkkala
K MalumS Tasky
Supervisor, Clinical Research,
Recipient DataManagement
K Gardner
Clinical Research Coordinator III
E JohnsonClinical Research
Coordinator IIA DraxlerA Mitsch
A MussetterK NutterC OlsonT Thole
Clinical Research Coordinator I
E MitchemP Wallace
Clinical Research
Coordinator IIIJ Bloomquist
S LoganE Love
Training & Development
SpecialistL Colt
Sr Research DeveloperM Unekis
TBD
Administrative AssistantM Ammi
Senior Manager,Monitoring & Auditing
D Christianson
Senior Clinical ResearchAssociateW Affield
A BirchM Petcoff
L Wendland Clinical Research
AssociateA Hendrickson
K KaneC Newcomb
K PhalenM ProueK Reilly
Clinical ResearchSpecialist
E EichAudit Coordinator
E Nordquist
Senior Manager,Prospective Research
R Drexler
Senior Clinical Research Specialist
A AdamsS Flesch
C JohnsonH Kobusingye
Clinical Research SpecialistR Besser
C PetroskeJ Vogel
Clinical Research Assistant
TBD
Prospective Research
CoordinatorJ Dworski
Survey Research Group
SupervisorD Mattila
Research Interviewer
C GriffithC Jacox
C KunakomS Lease
TBDSurvey
Research AssistantE Michem
Senior Human Research
Protection Specialist - IRB
T RouseSenior Human
Research Protection Specialist
C JobeHuman
Research Protection Specialist
J Tkachenko
Administrative SpecialistM Young
BMT CTN Project
ManagerA Foley
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 78
APPENDIX B3: COORDINATING CENTER LEADERSHIP
SENIOR LEADERSHIP
Mary M. Horowitz, MD, MS • Chief Scientific Director for the CIBMTR • Principal Investigator for the Data and Coordinating Center of the
BMT CTN • Research Director for the SCTOD • Robert A. Uihlein Professor of Hematologic Research at MCW • Chief of the Division of Hematology and Oncology at MCW • Attending physician in the MCW HCT program
Jeffrey Chell, MD • Executive Director for the CIBMTR • Chief Executive Officer of NMDP/Be The Match
Dennis Confer, MD • Associate Scientific Director for CIBMTR Minneapolis • Co-PI of the Data and Coordinating Center of the BMT CTN • Co-Scientific Director of the RCI BMT • Scientific Director of the Donor Health and Safety Working Committee • Chief Medical Officer of NMDP/Be The Match • Treasurer of the Executive Board of the Worldwide Network for Blood
and Marrow Transplantation
Daniel Weisdorf, MD • Senior Research Advisor for the CIBMTR • Scientific Director of the Acute Leukemia Working Committee • Vice President of the Worldwide Network for Blood and Marrow
Transplantation • Professor of Medicine at the University of Minnesota • Chief of the Division of Hematology, Oncology, and Transplant at the
University of Minnesota • Associate Chair of Clinical Research in the Department of Medicine at
the University of Minnesota • Attending physician in the University of Minnesota HCT program
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 79
Mei-Jie Zhang, PhD • Chief Statistical Director for the CIBMTR • Biostatistician for the Acute Leukemia and Graft Sources and
Manipulation Working Committees • Professor of Biostatistics at MCW
Mary Eapen, MBBS, MS • Senior Scientific Director of Research Operations for the CIBMTR • Scientific Director for the Graft Sources and Manipulation Working
Committee as well as the Primary Immune Deficiencies, Inborn Errors of Metabolism and Other Non-Malignant Marrow Disorders Working Committee
• Protocol Officer for several BMT CTN trials • Professor of Medicine at MCW • Attending physician in the MCW HCT program
J. Douglas Rizzo, MD, MS • Senior Scientific Director and Principal Investigator of the SCTOD for
the CIBMTR • Professor of Medicine at MCW • Associate Director of Clinical Operations for the Froedtert and MCW
Cancer Center • Attending physician in the MCW HCT program
Bronwen Shaw MD, PhD • Senior Scientific Director of Data Operations for the CIBMTR • Co-Scientific Director of the RCI BMT • Scientific Director of the Late Effects and Quality of Life Working
Committee as well as the Donor Health and Safety Working Committee
• Professor of Medicine at MCW • Attending physician in the MCW HCT program
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 80
Linda Burns, MD • Senior Scientific Director of the Health Services Research Program for
the CIBMTR • Co-Scientific Director of the RCI BMT • Vice President and Medical Director of NMDP/Be The Match • Immediate Past President of the American Society of Hematology
Marcelo Pasquini, MD, MS • Senior Scientific Director of CIBMTR Clinical Trials Support – BMT CTN
for the CIBMTR • Scientific Director for the Autoimmune Diseases and Cellular
Therapies Working Committee as well as the Regimen-Related Toxicity and Supportive Care Working Committee
• Protocol Officer and Director of Medical Monitors for the BMT CTN • CIBMTR representative to the Worldwide Network for Blood and
Marrow Transplantation • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program
Roberta King, MPH • Vice President for CIBMTR Minneapolis • Oversees the administrative, scientific, and statistical support
activities of CIBMTR Minneapolis, which include research administration, human subject protection program, data management, auditing and monitoring, observational research, prospective research, and IT
• Staff Liaison to the NMDP/Be The Match Donor and Patient Safety Monitoring Advisory Group
Patricia Steinert, PhD, MBA • Administrator for CIBMTR Milwaukee • Oversees the administrative, scientific, and statistical support
activities of CIBMTR Milwaukee, which include data operations, development, observational research and prospective research, IT and meetings
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 81
SCIENTIFIC DIRECTORS
Mukta Arora, MD, MBBS, MS • Scientific Director of the Graft-versus-Host Disease Working
Committee • Associate Professor of Medicine at the University of Minnesota • Attending physician in the University of Minnesota HCT program
Anita D’Souza, MD • Assistant Scientific Director of the Plasma Cell Disorders and Adult
Solid Tumors Working Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program
Parameswaran Hari, MD, MS • Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors
Working Committee • Armand J. Quick – William F. Stapp Professor of Hematology at MCW • Section Head of Hematologic Malignancies and Adult BMT Program at
MCW • Attending physician in the MCW HCT program
Mehdi Hamadani, MD • Scientific Director of the Lymphoma Working Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 82
Stephanie J. Lee, MD, MPH • Senior Scientific Director of Immunobiology Research for the CIBMTR • Scientific Director of the Immunobiology Working Committee • Professor of Medicine at the University of Washington • Attending physician in the Fred Hutchinson Cancer Research Center
HCT program
Martin Maiers, MS • Director of Bioinformatics Research for the CIBMTR • Staff liaison to the NMDP/Be The Match Histocompatibility Advisory
Group (also known as the CIBMTR Immunobiology Steering Committee)
• Co-Chair of Informatics: International Histocompatibility and Immunogenetics Workshop
• Member of World Health Organization HLA Nomenclature Committee
Marcie Riches, MD, MS • Scientific Director of the Infection and Immune Reconstitution
Working Committee • Protocol Officer and Medical Monitor for several BMT CTN trials • Clinical Associate Professor of Medicine at the University of North
Carolina at Chapel Hill • Director of BMT Clinical Research and Data Quality at the University of
North Carolina at Chapel Hill • Attending physician in the University of North Carolina Lineberger
Comprehensive Cancer Center HCT program
Wael Saber, MD, MS • Scientific Director of the Chronic Leukemia and Health Services and
International Studies Working Committees • Assistant Scientific Director of the Acute Leukemia Working
Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 83
Stephen Spellman, MBS • Scientific Director of the Graft-versus-Host Disease and
Immunobiology Working Committees • Director of Immunobiology and Observational Research for the
CIBMTR • Principal Investigator for the Research Repository • Staff liaison to the NMDP/Be The Match Cord Blood Advisory Group
Research subcommittee and Histocompatibility Advisory Group (also known as the CIBMTR Immunobiology Steering Committee)
• Program Manager for the NMDP/Be The Match Office of Naval Research Grant
Elizabeth Thiel, MD, MS • Scientific Director of the Pediatric Cancer Working Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW Palliative Care program
STATISTICAL DIRECTORS
Kwang Woo Ahn, PhD • Biostatistician for the Chronic Leukemia, Infection and Immune
Reconstitution, and Lymphoma Working Committees • Associate Professor of Biostatistics at MCW
Ruta Brazauskas, PhD • Biostatistician for the Health Services and International Studies
Working Committee as well as the Late Effects and Quality of Life and Autoimmune Disorders and Cellular Therapies Working Committees
• Assistant Professor of Biostatistics at MCW
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 84
Brent Logan, PhD • Biostatistician for the Donor Health and Safety Working Committee
and the Regimen-Related Toxicity and Supportive Care Working Committee
• Lead Statistician for the BMT CTN and Statistical Consultant to the NMDP/Be The Match
• Professor of Biostatistics at MCW • Director of the Division of Biostatistics at MCW
Tao Wang, PhD • Biostatistician for the Graft-versus-Host Disease and Immunobiology
Working Committees • Associate Professor of Biostatistics at MCW
OTHER LEADERSHIP STAFF
Erik Bergman, MBA, MS • Director of IT for the CIBMTR in Milwaukee • Leads the IT staff in Milwaukee, which is organized in four teams:
Database, Applications Development, Technology Services, and Project Management
• Oversees management of the Research Database, including extraction of data from source systems as well as their transformation and loading to the database
• Responsible for data retrievals from the Research Database as well as key solutions for sharing data with stakeholders
Janet Brunner-Grady, PA-C • Program Director of Data Operations for the CIBMTR • Manages the clinical research coordinators, develops training
programs, and monitors transplant center CPI • Assists clinical research coordinators on both campuses with clinical
transplant-related questions
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 85
Debra Christianson • Senior Manager of Auditing and Monitoring for the CIBMTR • Oversees the monitoring program for RCI BMT clinical trials and the
on-site source document audits on data submitted to the Research Database
• Responsible for the FormsNet Instruction Manual
Rebecca Drexler • Senior Manager of Prospective Research for the CIBMTR • Manages the activities of the RCI BMT, including the Survey Research
Group • Oversees the administration of the Clinical Trials Advisory Committee
Sherry Fisher • Director of Advancement for the CIBMTR • Manages the advancement activities and the Corporate Program,
which generates new revenue to create a continued source of non-federal financial support
• Oversees meetings and communications activities, including the annual BMT Tandem Meetings
Marie Matlack • Senior Manager of Data Management for the CIBMTR • Manages clinical research coordinators and senior research
programmers as well as data entry and imaging staff • Oversees form revision and development and the CPI program • Collaborates with the Minneapolis IT staff to develop enhancements
to FormsNet
CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL
Page | 86
Waleska S. Pérez, MPH • Program Director of Statistics and Clinical Outcomes Research for the
CIBMTR • Oversees the Master’s-level statisticians of CIBMTR Milwaukee • Provides administrative oversight of the Clinical Outcomes Research
Program
Matt Prestegaard • Director of IT for the CIBMTR in Minneapolis • Leads the IT staff in Minneapolis, including project managers,
programmer analysts, business systems and data analysts, quality assurance analysts, metadata analysts, and managers
• Develops, implements, and supports CIBMTR electronic data management, capture, and messaging systems (FormsNet and AGNIS); data marts and data warehouses; infrastructure for these applications; and the curation of common data elements within the Cancer Data Standards Registry and Repository
CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP
Page | 87
APPENDIX C: COMMITTEE MEMBERSHIP
CIBMTR committees provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of its scientific and medical communities. All committees and their functions are listed in Table 1.3.
APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP
The Advisory Committee provides oversight for CIBMTR policies and scientific agenda and also partners with the Working Committees to prioritize scientific studies. Members are elected to three-year terms by the CIBMTR Assembly and must be from qualifying CRF transplant centers. All Advisory Committee terms begin on March 1.
ELECTED MEMBERS
Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
Immediate Past Chair Thomas Shea, MD, University of North Carolina at Chapel Hill, NC
VICE CHAIRS
North America Helen Heslop, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX
Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, Birmingham, United Kingdom
Central / South America Carmem Bonfim, MD, Hospital de Clinicas – UFPR, Curitiba, Brazil
Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia
MEMBERS AT LARGE
North America Steven Pavletic, MD, MS, NIH-NCI Experimental Transplantation and Immunology Branch, Bethesda, MD
Joseph Pidala, MD, MS, H Lee Moffitt Cancer Center and Research Institute, Tampa, FL
Michael Pulsipher, MD, Primary Children’s Hospital, Salt Lake City, UT
Bipin Savani, MD, Vanderbilt University Medical Center, Brentwood, TN
Marcel van den Brink, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY
Kirsten Williams, MD, Children’s National Medical Center, Washington, DC
Non-North America Ernst Holler, MD, Klinikum der Universitaet Regensburg, Germany
William Hwang, MBBS, MRCP, Singapore General Hospital, Singapore
Shinichiro Okamoto, MD, PhD, Keio University, Shinjuku-ku Tokyo, Japan
CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP
Page | 88
Miguel Sanz, Hospital Universitario La Fe, Valencia, Spain
Alok Srivastava, MD, Christian Medical College, Vellore, India
Afonso Vigorito, MD, PhD, Unicamp – Hemocentro, Campinas, Brazil
APPOINTED MEMBERS
ASBMT Representative Christopher Bredeson, MD, MSc, The Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Canada
Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA
Business Representative Theresa Franco, MSN, BSN, Nebraska Medicine, Omaha, NE
Collection Center Representative Lee Ann Weitekamp, MD, Michigan Blood Cord Blood Bank, Grand Rapids, MI
Cord Blood Bank Representative Elizabeth Shpall, MD, MD Anderson Cord Blood Bank, Houston, TX
Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN
Patient / Family Representatives James Omel, MD, Chair, Consumer Advocacy Committee
Maureen Beaman, MBA, Chair, Consumer Advocacy Committee
EX OFFICIO MEMBERS
Executive Director Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN
Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI
Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI
Senior Scientific Director for SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI
Associate Scientific Director CIBMTR Minneapolis Dennis Confer, MD, CIBMTR, Minneapolis, MN
Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN
Research Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI
Vice President CIBMTR Minneapolis Roberta King, CIBMTR, Minneapolis, MN
Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN
NMDP / MCW / HRSA Contracting Officer Representative
Shelley Grant, MHSA, Rockville, MD
NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret)
MCW / HRSA Contracting Officer Representative
Christine Nishiguchi, MS, MPH, Rockville, MD
CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP
Page | 89
MCW / NCI Project Officer Roy Wu, PhD, Bethesda, MD
MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD
MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD
Nominating Committee Chair Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
CIBMTR 2015 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP
Page | 90
APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP
The Executive Committee, a subcommittee of the Advisory Committee, ensures that the organization carries out its mission and adheres to CIBMTR policies and procedures; it also provides advice and counsel to the Coordinating Center between meetings of the Advisory Committee. Specifically, the Executive Committee is responsible for providing scientific and policy advice to the Chief Scientific Director and Coordinating Center, reviewing audit results and making recommendations for improvement, and appointing a CIBMTR Co-Chair and additional CIBMTR representatives to the BMT Tandem Meetings Scientific Organizing Committee for the annual BMT Tandem Meetings. All Executive Committee terms begin on March 1.
ELECTED MEMBERS
Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
Immediate Past Chair Thomas Shea, MD, University of North Carolina at Chapel Hill, NC
VICE CHAIRS
North America Helen Heslop, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX
Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, Birmingham, United Kingdom
Central / South America Carmem Bonfim, MD, Hospital de Clinicas – UFPR, Curitiba, Brazil
Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia
APPOINTED MEMBERS
ASBMT Representative Christopher Bredeson, MD, MSc, The Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Canada
Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA
Business Representative Theresa Franco, MSN, BSN, Nebraska Medicine, Omaha, NE
Collection Center Representative Lee Ann Weitekamp, MD, Michigan Blood Cord Blood Bank, Grand Rapids, MI
Cord Blood Bank Representative Elizabeth Shpall, MD, MD Anderson Cord Blood Bank, Houston, TX
Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN
Patient / Family Representatives James Omel, MD, Chair, Consumer Advocacy Committee
Maureen Beaman, MBA, Chair, Consumer Advocacy Committee
CIBMTR 2015 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP
Page | 91
EX OFFICIO MEMBERS
Executive Director Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN
Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI
Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI
Senior Scientific Director for SCTOD
J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI
Associate Scientific Director CIBMTR Minneapolis
Dennis Confer, MD, CIBMTR, Minneapolis, MN
Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN
Research Administrator CIBMTR Milwaukee
Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI
Vice President CIBMTR Minneapolis
Roberta King, CIBMTR, Minneapolis, MN
Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN
NMDP / MCW /HRSA Contracting Officer Representative
Shelley Grant, MHSA, Rockville, MD
NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret)
MCW / HRSA Contracting Officer Representative
Christine Nishiguchi, MS, MPH, Rockville, MD
MCW / NCI Project Officer Roy Wu, PhD, Bethesda, MD
MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD
MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD
Nominating Committee Chair Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
CIBMTR 2015 Annual Report APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP
Page | 92
APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP
The Consumer Advocacy Committee communicates research results and data to the non-medical community and provides patient and donor perspectives during the development of the CIBMTR research agenda. Many committee members have personal experience as a donor, recipient, or family member of a recipient.
CHAIRS James Omel, MD
Maureen Beaman, MBA
MEMBERS
Beatrice Abetti, MSW, Leukemia and Lymphoma Society
Jack Aiello, MS
Gerardo Camarillo, JD, MD Anderson Cancer Center
Jeffrey Haertling, ARK Air Express
Hilary Hall, Dana-Farber Cancer Institute
Barry Schatz, Cardinal Bernardin Cancer Center, Loyola University Medical Center
Kristin Scheeler, University of Wisconsin Hospital and Clinics
SCIENTIFIC DIRECTOR
J. Douglas Rizzo, MD, MS, CIBMTR
EX OFFICIO MEMBERS
Robyn Ashton, RN, MSN, HRSA
Jeffrey Chell, MD, NMDP/Be The Match
Dennis Confer, MD, NMDP/Be The Match
Ellen Denzen, (NMDP/Be The Match liaison) NMDP/Be The Match
Carol Doleysh, (CIBMTR liaison) CIBMTR
Rebecca Drexler, CIBMTR
Jessica Gillis-Smith, MPH, CIBMTR
Shelley Grant, MHSA, HRSA
Darlene Haven, NMDP/Be The Match
Mary Horowitz, MD, MS, CIBMTR
Elizabeth Murphy, EdD, RN, (NMDP/Be The Match liaison) NMDP/Be The Match
Christine Nishiguchi, MS, MPH, HRSA
Patricia Steinert, PhD, MBA, CIBMTR
CIBMTR 2015 Annual Report APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP
Page | 93
APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP
The Nominating Committee consists of five members elected by the CIBMTR Assembly. Following an annual call for nominations, the Nominating Committee prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees. Elections are held annually by confidential electronic ballot. The Nominating Committee also makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments after seeking recommendations from the CIBMTR Assembly, Advisory Committee, and incumbent Working Committee Chairs. All terms begin on March 1.
CHAIR
Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
MEMBERS
Daniel Couriel, MD, University of Michigan, Ann Arbor, MI
Richard Champlin, MD, MD Anderson Cancer Center, Houston TX
Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
David Vesole, MD, PhD, Hackensack University Medical Center, Hackensack, NJ
CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
Page | 94
APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
For information on Scientific Working Committee structure and organization, see Section 1.3.1. For information on Working Committee studies and their accomplishments, see Section 2.1.1.
ACUTE LEUKEMIA WORKING COMMITTEE
Chairs Marcos de Lima, MD, University Hospitals Case Medical Center Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Hanna Khoury, MD, Emory University Hospital
Scientific Director Daniel Weisdorf, MD, CIBMTR Asst Scientific Dir Wael Saber, MD, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Hailin Wang, MPH, CIBMTR
AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE
Chairs Mitchell Cairo, MD, New York Medical College Ian Lewis, MBBS, PhD, Royal Adelaide Hospital David McKenna, MD, University of Minnesota Medical Center, Fairview
Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Kyle Bo-Subait, MPH, CIBMTR
CHRONIC LEUKEMIA WORKING COMMITTEE
Chairs Edwin Alyea, MD, Dana Farber Cancer Institute Uday Popat, MD, MD Anderson Cancer Center Ronald Sobecks, MD, Cleveland Clinic Foundation
Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Zhenhuan Hu, MS, CIBMTR
DONOR HEALTH AND SAFETY WORKING COMMITTEE
Chairs Paul O’Donnell, MD, PhD, Fred Hutchinson Cancer Research Center Michael Pulsipher, MD, Primary Children’s Hospital Galen Switzer, PhD, University of Pittsburgh Medical Center - Cancer Center Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Ex Officio Sr Advisor Dennis Confer, MD, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statisticians Deidre Kiefer, MPH, CIBMTR Pintip Chitphakdithai, PhD, CIBMTR Consumer Advocacy Committee Representatives
Beatrice Abetti, MSW, Leukemia and Lymphoma Society Maureen Beaman, MBA Jeffrey Haertling, ARK Air Express
CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
Page | 95
GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE
Chairs Miguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center Vanderson Rocha, MD, PhD, Churchill Hospital Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside
Hospital Scientific Director Mary Eapen, MBBS, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Andrew St. Martin, MS, CIBMTR
GRAFT-VERSUS-HOST DISEASE WORKING COMMITTEE
Chairs Daniel Couriel, MD, University of Michigan Amin Alousi, MD, MD Anderson Cancer Center Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute
Scientific Directors Mukta Arora, MD, MS, CIBMTR Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Hemmer, MS, CIBMTR Consumer Advocacy Committee Representatives
Hilary Hall, Dana-Farber Cancer Institute James Omel, MD
HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE
Chairs Yoshiko Atsuta, MD, PhD, Nagoya University Graduate School of Medicine Carmem Bonfim, MD, Hospital de Clinicas Curitiba Jignesh Dalal, MD, The Children’s Mercy Hospitals and Clinics Theresa Hahn, PhD, Roswell Park Cancer Institute Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital
Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Naya He, MPH, CIBMTR Consumer Advocacy Committee Representatives
Jack Aiello, MS Gerardo Camarillo, JD, MD Anderson Cancer Center
IMMUNOBIOLOGY WORKING COMMITTEE
Chairs Michael Verneris, MD, University of Minnesota Medical Center, Fairview Katharina Fleischhauer, MD, Universitätsklinikum Essen KMT Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center
Scientific Directors Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research Center Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Haagenson, MS, CIBMTR
CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
Page | 96
INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE
Chairs Jeffery Auletta, MD, Nationwide Children’s Hospital Caroline Lindemans, MD, PhD, University Medical Center Utrecht Krishna Komanduri, MD, University of Miami
Scientific Director Marcie Riches, MD, MS, CIBMTR, H Lee Moffitt Cancer Center and Research Institute
Statistical Directors Kwang Woo Ahn, PhD, CIBMTR Soyoung Kim, PhD, CIBMTR
Statistician Min Chen, MS, CIBMTR
LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE
Chairs Bipin Savani, MD, Vanderbilt University Medical Center Mary Flowers, MD, Fred Hutchinson Cancer Research Center Minoo Battiwalla, MD, MS, National Heart Lung and Blood Institute - NIH
Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR Consumer Advocacy Committee Representative
Barry Schatz, Loyola University Medical Center
LYMPHOMA WORKING COMMITTEE
Chairs Sonali Smith, MD, University of Chicago Hospitals Anna Sureda, MD, PhD Timothy Fenske, MD, MS, Froedtert Memorial Lutheran Hospital
Scientific Director Mehdi Hamadani, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Alyssa DiGilio, MS, CIBMTR
PEDIATRIC CANCER WORKING COMMITTEE
Chairs Gregory Hale, MD, All Children’s Hospital Parinda Mehta, MD, Cincinnati Children's Hospital Medical Center Angela Smith, MD, MS, University of Minnesota Medical Center, Fairview
Scientific Director Elizabeth Thiel, MS, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR
CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
Page | 97
PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE
Chairs Amrita Krishnan, MD, City of Hope National Medical Center Cristina Gasparetto, MD, Duke University Medical Center Yago Nieto, MD, PhD, MD Anderson Cancer Center Tomer Mark, MD, New York Presbyterian Hospital at Cornell
Scientific Director Parameswaran Hari, MD, MS, CIBMTR Asst Scientific Dir Anita D’Souza, MD, CIBMTR Statistical Directors Mei-Jie Zhang, PhD, CIBMTR Raphael Fraser, PhD, CIBMTR Statistician Jiaxing Huang, MS, CIBMTR Consumer Advocacy Committee Representative James Omel, MD
PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND OTHER NON-MALIGNANT MARROW DISORDERS WORKING COMMITTEE
Chairs Paolo Anderlini, MD, MD Anderson Cancer Center Neena Kapoor, MD, Children’s Hospital of Los Angeles Jaap-Jan Boelens, MD, PhD, University Medical Center Utrecht Vikram Mathews, MD, Christian Medical College Hospital
Scientific Director Mary Eapen, MBBS, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician TBD
REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE
Chairs Andrew Artz, MD, MS, University of Chicago Hospitals Alison Loren, MD, MS, Abramson Cancer Center University of Pennsylvania
Medical Center Shin Mineishi, MD, University of Alabama at Birmingham
Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statistician Xiaochun Zhu, MS, CIBMTR
CIBMTR 2015 Annual Report APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP
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APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP
The NMDP/Be The Match Histocompatibility Advisory Group also serves as the CIBMTR Immunobiology Steering Committee. This committee reviews and approves the use of donor-recipient specimens from the Research Repository in CIBMTR studies.
CHAIR
Carolyn K. Hurley, PhD, Diplomate ABHI, Georgetown University Hospital
ADVISORY GROUP MEMBERS
Juliet Barker, MD, Memorial Sloan Kettering Cancer Center
Sarah Cooley, MD, University of Minnesota
Mary Eapen, MD, MS, Medical College of Wisconsin
Marcelo Fernandez-Viña, PhD, Stanford Hospital and Clinics
Brent Logan, PhD, Medical College of Wisconsin
Carlheinz Mueller, MD, PhD, German National Bone Marrow Donor Registry (ZKRD)
Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center
David Porter, MD, Abramson Cancer Center University of Pennsylvania Medical Center
Raja Rajalingham, PhD, University of California, San Francisco
Bronwen Shaw, MD, PhD, Medical College of Wisconsin
EX OFFICIO MEMBERS
Dennis Confer, MD, NMDP/Be The Match
Jason Dehn, MPH, NMDP/Be The Match
Karen Dodson, NMDP/Be The Match
Melissa Greenwald, MD, HRSA
Robert Hartzman, MD, Capt. MC, USN (Ret.), Navy Representative, C.W. Bill Young Marrow Donor Recruitment and Research Program
Martin Maiers, CIBMTR
Stephen Spellman, MBS, CIBMTR
John Wingard, MD, NMDP/Be The Match Board Chair
CIBMTR 2015 Annual Report APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP
Page | 99
APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP
The Clinical Trials Advisory Committee assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT (Section 2.3.2).
CHAIR
John Levine, MD, MS, Mount Sinai Medical Center
MEMBERS
Colleen Delaney, MD, MSc, Fred Hutchinson Cancer Research Center
Marcos de Lima, MD, University Hospitals Case Medical Center
Hugo Fernandez, MD, H Lee Moffitt Cancer Center and Research Institute
John Koreth, MBBS, DPhil, Dana Farber Cancer Institute
Mark Litzow, MD, Mayo Clinic Rochester
Margaret MacMillan, MD, MSc, University of Minnesota Medical Center, Fairview
Sophie Paczesny, MD, PhD, Indiana University Hospital/Riley Hospital for Children
Katayoun Rezvani, MD, PhD, M.D. Anderson Cancer Center
Jan Storek, MD, PhD, University of Calgary
APPOINTED MEMBERS
Maureen Beaman, MBA
James Omel, MD
EX OFFICIO MEMBERS
Dennis Confer, MD, CIBMTR
Rebecca Drexler, BS, CIBMTR
Robert Hartzman, MD, Capt MC, USN (Ret.)
Mary Horowitz, MD, MS, CIBMTR
Roberta King, CIBMTR
Brent Logan, PhD, CIBMTR
Nancy Poland, MA, NMDP/Be The Match Operations
Marcie Riches, MD, MS, The University of North Carolina at Chapel Hill
Kevin Weber, NMDP/Be The Match Operations
Daniel Weisdorf, MD, CIBMTR
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 100
APPENDIX D: PUBLICATIONS
The PMCID number is assigned by PubMed Central, the NIH’s free digital archive of biomedical and life sciences journal literature, and is in compliance with the NIH policy on public access.
APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
The following publications were generated by Scientific Working Committees within the Clinical Outcomes Research Program. For more information about the Working Committees, see Section 2.1.1.
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Authors Title Citation PMCID
Logan AC, Wang Z, Alimoghaddam K, Wong RM, Lai T, Negrin RS, Grumet C, Logan BR, Zhang M-J, Spellman SR, Lee SJ, Miklos DB
ABO mismatch is associated with increased nonrelapse mortality after allogeneic hematopoietic cell transplantation
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):746-754. doi:10.1016/j.bbmt. 2014.12.036. Epub 2015 Jan 5
PMC4363312
Gadalla SM, Wang T, Haagenson M, Spellman SR, Lee SJ, Williams KM, Wong JY, De Vivo I, Savage SA
Association between donor leukocyte telomere length and survival after unrelated allogeneic hematopoietic cell transplantation for severe aplastic anemia
JAMA: The Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. doi:10.1001/ jama.2015.7. Epub 2015 Feb 10
PMC4388056
Chen Y-B, Lane AA, Logan BR, Zhu X, Akpek G, Aljurf MD, Artz AS, Bredeson CN, Cooke KR, Ho VT, Lazarus HM, Olsson RF, Saber W, McCarthy PL, Pasquini MC
Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous hematopoietic cell transplantation
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046-1053. doi:10.1016/ j.bbmt.2015.02.005. Epub 2015 Feb 13
PMC4426014
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
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SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Veys PA, Nanduri V, Baker KS, He W, Bandini G, Biondi A, Dalissier A, Davis JH, Eames GM, Egeler RM, Filipovich AH, Fischer A, Jürgens H, Krance R, Lanino E, Leung WH, Matthes S, Michel G, Orchard PJ, Pieczonka A, Ringdén O, Schlegel PG, Sirvent A, Vettenranta K, Eapen M
Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning
British Journal of Haematology. 2015 Jun 1; 169(5):711-718. doi:10.1111/bjh.13347. Epub 2015 Mar 27
PMC4433436
Inamoto Y, Shah NN, Savani BN, Shaw BE, Abraham AA, Ahmed IA, Akpek G, Atsuta Y, Baker KS, Basak GW, Bitan M, DeFilipp Z, Gregory TK, Greinix HT, Hamadani M, Hamilton BK, Hayashi RJ, Jacobsohn DA, Kamble RT, Kasow KA, Khera N, Lazarus HM, Malone AK, Lupo-Stanghellini MT, Margossian SP, Muffly LS, Norkin M, Ramanathan M, Salooja N, Schoemans H, Wingard JR, Wirk B, Wood WA, Yong A, Duncan CN, Flowers MED, Majhail NS
Secondary solid cancer screening following hematopoietic cell transplantation
Bone Marrow Transplantation. 2015 Aug 1; 50(8):1013-1023. doi:10.1038/ bmt.2015.63. Epub 2015 Mar 30
N/A
Holter-Chakrabarty JL, Pierson N, Zhang M-J, Zhu X, Akpek G, Aljurf MD, Artz AS, Baron F, Bredeson CN, Dvorak CC, Epstein RB, Lazarus HM, Olsson RF, Selby GB, Williams KM, Cooke KR, Pasquini MC, McCarthy PL
The sequence of cyclophosphamide and myeloablative total body irradiation in hematopoietic cell transplantation for patients with acute leukemia
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1251-1257. doi:10.1016/j.bbmt. 2015.03.017. Epub 2015 Mar 31
PMC4465990
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 102
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Sorror ML, Logan BR, Zhu X, Rizzo JD, Cooke KR, McCarthy PL, Ho VT, Horowitz MM, Pasquini MC
Prospective validation of the predictive power of the hematopoietic cell transplantation comorbidity index: a Center for International Blood and Marrow Transplant Research study
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1479-1487. doi:10.1016/ j.bbmt.2015.04.004. Epub 2015 Apr 7
PMC4512746
Mehta PA, Zhang M-J, Eapen M, He W, Seber A, Gibson B, Camitta BM, Kitko CL, Dvorak CC, Nemecek ER, Frangoul HA, Abdel-Azim H, Kasow KA, Lehmann L, Gonzalez Vicent M, Diaz Pérez MA, Ayas M, Qayed M, Carpenter PA, Jodele S, Lund TC, Leung WH, Davies SM
Transplant outcomes for children with hypodiploid acute lymphoblastic leukemia
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1273-1277. doi:10.1016/j.bbmt. 2015.04.008. Epub 2015 Apr 10
PMC4465998
Olsson RF, Logan BR, Chaudhury S, Zhu X, Akpek G, Bolwell BJ, Bredeson CN, Dvorak CC, Gupta V, Ho VT, Lazarus HM, Marks DI, Ringdén OTH, Pasquini MC, Schriber JR, Cooke KR
Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies
Leukemia. 2015 Aug 1; 29(8):1754-1762. doi:10.1038/leu. 2015.75. Epub 2015 Apr 24
PMC4527886
Goyal SD, Zhang MJ, Wang HL, Akpek G, Copelan EA, Freytes C, Gale RP, Hamadani M, Inamoto Y, Kamble RT, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Reshef R, Ritchie DS, Saber W, Savani BN, Seber A, Shea TC, Tallman MS, Wirk B, Bunjes DW, Devine SM, de Lima M, Weisdorf DJ, Uy GL
Allogeneic hematopoietic cell transplant for AML: no impact of pre-transplant extramedullary disease on outcome
Bone Marrow Transplantation. 2015 Aug 1; 50(8):1057-1062. doi:10.1038/ bmt.2015.82. Epub 2015 Apr 27
PMC4527880
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 103
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Sobecks RM, Wang T, Askar M, Gallagher MM, Haagenson M, Spellman S, Fernandez-Vina M, Malmberg K-J, Müller C, Battiwalla M, Gajewski J, Verneris MR, Ringdén O, Marino S, Davies S, Dehn J, Bornhäuser M, Inamoto Y, Woolfrey A, Shaw P, Pollack M, Weisdorf D, Milller J, Hurley C, Lee SJ, Hsu K
Impact of KIR and HLA genotypes on outcomes after reduced-intensity conditioning hematopoietic cell transplantation
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1589-1596. doi:10.1016/ j.bbmt.2015.05.002. Epub 2015 May 8
PMC4537837
Bachanova V, Burns LJ, Ahn KW, Laport GG, Akpek G, Kharfan-Dabaja MA, Nishihori T, Agura E, Armand P, Jaglowski SM, Cairo MS, Cashen AF, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Ghosh N, Holmberg LA, Inwards DJ, Kanate AS, Lazarus HM, Malone AK, Munker R, Mussetti A, Norkin M, Prestidge TD, Rowe JM, Satwani P, Siddiqi T, Stiff PJ, William BM, Wirk B, Maloney DG, Smith SM, Sureda AM, Carreras J, Hamadani M
Impact of pretransplantation 18F-fluorodeoxy glucose-positron emission tomography status on outcomes after allogeneic hematopoietic cell transplantation for non-Hodgkin Lymphoma
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1605-1611. doi:10.1016/ j.bbmt.2015.05.007. Epub 2015 May 14
PMC4558181
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 104
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Urbano-Ispizua A, Pavletic SZ, Flowers ME, Klein JP, Zhang M-J, Carreras J, Montoto S, Perales M-A, Aljurf MD, Akpek G, Bredeson CN, Costa LJ, Dandoy C, Freytes CO, Fung HC, Gale RP, Gibson J, Hamadani M, Hayashi RJ, Inamoto Y, Inwards DJ, Lazarus HM, Maloney DG, Martino R, Munker R, Nishihori T, Olsson RF, Rizzieri DA, Reshef R, Saad A, Savani BN, Schouten HC, Smith SM, Socié G, Wirk B, Yu LC, Saber W
The impact of graft-versus-host disease on the relapse rate in patients with lymphoma depends on the histological subtype and the intensity of the conditioning regimen
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1746-1753. doi:10.1016/ j.bbmt.2015.05.010. Epub 2015 May 15
PMC4568162
Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, Al-Seraihy A, Ayas M, Bonfim CM, Boulad F, Lund T, Buchbinder DK, Kapoor N, O'Brien TA, Diaz Perez MA, Veys PA, Eapen M
Hematopoietic stem cell transplantation for infantile osteopetrosis
Blood. 2015 Jul 9; 126(2):270-276. doi:10.1182/blood-2015-01-625541. Epub 2015 May 26
PMC4497967
Hahn T, Sucheston-Campbell LE, Preus L, Zhu X, Hansen JA, Martin PJ, Yan L, Liu S, Spellman S, Tritchler D, Clay A, Onel K, Pasquini M, McCarthy PL
Establishment of definitions and review process for consistent adjudication of cause-specific mortality after allogeneic unrelated-donor hematopoietic cell transplantation
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1679-1686. doi:10.1016/ j.bbmt.2015.05.019. Epub 2015 May 29
PMC4537799
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 105
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Inamoto Y, Flowers MED, Wang T, Urbano-Ispizua A, Hemmer MT, Cutler CS, Couriel DR, Alousi AM, Antin JH, Gale RP, Gupta V, Hamilton BK, Kharfan-Dabaja MA, Marks DI, Ringdén OTH, Socié G, Solh MM, Akpek G, Cairo MS, Chao NJ, Hayashi RJ, Nishihori T, Reshef R, Saad A, Shah A, Teshima T, Tallman MS, Wirk B, Spellman SR, Arora M, Martin PJ
Tacrolimus versus cyclosporine after hematopoietic cell transplantation for acquired aplastic anemia
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1776-1782. doi:10.1016/ j.bbmt.2015.05.023. Epub 2015 May 30
PMC4568149
Verneris MR, Lee SJ, Ahn KW, Wang H-L, Battiwalla M, Inamoto Y, Fernandez-Vina MA, Gajewski J, Pidala J, Munker R, Aljurf M, Saber W, Spellman S, Koreth J
HLA mismatch Is associated with worse outcomes after unrelated donor reduced-intensity conditioning hematopoietic cell transplantation: an analysis from the Center for International Blood and Marrow Transplant Research
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1783-1789. doi:10.1016/ j.bbmt.2015.05.028. Epub 2015 Jun 6
PMC4568127
Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ
Comparison of characteristics and outcomes of trial participants and nonparticipants: example of Blood and Marrow Transplant Clinical Trials Network 0201 trial
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1815-1822. doi:10.1016/ j.bbmt.2015.06.004. Epub 2015 Jun 11
PMC4568172
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 106
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Ayas M, Eapen M, Le-Rademacher J, Carreras J, Abdel-Azim H, Alter BP, Anderlini P, Battiwalla M, Bierings M, Buchbinder DK, Bonfim C, Camitta BM, Fasth AL, Gale RP, Lee MA, Lund TC, Myers KC, Olsson RF, Page KM, Prestidge TD, Radhi M, Shah AJ, Schultz KR, Wirk B, Wagner JE, Deeg HJ
Second allogeneic hematopoietic cell transplantation for patients with Fanconi anemia and bone marrow failure
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1790-1795. doi:10.1016/ j.bbmt.2015.06.012. Epub 2015 Jun 23
PMC4568139
Shaw BE, Logan BR, Kiefer DM, Chitphakdithai P, Pedersen TL, Abdel-Azim H, Abidi MH, Akpek G, Diaz MA, Artz AS, Dandoy C, Gajewski JL, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Liesveld JL, Majhail NS, O'Donnell PV, Olsson RF, Savani BN, Schears RM, Stroncek DF, Switzer GE, Williams EP, Wingard JR, Wirk BM, Confer DL, Pulsipher MA
Analysis of the effect of race, socioeconomic status, and center size on unrelated National Marrow Donor Program donor outcomes: donor toxicities are more common at low-volume bone marrow collection centers
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1830-1838. doi:10.1016/ j.bbmt.2015.06.013. Epub 2015 Jun 23
PMC4568129
Ciurea SO, Zhang M-J, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales M-A, Pingali SR, Porter DL, Riches MR, Ringdén OTH, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M
Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia
Blood. 2015 Aug 20; 126(8):1033-1040. doi:10.1182/blood-2015-04-639831. Epub 2015 Jun 30
PMC4543223
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
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SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Satwani P, Ahn KW, Carreras J, Abdel-Azim H, Cairo MS, Cashen A, Chen AI, Cohen JB, Costa LJ, Dandoy C, Fenske TS, Freytes CO, Ganguly S, Gale RP, Ghosh N, Hertzberg MS, Hayashi RJ, Kamble RT, Kanate AS, Keating A, Kharfan-Dabaja MA, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Prestidge TD, Rolon JM, Savani BN, Vose JM, Wood WA, Inwards DJ, Bachanova V, Smith SM, Maloney DG, Sureda A, Hamadani M
A prognostic model predicting autologous transplantation outcomes in children, adolescents and young adults with Hodgkin lymphoma
Bone Marrow Transplantation. 2015 Nov 1; 50(11):1416-1423. doi:10.1038/ bmt.2015.177. Epub 2015 Aug 3
PMC4633349
Klyuchnikov E, Bacher U, Kröger NM, Hari PN, Ahn KW, Carreras J, Bachanova V, Bashey A, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Hertzberg MS, Holmberg LA, Kharfan-Dabaja MA, Klein A, Ku GH, Laport GG, Lazarus HM, Miller AM, Mussetti A, Olsson RF, Slavin S, Usmani SZ, Vij R, Wood WA, Maloney DG, Sureda AM, Smith SM, Hamadani M
Reduced-intensity allografting as first transplantation approach in relapsed/refractory grades one and two follicular lymphoma provides improved outcomes in long-term survivors
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2091-2099. doi:10.1016/ j.bbmt.2015.07.028. Epub 2015 Aug 4
PMC4639453
Petersdorf EW, Malkki M, O'hUigin C, Carrington M, Gooley T, Haagenson MD, Horowitz MM, Spellman SR, Wang T, Stevenson P
High HLA-DP expression and graft-versus-host disease
New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. doi:10.1056/ NEJMoa1500140. Epub 2015 Aug 13
PMC4560117
Knight JM, Rizzo JD, Logan BR, Wang T, Arevalo JM, Ma J, Cole SW
Low socioeconomic status, adverse gene expression profiles, and clinical outcomes in hematopoietic stem cell transplant recipients
Clinical Cancer Research. Epub 2015 Aug 18
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 108
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Uy GL, Costa LJ, Hari PN, Zhang M-J, Huang J-X, Anderson KC, Bredeson CN, Callander NS, Cornell RF, Perez MAD, Dispenzieri A, Freytes CO, Gale RP, Garfall A, Gertz MA, Gibson J, Hamadani M, Lazarus HM, Kalaycio ME, Kamble RT, Kharfan-Dabaja MA, Krishnan AY, Kumar SK, Kyle RA, Landau HJ, Lee CH, Maiolino A, Marks DI, Mark TM, Munker R, Nishihori T, Olsson RF, Ramanathan M, Rodriguez TE, Saad AA, Savani BN, Schiller GJ, Schouten HC, Schriber JR, Scott E, Seo S, Sharma M, Ganguly S, Stadtmauer EA, Tay J, To LB, Vesole DH, Vogl DT, Wagner JL, Wirk B, Wood WA, D'Souza A
Contribution of chemotherapy mobilization to disease control in multiple myeloma treated with autologous hematopoietic cell transplantation
Bone Marrow Transplantation. doi:10.1038/ bmt.2015.190. Epub 2015 Aug 24
PMC4548821
Burke MJ, Verneris MR, Le Rademacher J, He W, Abdel-Azim H, Abraham AA, Auletta JJ, Ayas M, Brown VI, Cairo MS, Chan KW, Diaz Perez MA, Dvorak CC, Egeler RM, Eldjerou L, Frangoul H, Guilcher GMT, Hayashi RJ, Ibrahim A, Kasow KA, Leung WH, Olsson RF, Pulsipher MA, Shah N, Shah NN, Thiel E, Talano JA, Kitko CL
Transplant outcomes for children with T cell acute lymphoblastic leukemia in second remission: a report from the Center for International Blood and Marrow Transplant Research
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2154-2159. doi:10.1016/ j.bbmt.2015.08.023. Epub 2015 Aug 29
PMC4654112
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 109
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Ponce DM, Eapen M, Sparapani R, O'Brien TA, Chan KW, Chen J, Craddock J, Schultz KR, Wagner JE, Perales M-A, Barker JN
In vivo T cell depletion with myeloablative regimens on outcomes after cord blood transplantation for acute lymphoblastic leukemia in children
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2173-2179. doi:10.1016/ j.bbmt.2015.08.022. Epub 2015 Aug 29
PMC4639413
Pasquini MC, Zhang M-J, Medeiros BC, Armand P, Hu Z-H, Nishihori T, Aljurf MD, Akpek G, Cahn J-Y, Cairo MS, Cerny J, Copelan EA, Deol A, Freytes CO, Gale RP, Ganguly S, George B, Gupta V, Hale GA, Kamble RT, Klumpp TR, Lazarus HM, Luger SM, Liesveld JL, Litzow MR, Marks DI, Martino R, Norkin M, Olsson RF, Oran B, Pawarode A, Pulsipher MA, Ramanathan M, Reshef R, Saad AA, Saber W, Savani BN, Schouten HC, Ringdén O, Tallman MS, Uy GL, Wood WA Jr, Wirk B, Pérez WS, Batiwalla M, Weisdorf DJ
Hematopoietic cell transplantation outcomes in monosomal karyotype myeloid malignancies
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.08.024. Epub 2015 Aug 29
[PMC Journal – In Process]
Jindra PT, Conway SE, Ricklefs SM, Porcella SF, Anzick SL, Haagenson M, Wang T, Spellman S, Milford E, Kraft P, McDermott DH, Abdi R
Analysis of a genetic polymorphism in the costimulatory molecule TNFSF4 with HSCT outcomes
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.08.037. Epub 2015 Sep 5
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 110
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
D'Souza A, Dispenzieri A, Wirk B, Zhang M-J, Huang J, Gertz MA, Kyle RA, Kumar S, Comenzo RL, Gale RP, Lazarus HM, Savani BN, Cornell RF, Weiss BM, Vogl DT, Freytes CO, Scott EC, Landau HJ, Moreb JS, Costa LJ, Ramanathan M, Callander NS, Kamble RT, Olsson RF, Ganguly S, Nishihori T, Kindwall-Keller TL, Wood WA, Mark TM, Hari P
Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a Center for International Blood and Marrow Transplant Research study
Journal of Clinical Oncology. 2015 Nov 10; 33(32):3741-3749. doi:10.1200/JCO. 2015.62.4015. Epub 2015 Sep 14
[PMC Journal – In Process]
Ehrhardt MJ, Brazauskas R, He W, Rizzo JD, Shaw BE
Survival of patients who develop solid tumors following hematopoietic stem cell transplantation
Bone Marrow Transplantation. doi:10.1038/bmt. 2015.203. Epub 2015 Sep 14
PMC4570237
Klyuchnikov E, Bacher U, Woo Ahn K, Carreras J, Kröger NM, Hari PN, Ku GH, Ayala E, Chen AI, Chen Y-B, Cohen JB, Freytes CO, Gale RP, Kamble RT, Kharfan-Dabaja MA, Lazarus HM, Martino R, Mussetti A, Savani BN, Schouten HC, Usmani SZ, Wiernik PH, Wirk B, Smith SM, Sureda A, Hamadani M
Long-term survival outcomes of reduced-intensity allogeneic or autologous transplantation in relapsed grade 3 follicular lymphoma
Bone Marrow Transplantation. doi:10.1038/bmt. 2015.223. Epub 2015 Oct 5
[PMC Journal – In Process]
Kollman C, Spellman SR, Zhang M-J, Hassebroek A, Anasetti C, Antin JH, Champlin RE, Confer D, DiPersio JF, Fernandez-Viña M, Hartzman RJ, Horowtiz MM, Hurley CK, Karanes C, Maiers M, Mueller CR, Perales M-A, Setterholm M, Woolfrey AE, Yu N, Eapen M
The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy
Blood. doi:10.1182/blood-2015-08-663823. Epub 2015 Nov 2
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
Page | 111
SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID
Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papinicolou GA, Wingard JR, Young J-AH, Bennett CL
Risk factors and impact of non-Aspergillus mold infections following allogeneic HCT: a CIBMTR infection and immune reconstitution analysis
Bone Marrow Transplantation. doi:10.1038/bmt. 2015.263. Epub 2015 Nov 2
[PMC Journal – In Process]
Anthias C, Shaw BE, Kiefer DM, Liesveld JL, Yared J, Kamble RT, D'Souza A, Hematti P, Seftel MD, Norkin M, DeFilipp ZM, Kasow KA, Abidi MH, Savani BN, Shah NN, Anderlini P, Diaz MA, Malone AK, Halter JP, Lazarus HM, Logan BR, Switzer GE, Pulsipher MA, Confer DL, O'Donnell PV
Significant improvements in the practice patterns of adult related donor care in US transplant centers
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.11.008. Epub 2015 Nov 18
[PMC Journal – In Process]
Kanate AS, Mussetti A, Kharfan-Dabaja MA, Ahn KW, DiGilio A, Beitinjaneh A, Chhabra S, Fenske TS, Freytes C, Gale RP, Ganguly S, Hertzberg M, Klyuchnikov E, Lazarus HM, Olsson R, Perales M-A, Rezvani A, Riches M, Saad A, Slavin S, Smith SM, Sureda A, Yared J, Ciurea S, Armand P, Salit R, Bolaños-Meade J, Hamadani M
Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched unrelated donors
Blood. doi:10.1182/ blood-2015-09-671834. Epub 2015 Dec 15
[PMC Journal – In Process]
Seftel MD, Neuberg D, Zhang M-J, Wang HL, Ballen KK, Bergeron J, Couban S, Freytes CO, Hamadani M, Kharfan-Dabaja MA, Lazarus HM, Nishihori T, Paulson K, Saber W, Sallan SE, Soiffer R, Tallman MS, Woolfrey AE, DeAngelo DJ, Weisdorf DJ
Pediatric-inspired therapy compared to allografting for Philadelphia chromosome negative adult ALL in first complete remission
American Journal of Hematology. doi:10.1002/ajh.24285. Epub 2015 Dec 23
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS
Page | 112
APPENDIX D2: BMT CTN PUBLICATIONS
The following publications were generated by the BMT CTN, a component of the Clinical Trials Support Program, which conducts multi-institutional Phase II and III trials focused on HCT. The BMT CTN Data and Coordinating Center maintains continuity of operations and facilitates effective communications. The Data and Coordinating Center effort is a collaboration of the CIBMTR, NMDP/Be The Match, and the Emmes Corporation. For more information, see Section 2.3.1.
BMT CTN PUBLICATIONS Authors Title Citation PMCID
(2014 publication not previously reported) Levine JE, Braun TM, Harris AC, Holler E, Taylor A, Miller H, Magenau J, Weisdorf DJ, Ho VT, Bolaños-Meade J, Alousi AM, Ferrara JLM
A prognostic score for acute graft-versus-host disease based on biomarkers: a multicentre study
The Lancet Haematology. 2(1):e21-e29. doi:10.1016/S2352-3026(14)00035-0. Epub 2014 Dec 23
PMC4340092
MacMillan ML, Robin M, Harris AC, DeFor TE, Martin PJ, Alousi A, Ho VT, Bolaños-Meade J, Ferrara JLM, Jones R, Arora M, Blazar BR, Holtan SG, Jacobsohn D, Pasquini M, Socie G, Antin JH, Levine JE, Weisdorf DJ
A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):761-767. doi:10.1016/j.bbmt. 2015.01.001. Epub 2015 Jan 10
PMC4359643
Holtan SG, Verneris MR, Schultz KR, Newell LF, Meyers G, He F, DeFor TE, Vercellotti GM, Slungaard A, MacMillan ML, Cooley SA, Blazar BR, Panoskaltsis-Mortari A, Weisdorf DJ
Circulating angiogenic factors associated with response and survival in patients with acute graft-versus-host disease: results from Blood and Marrow Transplant Clinical Trials Network 0302 and 0802
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1029-1036. doi:10.1016/ j.bbmt.2015.02.018. Epub 2015 Mar 7
PMC4426052
CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS
Page | 113
BMT CTN PUBLICATIONS Authors Title Citation PMCID
Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ
Comparison of characteristics and outcomes of trial participants and nonparticipants: example of Blood and Marrow Transplant Clinical Trials Network 0201 trial
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1815-1822. doi:10.1016/ j.bbmt.2015.06.004. Epub 2015 Jun 11
PMC4568172
Anderlini P, Wu J, Gersten I, Ewell M, Tolar J, Antin JH, Adams R, Arai S, Eames G, Horwitz ME, McCarty J, Nakamura R, Pulsipher MA, Rowley S, Leifer E, Carter SL, DiFronzo NL, Horowitz MM, Confer D, Deeg HJ, Eapen M
Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study
Lancet Haematology. 2015 Sep 1; 2(9):e367-375. doi:10.1016/ S2352-3026(15)00147-7. Epub 2015 Sep 2
[PMC Journal – In Process]
Young JAH, Logan BR, Wu J, Wingard JR, Weisdorf DJ, Mudrick C, Knust K, Horowitz MM, Confer DL, Dubberke ER, Pergam SA, Marty FM, Strasfeld LM, Brown JM, Langston AA, Schuster MG, Kaul DR, Martin SI, Anasetti C
Infections following transplantation of bone marrow or peripheral-blood stem cells from unrelated donors
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.09.013. Epub 2015 Sep 23
[PMC Journal – In Process]
Wood WA, Le-Rademacher J, Syrjala KL, Jim H, Jacobsen PB, Knight JM, Abidi MH, Wingard JR, Majhail NS, Geller NL, Rizzo JD, Fei M, Wu J, Horowitz MM, Lee SJ
Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902)
Cancer. doi: 10.1002/ cncr.29717. Epub 2015 Oct 6
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS
Page | 114
BMT CTN PUBLICATIONS Authors Title Citation PMCID
Giralt S, Garderet L, Durie B, Cook G, Gahrton G, Bruno B, Hari P, Lokhorst H, McCarthy P, Krishnan A, Sonneveld P, Goldschmidt H, Jagannath S, Barlogie B, Mateos M, Gimsing P, Sezer O, Mikhael J, Jin L, Dimopoulos M, Mazumder A, Palumbo A, Abonour R, Anderson K, Attal M, Blade J, Bird J, Cavo M, Comenzo R, de la Rubia J, Einsele H, Sanz RG, Hillengass J, Holstein S, Johnsen H, Joshua D, Koehne G, Kumar S, Kyle R, Leleu X, Lonial S, Ludwig H, Nahi H, Nooka A, Orlowski R, Rajkumar V, Reiman A, Richardson P, Rivas E, San Miguel J, Turreson I, Usmani S, Vesole D, Bensinger W, Qazilbash M, Efebera Y, Mohty M, Gasparreto C, Gajewski J, LeMaistre C, Bredeson C, Moreau P, Pasquini M, Kroeger N, Stadtmauer E
American Society of Blood and Marrow Transplant, European Society of Blood and Marrow Transplantation, Blood and Marrow Transplant Clinical Trials Network and International Myeloma Working Group Consensus Conference on Salvage Hematopoietic Cell Transplantation in Patients with Relapsed Multiple Myeloma
Biology of Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2039-2051. doi: 10.1016/ j.bbmt.2015.09.016. Epub 2015 Sep 30
[PMC Journal – In Process]
Devine SM, Owzar K, Blum W, Mulkey F, Stone RM, Hsu JW, Champlin RE, Chen Y-B, Vij R, Slack J, Soiffer RJ, Larson RA, Shea TC, Hars V, Sibley AB, Geralt S, Carter S, Horowitz MM, Linker C, Alyea EP
Phase II study of allogeneic transplantation for older patients with acute myeloid leukemia in first complete remission using a reduced-intensity conditioning regimen: results from cancer and leukemia group B 100103 (alliance for clinical trials in oncology)/blood and marrow transplant clinical trial network 0502
Journal of Clinical Oncology. Epub 2015 Nov 2
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS
Page | 115
APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS
The following publications were generated by the Health Services Research program, through which the CIBMTR conducts research in health disparities, health policy, and system capacity issues involving HCT. For more information, see Section 2.4.
HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS
Authors Title Citation PMCID
Besse KL, Preussler JM, Murphy EA, Denzen EM, Lill MC, Chell JW, Senneka MK, Majhail NS, Williams EP
Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems
Journal of Oncology Practice. 11(2):e120-e130. doi:10.1200/ JOP.2014.000794. Epub 2015 Mar 1
PMC4371120
Majhail NS, Mau L-W, Chitphakdithai P, Payton T, Eckrich M, Joffe S, Lee SJ, LeMaistre CF, LeRademacher J, Loberiza F, Logan B, Parsons SK, Repaczki-Jones R, Robinett P, Rizzo JD, Murphy E, Denzen EM
National survey of hematopoietic cell transplantation center personnel, infrastructure, and models of care delivery
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1308-1314. doi:10.1016/ j.bbmt.2015.03.020. Epub 2015 Mar 31
PMC4466059
Clauser SB, Gayer C, Murphy E, Majhail NS, Baker KS
Patient centeredness and engagement in quality-of-care oncology research
Journal of Oncology Practice. 2015 May 1; 11(3):176-179. doi:10.1200/JOP.2015.003749. Epub 2015 Apr 7
[PMC Journal – In Process]
Preussler JM, Mau L-W, Majhail NS, Bevans M, Clancy E, Messner C, Parran L, Pederson KA, Ferguson SS, Walters W, Murphy EA, Denzen EM
Patient housing barriers to hematopoietic cell transplantation: results from a mixed-methods study of transplant center social workers
Supportive Care in Cancer. doi:10.1007/ s00520-015-2872-9. Epub 2015 Aug 15
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS
Page | 116
APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS
The following publications were generated by the Bioinformatics Research Program, which provides expertise in, and conducts research on, translational and operational bioinformatics. For more information, see Section 2.5.
BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID
Besse KL, Preussler JM, Murphy EA, Denzen EM, Lill MC, Chell JW, Senneka MK, Majhail NS, Williams EP
Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems
Journal of Oncology Practice. 11(2):e120-e130. doi:10.1200/ JOP.2014.000794. Epub 2015 Mar 1
PMC4371120
Slater N, Louzoun Y, Gragert L, Maiers M, Chatterjee A, Albrecht M
Power laws for heavy-tailed distributions: modeling allele and haplotype diversity for the National Marrow Donor Program
PLoS Computational Biology. 11(4):e1004204. doi:10.1371/journal.pcbi.1004204. Epub 2015 Apr 22
PMC4406525
Petz LD, Burnett JC, Li H, Li S, Tonai R, Bakalinskaya M, Shpall EJ, Armitage S, Kurtzberg J, Regan DM, Clark P, Querol S, Gutman JA, Spellman SR, Gragert L, Rossi JJ
Progress toward curing HIV infection with hematopoietic cell transplantation
Stem Cells and Cloning: Advances and Applications. 2015(8):109-116. doi:10.2147/SCCAA.S56050. Epub 2015 Jul 28
PMC4524463
Hollenbach JA, Saperstein A, Albrecht M, Vierra-Green C, Parham P, Norman PJ, Maiers M
Race, ethnicity and ancestry in unrelated transplant matching for the National Marrow Donor Program: a comparison of multiple forms of self-identification with genetics
PLoS One. doi:10.1371/journal.pone.0135960. Epub 2015 Aug 19
PMC4545604
CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS
Page | 117
BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID
Besse K, Maiers M, Confer D, Albrecht M
On modeling HLA-identical sibling match probability for alloHCT: estimating the need for an unrelated donor source
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.09.012. Epub 2015 Sep 5
N/A
Single RM, Strayer N, Thomson G, Paunic V, Albrecht M, Maiers M
Asymmetric linkage disequilibrium: tools for assessing multiallelic LD
Human Immunology. doi:10.1016/j.humimm.2015.09.001. Epub 2015 Sep 7
N/A
Mack SJ, Milius RP, Gifford BD, Sauter J, Hofmann J, Osoegawa K, Robinson J, Groeneweg M, Turenchalk GS, Adai A, Holcomb C, Rozemuller EH, Penning MT, Heuer ML, Wang C, Salit ML, Schmidt AH, Parham PR, Müller C, Hague T, Fischer G, Fernandez-Viña M, Hollenbach JA, Norman PJ, Maiers M
Minimum information for reporting next generation sequence genotyping (MIRING): guidelines for reporting HLA and KIR genotyping via next generation sequencing
Human Immunology. doi:10.1016/j.humimm.2015.09.011. Epub 2015 Sep 25
[PMC Journal – In Process]
Magalon J, Maiers M, Kurtzberg J, Navarrete C, Rubinstein P, Brown C, Schramm C, Larghero J, Katsahian S, Chabannon C, Picard C, Platz A, Schmidt A, Katz G
Banking or bankrupting: strategies for sustaining the economic future of public cord blood banks
PLoS One. 10(12):e0143440. Epub 2015 Dec 1
PMC4666404
CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS
Page | 118
BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID
Buck K, Wadsworth K, Setterholm M, Maiers M, Confer D, Hartzman R, Schmidt A, Yang SY, Dehn J
7/8 and 9/10 or better high resolution match rate for the Be The Match® Unrelated Donor Registry
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.12.012. Epub 2015 Dec 24
N/A
CIBMTR 2015 Annual Report APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS
Page | 119
APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS
The following publications were generated by the Statistical Methodology Research Program, which develops and evaluates the statistical models used in HCT. For more information, see Section 2.6.
STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS
Authors Title Citation PMCID
Keiding N, Andersen PK, Zhang MJ
Editorial: To the memory of John P. Klein
Lifetime Data Analysis. 2015 Apr 1; 21(2):157-159. doi:10.1007/ s10985-015-9320-5. Epub 2015 Feb 7
N/A
Wang T, He P, Ahn KW, Wang X, Ghosh S, Laud P
A re-formulation of generalized linear mixed models to fit family data in genetic association studies
Frontiers in Genetics. 6(120):1-10. doi:10.3389/ fgene.2015.00120. Epub 2015 Mar 31
PMC4379931
Eriksson F, Li J, Scheike T, Zhang M-J
The proportional odds cumulative incidence model for competing risks
Biometrics. 2015 Sep 1; 71(3):687-695. doi:10.1111/biom.12330. Epub 2015 May 26
PMC4608382
He P, Eriksson F, Scheike TH, Zhang M-J
A proportional hazards regression model for the subdistribution with covariates-adjusted censoring weight for competing risks data
Scandinavian Journal of Statistics, Theory and Applications. doi:10.1111/sjos.12167. Epub 2015 Jun 5
[PMC Journal – In Process]
Brazauskas R, Logan BR Observational studies: matching or regression?
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.12.005. Epub 2015 Dec 19
[PMC Journal – In Process]
CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS
Page | 120
APPENDIX D6: COORDINATING CENTER PUBLICATIONS
The following publications incorporated major contributions from the CIBMTR Coordinating Center and Scientific Directors.
COORDINATING CENTER PUBLICATIONS
Authors Title Citation PMCID
Majhail NS, Giralt S, Bonagura A, Crawford S, Farnia S, Omel JL, Pasquini M, Saber W, LeMaistre CF
Guidelines for defining and implementing standard episode of care for hematopoietic stem cell transplantation within the context of clinical trials
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):583-588. doi:10.1016/ j.bbmt.2014.12.030. Epub 2015 Jan 29
N/A
Sucheston-Campbell LE, Clay A, McCarthy PL, Zhu Q, Preus L, Pasquini M, Onel K, Hahn T
Identification and utilization of donor and recipient genetic variants to predict survival after HCT: are we ready for primetime?
Current Hematologic Malignancy Reports. 2015 Mar 1; 10(1):45-58. doi:10.1007/ s11899-014-0246-x. Epub 2015 Feb 21
PMC4352187
Gratwohl A, Pasquini MC, Aljurf M, Atsuta Y, Baldomero H, Foeken L, Gratwohl M, Bouzas LF, Confer D, Frauendorfer K, Gluckman E, Greinix H, Horowitz M, Iida M, Lipton J, Madrigal A, Mohty M, Noel L, Novitzky N, Nunez J, Oudshoorn M, Passweg J, van Rood J, Szer J, Blume K, Appelbaum F, Kodera Y, Niederwieser D
One million haemopoietic stem-cell transplants: a retrospective observational study
The Lancet Haematology. 2(3):e91-e100. doi:10.1016/S2352-3026(15)00028-9. Epub 2015 Mar 1
N/A
CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS
Page | 121
COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID
Shah N, Callander N, Ganguly S, Gul Z, Hamadani M, Costa L, Sengsayadeth S, Abidi M, Hari P, Mohty M, Chen Y-B, Koreth J, Landau H, Lazarus H, Leather H, Majhail N, Nath R, Osman K, Perales M-A, Schriber J, Shaughnessy P, Vesole D, Vij R, Wingard J, Giralt S, Savani BN
Hematopoietic stem cell transplantation for multiple myeloma: guidelines from the American Society for Blood and Marrow Transplantation
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1155-1166. doi:10.1016/j.bbmt. 2015.03.002. Epub 2015 Mar 11
[PMC Journal – In Process]
Eapen M Hematopoietic cell transplantation for acute leukemia: selecting donors
Haematologica. 2015 Apr 1; 100(4):414-415. doi:10.3324/haematol. 2015.124974. Epub 2015 Apr 1
PMC4380712
Khera N From evidence to clinical practice in blood and marrow transplantation
Blood Reviews. doi:10.1016/j.blre. 2015.04.001. Epub 2015 Apr 9
PMC4610823
Graff TM, Singavi AK, Schmidt W, Eastwood D, Drobyski WR, Horowitz M, Palmer J, Pasquini M, Rizzo DJ, Saber W, Hari P, Fenske TS
Safety of outpatient autologous hematopoietic cell transplantation for multiple myeloma and lymphoma
Bone Marrow Transplantation. 2015 Jul 1; 50(7):947-953. doi:10.1038/ bmt.2015.46. Epub 2015 Apr 13
PMC4490016
Martin PJ, Lee SJ, Przepiorka D, Horowitz MM, Koreth J, Vogelsang GB, Walker I, Carpenter PA, Griffith LM, Akpek G, Mohty M, Wolff D, Pavletic SZ, Cutler CS
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1343-1359. doi:10.1016/ j.bbmt.2015.05.004. Epub 2015 May 15
PMC4506719
CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS
Page | 122
COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID
Gale RP, Eapen M Who is the best alternative allotransplant donor?
Bone Marrow Transplantation. 2015 Jun 1; 50(S2):S40-S42. doi:10.1038/ bmt.2015.94. Epub 2015 Jun 4
PMC4520408
Renner R, Carlis J, Maiers M, Rizzo JD, O'Neill C, Horowitz M, Gee K, Confer D
Integration of hematopoietic cell transplantation outcomes data: data standards are not enough
Data Integration in the Life Sciences. 2015 Jul 8; 9162:139-146. doi:10.1007/978-3-319-21843-4_11
N/A
Booth GS, Gehrie EA, Jagasia MH, Shaw BE, Savani BN
When can you discard stem cells?
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2015.07.024. Epub 2015 Aug 4
N/A
Majhail NS, Farnia SH, Carpenter PA, Champlin RE, Crawford S, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Savani BN, Veys PA, Bredeson CN, Giralt SA, LeMaistre CF
Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American Society for Blood and Marrow Transplantation
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2015.07.032. Epub 2015 Aug 7
N/A
Deeg HJ, Bredeson C, Farnia S, Ballen K, Gupta V, Mesa RA, Popat U, Hari P, Saber W, Seftel M, Tamari R, Petersdorf EW
Hematopoietic cell transplantation as curative therapy for patients with myelofibrosis: long-term success in all age groups
Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Nov 1; 21(11):1883-1887. doi:10.1016/ j.bbmt.2015.09.005. Epub 2015 Sep 11
PMC4604067
CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS
Page | 123
COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID
Pagliuca S, Gérard L, Kulasekararaj A, Eapen M, Boutboul D, Martin H, Salvino MA, Knol-Bout C, Dufour C, Peffault de Latour R, Marsh J
Characteristics and outcomes of aplastic anemia in HIV patients: a brief report from the severe aplastic anemia working party of the European Society of Blood and Bone Marrow Transplantation
Bone Marrow Transplantation. doi:10.1038/ bmt.2015.252. Epub 2015 Oct 19
N/A
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 124
APPENDIX E: PRESENTATIONS
2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI
BMT CTN 0201
5 year results of BMT CTN 0201: unrelated donor bone marrow is associated with better psychological well-being and less burdensome chronic graft-versus-host disease symptoms than peripheral blood * Selected as a 2016 Highlight of ASH
Oral SJ Lee
BMT CTN 0601
A multicenter Phase II trial of unrelated donor reduced intensity bone marrow transplantation for children with severe sickle cell disease (SCURT): Results of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0601) Study
Oral S Shenoy
BMT CTN 0805
Multicenter US intergroup study of intensive chemotherapy plus dasatinib followed by allogeneic stem cell transplant in patients with Philadelphia chromosome positive acute lymphoblastic leukemia younger than 60 * Late Breaking Abstract
Oral F Ravandi
BMT CTN 0901
Results of a Phase III randomized, multi-center study of allogeneic stem cell transplantation after high vs reduced intensity conditioning in patients with myelodysplastic syndrome or acute myeloid leukemia * Late Breaking Abstract
Oral BL Scott
CK11-02 A prognostic system predictive of outcomes in persons undergoing allogeneic hematopoietic cell transplantation for myelodysplastic syndrome
Oral B Shaffer
CK14-01 Outcomes after umbilical cord blood transplantation for myelodysplastic syndromes: a Center for International Blood and Marrow Transplant Registry study
Poster A Gerds
GV12-01 Outcomes of grades II-IV acute graft-versus-host disease post allogeneic hematopoietic stem cell transplantation: How much progress was achieved?
Poster H Khoury
GV12-02
Upper gastrointestinal acute graft-versus-host disease adds minimal prognostic value when present in isolation or in addition to grade I or other grade II-defining GvHD manifestations
Oral S Nikiforow
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 125
2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI
HS13-01 The impact of pre-transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation
Oral A El-Jawahri
HS13-02 A study of predictors of clinical outcomes and healthcare utilization in children with sickle cell disease undergoing allogeneic hematopoietic cell transplantation
Oral S Arnold
IB11-01
Evaluation of the impact of non-inherited maternal antigens on the outcome of HLA mismatched unrelated donor hematopoietic stem cell transplantation for hematological malignancies on behalf of the ALWP of the EBMT and the CIBMTR
Poster J Pingel
IB12-03 Investigating effect of genetic admixture and donor / recipient genetic disparity on transplant outcomes Poster A Madbouly
LK13-04
Comparison of post allogeneic hematopoietic cell transplantation outcomes after matched related donor versus matched unrelated donor HCT in adults with acute lymphoblastic leukemia
Poster E Segal
LK14-02 Outcomes of allogeneic transplantation in patients aged ≤ 60 years with acute myeloid leukemia in second complete remission: a CIBMTR cohort analysis
Poster F Michelis
LK14-03 Autologous transplant, and not ATO alone, remains the preferred therapy for relapsed APL: a report from the CIBMTR, EBMT, and two specialized centers
Oral M Tallman
LY06-03
Allogeneic stem cell transplantation for relapsed / refractory follicular lymphoma: a joint study between the European Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research
Oral A Sureda
LY14-02
Reduced-intensity allogeneic hematopoietic cell transplantation provides durable progression-free survival in a subset of diffuse large B-cell lymphoma patients relapsing after autologous HCT
Oral T Fenske
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 126
2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI
LY15-01
Survival after T-cell replete haploidentical related donor transplant using post-transplant cyclophosphamide compared with matched unrelated donor transplant for lymphoid malignancies * Selected as a 2016 Highlight of ASH
Oral M Kharafan-Dabaja
MM13-02 Autologous hematopoietic cell transplantation in patients with high risk multiple myeloma: post-transplant responses do not translate to longer survival
Poster M Sharma
MM15-01 Post transplant therapy is more important than induction regimen choice in autologous hematopoietic cell transplantation recipients for multiple myeloma
Oral R Cornell
RCI BMT 09PLEX
A Phase II study evaluating the safety and efficacy of subcutaneous plerixafor for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells in recipients with hematologic malignancies
Oral S Devine
RT09-04 / IB09-06
Combined donor and recipient non-HLA genotypes show evidence of genome-wide association with transplant related mortality after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study)
Oral L Sucheston-Campbell
RT09-04 / IB09-06
Genome wide association study of overall and progression-free survival after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study)
Oral T Hahn
RT09-04c
Evidence for heterogeneous genetic associations with acute lymphoblastic leukemia by cytogenetics and sex in high risk patients treated with matched unrelated donor allogeneic blood or marrow transplant
Poster A Clay
SC11-06 Outcome of patients 65 years and older with myelodysplastic syndrome receiving allogeneic hematopoietic stem cell transplantation compared to patients 55-64 years of age
Oral E Atallah
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 127
2015 BMT TANDEM MEETINGS
Study Title Type Principal Investigator
Bio- informatics Metadata / BRIDG integration process Oral J Pollack
Bio- informatics Stem cell transplant interoperability using BRIDG Oral RP Milius
BMT CTN More infections with transplantation of bone marrow versus peripheral-blood stem cells from unrelated donors Oral J Young
BMT CTN 0302 / 0802
Prognostic impact of follistatin in acute graft-versus-host disease: results from BMT CTN 0302 and 0802 Oral S Holtan
CK13-02 Allogeneic hematopoietic cell transplantation for adult chronic myelomonocytic leukemia Oral H Duong
DS05-02b & RCI BMT
06-DON
Health-related quality of life among older adult related hematopoietic stem cells donors (>60 yrs.) is equivalent to or better than that of younger adult related donors (18-60 yrs.)
Oral G Switzer
DS05-02c / RCI BMT 06-DON
Acute toxicities of related adult donors compared to unrelated adult Oral M Pulsipher
DS09-04
The effect of race, socioeconomic status, and collection center size on bone marrow and peripheral blood stem cell donor experiences at National Marrow Donor Program collection centers
Oral M Pulsipher
HS13-03 Clinical outcomes among unrelated donor transplant recipients for acute myelogenous leukemia as a function of socioeconomic status and related transcriptome differences
Oral J Knight
HSR 13-01 Administrative claims data for cost analyses in hematopoietic cell transplantation: the good, the bad and the ugly Poster J Preussler
HSR 13-02 Patient and provider preferences for survivorship care plans for allogenic hematopoietic cell transplantation survivors: a qualitative study
Poster E Murphy
HSR 14-02 Hematopoietic cell transplantation multidisciplinary care teams: burnout, moral distress and career satisfaction Poster J Neumann
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 128
2015 BMT TANDEM MEETINGS
Study Title Type Principal Investigator
IB13-05
MHC class I chain-related gene A (MICA) donor-recipient mismatches and MICA-129 polymorphism in unrelated donor hematopoietic stem cell transplants for hematological malignancies: a CIBMTR study
Poster M Askar
IS13-01
Impact of race on graft-versus-host disease rates after HLA-matched sibling bone marrow or peripheral blood hematopoietic cell transplantation: Comparison of North American Caucasian versus Japanese populations
Oral J Kanda
LK13-03 Allogeneic transplant for acute biphenotypic leukemia: characteristics and outcome in the CIBMTR database Oral R Munker
LY12-01 Positive pre-allogeneic hematopoietic cell transplantation PET scan in patients with non-Hodgkin lymphoma predicts higher risk of relapse but has no impact on survival
Oral V Bachanova
LY13-02
Risk factors predicting outcomes of autologous hematopoietic cell transplantation in children, adolescents and young adults with relapsed / refractory classical Hodgkin lymphoma: a CIBMTR analysis
Oral P Satwani
2015 AMERICAN SOCIETY OF CLINICAL ONCOLOGY ANNUAL MEETING
Study Title Type Principal Investigator
LY13-03
Reduced intensity conditioning allograft as first transplant approach in relapsed / refractory grade III follicular lymphoma is associated with improved outcomes in long-term survivors
Oral E Klyuchnikov
BMT CTN 0704
Updated analysis of CALGB / ECOG / BMT CTN 100104: Lenalidomide vs. placebo maintenance therapy after single autologous stem cell transplant for multiple myeloma
Poster SA Holstein
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 129
2015 EUROPEAN GROUP FOR BLOOD AND MARROW TRANSPLANTATION (EBMT) ANNUAL MEETING
Study Title Type Principal Investigator
Bio-informatics
HML 1.0
Reporting NGS-based HLA & KIR genotyping using MIRING principles Oral RP Milius
DS05-02f / RCI BMT 06-DON
Health-related quality of life among pediatric hematopoietic stem cell donors Oral G Switzer
DS14-01 (US)
Significant improvements in the practice patterns of related donor care in US transplant centres Poster C Anthias
DS14-01 (EBMT)
JACIE accreditation significantly improves compliance with international recommendations for related donor care in EBMT transplant centres
Poster C Anthias
IB12-02b Uni-directional and bi-directional non-permissive HLA-DPB1 T cell epitope group mismatches have similar risk associations in 10/10 matched unrelated donor HCT
Oral K Fleischhauer
IB12-04b Effect of HLA-C allele matching in the context of patients HLA-C encoded KIR ligand grouping (C1 or C2) on outcomes of unrelated hematopoietic stem cell transplantation
Oral J Fischer
IB12-06
KIR B genotype in HLA-matched unrelated donor protects from relapse and improves progression-free survival after allogeneic transplantation for relapsed / refractory non-Hodgkin lymphoma (This presentation was withdrawn because the PI was unable to attend the meeting)
Poster V Bachanova
2015 EUROPEAN HEMATOLOGY ASSOCIATION CONGRESS
Study Title Type Principal Investigator
LY13-03 Reduced intensity conditioning allo transplantation is associated with superior long-term disease control in relapsed / refractory grade I/II follicular lymphoma
Oral E Klyuchnikov
BMT CTN 0102
Heavy light chain ratio normalization allows identification of electrophoretic non-complete response patients with improved outcomes: a long term follow up update for BMT CTN 0102 correlative study
Poster A D’Souza
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 130
2015 EUROPEAN IMMUNOGENETICS AND HISTOCOMPATIBILITY CONFERENCE
Study Title Type Principal Investigator
Bio-informatics Using SNPs to improve phasing of HLA haplotypes Oral V Paunic
Bio-informatics Impudigree: an imputation-based automated pedigree tool Oral J Freeman
Bio-informatics
Improved HLA-based race / ethnic classification using donor geography and census demographic data Poster L Gragert
Bio-informatics HLA diversity in the Ezer Mizion Registry Poster M Halagan
Bio-informatics
HLA class II six-locus DRB3/4/5~DRB1~DQA1~DQB1~DPA1~ DPB1 high resolution haplotype frequencies of the major US populations
Poster M Halagan
Bio-informatics
HLA allele and haplotype frequencies for Christian and Muslim Arab donors in Hadassah Registry Poster A Bishara
2015 INSTITUTE FOR HEALTHCARE ADVANCEMENT’S ANNUAL HEALTH LITERACY CONFERENCE
Study Title Type Principal Investigator
BMT CTN 1205
Easy-to-read informed consent forms for multi-center hematopoietic cell transplant clinical trials Poster H Moore
2015 INTERNATIONAL CONFERENCE ON LONG-TERM COMPLICATIONS OF TREATMENT OF CHILDREN AND ADOLESCENTS FOR CANCER
Study Title Type Principal Investigator
LE13-04 Survival of patients who develop solid tumors following hematopoietic stem cell transplantation Poster M Ehrhardt
CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS
Page | 131
2015 PEDIATRIC BLOOD AND MARROW TRANSPLANT CONSORTIUM ANNUAL MEETING
Study Title Type Principal Investigator
LE13-04 Survival of patients who develop solid tumors following hematopoietic stem cell transplantation Oral M Ehrhardt
2015 WORLD CONGRESS OF PSYCHO-ONCOLOGY (hosted by the International Psycho-Oncology Society and
American Psychosocial Oncology Society)
Study Title Type Principal Investigator
BMT CTN 0902
Cancer and treatment distress measurement over time in a multicenter cohort of hematopoietic cell transplantation recipients
Oral K Syrjala
BMT CTN 0902
Pre-transplant health-related quality of life factors as predictors of outcomes following hematopoietic cell transplantation
Oral J Knight
2015 WORLD MARROW DONOR ASSOCIATION MEETING
Study Title Type Principal Investigator
Bio-informatics
Key driver analysis of HLA diversity: analytically focused recruitment strategies for improving registry quality Poster A
Sivasankaran
Bio-informatics
Enhancing HML for electronic reporting of NGS-based HLA and KIR genotyping results Poster J Kathryn
CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Page | 132
APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
This study development and management process pertains to studies for which the CIBMTR provides data and statistical support. Data sets are also made available to investigators who have their own statistical resources. Final analyses and manuscripts resulting from these analyses are reviewed and approved by the CIBMTR prior to journal submission.
STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Planned Protocol pending. Proposals remain in this preliminary stage until the PI creates a draft protocol.
In Progress
Draft protocol received. When a PI submits a draft protocol, Coordinating Center staff review it.
Protocol development. During the development process, the Working Committee biostatisticians, Scientific Director, and Chairs refine the submission into a comprehensive study protocol. They add a table with a preliminary description of the proposed study population and present the draft protocol for discussion at a weekly Coordinating Center statistical meeting. When a protocol is approved, Coordinating Center personnel invite Working Committee members to participate in a Writing Committee.
Sample typing. If applicable, the PIs perform laboratory tests (e.g., genotyping) on samples from the CIBMTR Research Repository. The testing data will be used in the analysis to determine any correlation with clinical outcome.
Supplemental forms / data collection. Most studies use routinely-collected data. If necessary, Coordinating Center staff, in collaboration with the PI and relevant Working Committee Chairs, develop a supplemental form, which is approved prior to soliciting centers for additional data. Use of supplemental data (e.g., data not collected on standard CIBMTR data collection forms) is discouraged unless it will result in a particularly meaningful publication and/or external funding can support the extra burden placed on transplant centers and supplement forms reimbursement costs.
CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Page | 133
STUDY DEVELOPMENT AND MANAGEMENT PROCESS
In Progress (continued)
Data file preparation. The objective of data file preparation is to create a file of eligible subjects who are consecutively treated at participating centers with adequate follow-up, with minimal missing data fields, and in large enough numbers to give the analysis sufficient statistical power to meet the stated study objectives. This process involves a series of steps by the MS-level statistician, sometimes working with the Clinical Research Coordinator, to ensure data quality: • Verifying selection criteria • Including and excluding patients so that the investigators can determine
whether the final study population is representative of the target population • Assessing follow-up • Determining the extent and nature of missing values and their potential effect
on the study • Resolving and reconciling data discrepancies / outliers by examining data
collection forms and communicating with centers and the PI
Analysis in progress. Analysis proceeds in several phases. The first generally includes a detailed description of the patient population and univariate and multivariate analyses of study endpoints. Coordinating Center personnel present these data for discussion at a weekly Coordinating Center statistical meeting and then distribute them to Writing Committee members for suggestions and comments. The PI works with Coordinating Center staff in an iterative process to review comments from the Writing Committee. The process repeats until final analysis, which serves as the basis for the manuscript.
Ongoing. A study in ongoing status is long-term and often involves multiple grants and/or renewals outside of the CIBMTR in order to reach its objectives. The study has its own Statistical Director for analysis, but it requires data from the CIBMTR, usually each year.
Preliminary Results
Manuscript preparation. The PI is primarily responsible for manuscript preparation and is expected to prepare a draft manuscript within 30 days of receiving analysis results. The Working Committee leadership review and revise the document, ensuring that the description and interpretation of the statistical analyses are accurate and contribute to the fundamental message of the manuscript. The Coordinating Center then distributes the approved first draft to the Writing Committee and solicits feedback. The PI incorporates comments from the Writing Committee and creates a revised draft, which is reviewed in an iterative process by the Writing Committee until reaching a reasonable consensus on a final manuscript.
CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Page | 134
STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Preliminary Results
(continued)
Submitted. The Coordinating Center staff is responsible for submitting the manuscript and corresponding with the chosen journal. The Working Committee Scientific Director often serves as corresponding author, and the study statistician forwards all editor and reviewer comments to the PI and Statistical Director. The PI is expected to prepare a response, working with Coordinating Center staff who provide additional analyses of data, as needed. Coordinating Center personnel communicate with the journal, including re-submissions, in most cases.
In press. A publication is in press when it has been approved but does not yet have a citation.
Completed Published. A manuscript is considered published when a citation is available, including a PMCID number, if applicable. For a list of 2015 publications, see Appendix D.
CIBMTR 2015 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS
Page | 135
APPENDIX G: CLINICAL TRIALS
Through the Clinical Trials Support Program, the Coordinating Center supports clinical trial planning and interpretation; data collection, including long-term follow-up data; and real-time accrual assessment. See Section 2.3 for more information.
APPENDIX G1: BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT
The BMT CTN (Section 2.3.1) is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation. A status of BMT CTN trials open for enrollment is included in this appendix and is available on the BMT CTN website. For additional information on completed Network trials, see the annual progress report on the BMT CTN website.
BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT
Protocol Number Title Status to Date
BMT CTN 1101
Phase III study comparing HLA-haploidentical related donor bone marrow versus double umbilical cord blood with reduced-intensity conditioning for patients with hematologic malignancies
• Opened to accrual Jun 2012 • 219 of 410 patients enrolled • Anticipated accrual completion in
2017
BMT CTN 1102
A multi-center biologic assignment trial comparing reduced intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate-2 and high risk myelodysplastic syndrome
• Opened to accrual Dec 2013 • 147 of 338 patients enrolled • Anticipated accrual completion in
early 2018
BMT CTN 1202
Prospective multi-center cohort for the evaluation of biomarkers predicting risk of complications and mortality following allogeneic HCT
• Opened to accrual Jun 2013 • 1,708 of 1,500 patients enrolled;
accrual target increased to enhance pediatric and minority patient accrual
• Anticipated accrual completion in Jun 2016
BMT CTN 1203
A multi-center Phase II trial randomizing novel approaches for graft-versus-host disease prevention compared to contemporary controls
• Opened to accrual Sep 2014 • 209 of 270 patients enrolled • Anticipated accrual completion in
April 2016
CIBMTR 2015 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS
Page | 136
BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Number Title Status to Date
BMT CTN 1205
Easy-to-read informed consent (ETRIC) for hematopoietic cell transplantation clinical trials
• Opened to accrual Nov 2013 • 138 of 160 patients enrolled,
although accrual target may be increased
• Anticipated accrual completion in 2016
BMT CTN 1301
Phase III trial of calcineurin inhibitor-free interventions for prevention of graft-versus host-disease
• Opened to accrual Aug 2015 • 9 of 345 patients enrolled • Anticipated accrual completion in
early 2019
BMT CTN 1302
Phase II, double-blind Placebo controlled trial of maintenance ixazomib after allogeneic HCT for high risk multiple myeloma
• Opened to accrual Aug 2015 • 6 of 138 patients enrolled • Anticipated accrual completion in
June 2018
BMT CTN 1304
(DFCI 10-106)
A randomized Phase III study comparing conventional dose treatment using a combination of lenalidomide, bortezomib, and dexamethasone (RVD) to high-dose treatment with peripheral stem cell transplant in the initial management of myeloma in patients up to 65 years of age
• Opened to accrual by BMT CTN Nov 2013
• 514 of 660 patients enrolled • Anticipated accrual completion in
2016
BMT CTN 1505
(UT RECRUIT)
Randomized minority patient recruitment intervention trial
• Opened to accrual by BMT CTN May 2015
• Patient enrollment: N/A, BMT CTN centers randomized to intervention and control arms; center staff are research subjects
• Anticipated accrual completion in May 2017
CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS
Page | 137
APPENDIX G2: RCI BMT CLINICAL TRIALS
The RCI BMT (Section 2.3.2) provides researchers in the field of HCT with infrastructure and expertise in HCT clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting into such groups as the BMT CTN or the national cancer cooperative groups. A status of its projects is included in this appendix.
RCI BMT CLINICAL TRIALS Protocol # Title Status To Date
Astellas
Unrelated donor sample collection for donors whose recipient is enrolled in a randomized, double-blind, placebo-controlled, Phase III trial to evaluate the protective efficacy and safety of a therapeutic vaccine, ASP0113, in cytomegalovirus-seropositive recipients undergoing allogeneic hematopoietic cell transplant
• Donor sample collection only • Opened to accrual Apr 2014 • Two donors enrolled • RCI BMT closed study Dec 2014
12-MOXE
Study of the anti-CD22 recombinant immunotoxin moxetumomab pasudotox (CAT-8015, HA 22) in children with B-lineage acute lymphoblastic leukemia and minimal residual disease prior to allogeneic hematopoietic cell transplantation
• Opened to accrual May 2015 • One patient enrolled • Study closed Sep 2015 due to
pharmaceutical company decision
09-SQOL Pilot study to assess the feasibility of collecting quality of life data in collaboration with the Stem Cell Therapeutic Outcomes Database
• Closed to accrual Sep 2013 • 301 adults and 89 pediatric
recipients enrolled • Final subject follow-up expected
by end of 2014 • Data analysis in process
11-TREO Multi-center study evaluating treosulfan, fludarabine, and low-dose TBI in children with AML / MDS undergoing allogeneic HCT
• Closed to accrual April 2014, about one year earlier than expected
• 40 of 40 targeted enrollment • Follow-up continues • Abstract submitted to 2016
EBMT Annual Meeting
COG-KIR A multi-center study examining donor NK-cell receptors and patient outcomes
• COG closed accrual May 2014 • URD sample management • 609 of 1,200 samples collected • 145 out of 400 targeted
enrollment
CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS
Page | 138
RCI BMT CLINICAL TRIALS Protocol # Title Status To Date
DS05-02, 06-DON
RDSafe: A multi-institutional study of hematopoietic stem cell donor safety and quality of life
• Closed to accrual July 2014 • 1,812 donors enrolled • Follow up assessments
completed July 2015 • Abstract presented at 2015 BMT
Tandem Meetings • Data review and analysis as well
as manuscript planning in progress
PBMTC ONC 1001 / 09-
MRD
A multi-center study to determine the role of minimal residual disease testing before and after HCT for pediatric acute myeloid leukemia
• Closed to accrual Oct 2014 • 150 total enrollment • Follow-up continues
09-PLEX
A phase II study evaluating the safety and efficacy of intravenous plerixafor for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells in recipients with hematological malignancies
• Closed to accrual Dec 2014 • Follow-up completed Feb 2016 • Oral abstract presented at 2015
ASH Annual Meeting • Data review and analysis as well
as manuscript planning in process
rHuG-CSF
Long-term follow-up study evaluating hematologic and non-hematologic cancers, thrombotic events, and autoimmune disorders in unrelated donors undergoing bone marrow harvest versus peripheral blood stem cell mobilization with recombinant human granulocyte colony-stimulating factor
• Closed to accrual Oct 2015 • 21,569 total enrollment • Follow-up continues through
2020
PBSC Filgrastim-mobilized peripheral blood stem cells for allogeneic transplantation with unrelated donors
• Opened to accrual Apr 1996 • Just under 25,000 URDs enrolled • Will close to accrual upon FDA
license; another protocol will open for all unlicensed product
10-CBA
A multi-center access and distribution protocol for unlicensed cryopreserved cord blood units for transplantation in pediatric and adult patients with hematologic malignancies and other indications
• Opened to accrual Oct 2010 • 2,757 enrolled • Open indefinitely to allow
distribution and access to unlicensed cord units
10 CMS-MDS-1
Assessment of allogeneic HCT in Medicare beneficiaries with MDS and related disorders
• Opened to accrual Dec 2010 • 1,294 patients enrolled
CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS
Page | 139
RCI BMT CLINICAL TRIALS Protocol # Title Status To Date
KIR-DS A multi-center study looking at the selection of a favorable KIR donor
• Opened to accrual Jun 2011 • URD sample management • 1,942 samples facilitated • Amendment in process to alter
donor sample logistics, which will eliminated CIBMTR staff involvement
Statin Impact of donor statin use on graft-versus-host disease after unrelated donor HCT; URD data collection
• Opened to accrual Oct 2011 • 5,569 of 7,000 targeted donors
enrolled
BMT CTN 1102-QOL
A study comparing reduced intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate-2 and high risk myelodysplastic syndrome
• Survey Research Group performing QOL assessments
• 210 total assessments completed
13-TLEC Prospective non-therapeutic study, assessing the long-term toxicity of HCT for childhood leukemia
• Opened to accrual Mar 2015 • 53 of 340 patients enrolled
13-SCP
A randomized study to evaluate the impact of survivorship care planning on cancer survivors self-management and adherence to care recommendations and utilization of follow-up care
• In collaboration with Health Services Research Program
• Opened to accrual Apr 2015 • 181 of 495 patients enrolled
BMT CTN 1102-
Ancillary CEA study
A cost effectiveness ancillary study to the parent study 1102 above
• Collaborating with Fred Hutchinson Cancer Research center to perform Cost Effectiveness Analysis (CEA) study
• Survey Research Group to perform CEA survey collection
• First subject contacted in Oct 2015
HPD High priority donor project supporting Be The Match Operations
• First team meeting held in June 2015
• First subjects contacts by the Survey Research Group in Sep 2015
15-MMUD Study of transplantation of HLA-mismatched unrelated donor bone marrow for patients with hematologic malignancies
• Protocol team established Oct 2015
• Protocol development in process
CIBMTR 2015 Annual Report APPENDIX H: FORMS SUBMISSION PROCESS
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APPENDIX H: FORMS SUBMISSION PROCESS
• Center submits CRID Assignment Form (Form 2804), and CRID is generated • Indication for CRID Assignment Form (Form 2814) is added to Forms Due list • Center completes Indication Form and reports indication as HCT • Pre-TED (Form 2400) is added to Forms Due list • Center completes and submits Pre-TED • Pre-TED data are processed through the selection algorithm resulting in CRF or TED track
o If autologous recipient declines consent for research, stop here. Otherwise, follow the appropriate track below
CRF Track TED Track
1 Forms 2004, 2005, and 2006 are added, depending on donor type and if the donor has been used for a prior transplant.*
Forms 2004, 2005, and 2006 are added, depending on donor type, consent for sample repository, and if the donor has been used for a prior transplant.*
2 Baseline form 2000, disease specific inserts, and Follow-up Forms are added to Forms Due list.
Post-TED Follow-up Form 2450 is added to Forms Due list.
3 Center completes Baseline form after infusion. Center completes designated Post-TED Forms at appropriate time points.
4 Center completes designated CRF Follow-up Forms at appropriate time points.
Is recipient alive? If yes, go to Step 5. If no, report the death on the follow-up form, and go to Reporting Recipient Death.
5 Is recipient alive? If yes, go to Step 6. If no, report the death on the follow-up form, and go to Reporting Recipient Death.
Did recipient have a subsequent transplant? If yes, go to Step 6. If no, continue reporting at next time point (Step 3).
6 Did recipient have subsequent transplant? If yes, go to Step 7. If no, continue reporting at next time point (Step 4).
Subsequent transplant is reported on the next available follow-up form.
7 When the form reporting the subsequent transplant is in complete status, future forms for the prior transplant will be automatically deleted from FormsNet.
When the form reporting the subsequent transplant is in complete status, future forms for the prior transplant will be automatically deleted from FormsNet.
8 Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant.
Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant.
Reporting Recipient Death
Death Form 2900 is completed to report the recipient’s death.**
The recipient’s death is reported on the Post TED. A 2900 Death Form should not be completed for patients on the TED track.
* For more details regarding when Forms 2004, 2005, and 2006 are required, see “How Forms Come Due (2004, 2005, and 2006)”.
**Complete Death Form 2900 even if autopsy is pending. Another death form will be requested to confirm cause of death if autopsy was pending.
CIBMTR 2015 Annual Report APPENDIX I: WEBSITES
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APPENDIX I: WEBSITES
Throughout this report, electronic links to webpages and documents are provided. If you are unable to access items using the links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). URLs for the websites mentioned in this report are provided here.
Name URL
Be The Match bethematch.org Be The Match Clinical bethematchclinical.org BMT CTN bmtctn.net CIBMTR cibmtr.org CIBMTR Collaborate collaborate.cibmtr.org CIBMTR Portal portal.cibmtr.org HRSA Blood Cell Transplant bloodcell.transplant.hrsa.gov
CIBMTR 2015 Annual Report APPENDIX J: GLOSSARY
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APPENDIX J: GLOSSARY
Abbreviation/ Acronym Meaning
AGNIS A Growable Network Information System alloHCT allogeneic hematopoietic cell transplantation AML acute myeloid (myelogenous) leukemia ASBMT American Society of Blood and Marrow Transplantation ASH American Society of Hematology autoHCT autologous hematopoietic cell transplantation BMT bone marrow transplant BMT CTN Blood and Marrow Transplant Clinical Trials Network BRIDG Biomedical Research Integrated Domain Group CALGB Cancer and Leukemia Group B (member Alliance for Clinical Trials in Oncology) CED Coverage with Evidence Determination CIBMTR Center for International Blood and Marrow Transplant Research CME continuing medical education CMS Centers for Medicare and Medicaid Services COG Children’s Oncology Group CPI Continuous Process Improvement CRF Comprehensive Report Form CRID CIBMTR Recipient Identification Number DBtC Data Back to Centers application DFCI Dana Farber Cancer Institute DISCO Data and Information for Statistical Center Operations DRI Disease Risk Index EBMT European Group for Blood and Marrow Transplantation eDBtC enhanced Data Back to Centers application FACT Foundation for the Accreditation of Cellular Therapy FDA Food and Drug Administration HCT hematopoietic cell transplantation HIPAA Health Insurance Portability and Accountability Act HIV human immunodeficiency virus HLA human leukocyte antigen HRSA Health Resources and Services Administration IND Investigational new drug IRB Institutional Review Board IT Information Technology KIR killer-cell immunoglobulin-like receptors MCW Medical College of Wisconsin MDS myelodysplastic syndrome MED-A, MED-B Minimum Essential Data forms N/A not applicable NCI National Cancer Institute
CIBMTR 2015 Annual Report APPENDIX J: GLOSSARY
Page | 143
Abbreviation/ Acronym Meaning
NK natural killer (cell) NHLBI National Heart, Lung, and Blood Institute NIAID National Institute of Allergy and Infectious Disease NIH National Institutes of Health NMDP National Marrow Donor Program PBMTC Pediatric Blood and Marrow Transplant Consortium PBSC peripheral blood stem cell PI principal investigator PMCID PubMed Central unique identifier QOL quality of life RCI BMT Resource for Clinical Investigations in Blood and Marrow Transplant RDSafe Related Donor Safety Study RITN Radiation Injury Treatment Network SCTOD Stem Cell Therapeutic Outcomes Database TBD to be determined TED Transplant Essential Data URD unrelated donor US United States vs versus
Milwaukee CampusMedical College of Wisconsin 9200 W. Wisconsin Ave., Suite C5500Milwaukee, WI 53226 USA(414) 805-0700cibmtr.org
Minneapolis CampusNational Marrow Donor Program/ Be The Match 500 N 5th StreetMinneapolis, MN 55401 USA(612) 627-5800cibmtr.org
© 2016 The Medical College of Wisconsin, Inc. and the National Marrow Donor Program
CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and Medical College of Wisconsin
The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID).