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ANNUAL REPORT

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MissionThe Aplastic Anemia & MDS International Foundation (AA&MDSIF) is the world’s leading nonpro�t health organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and related bone marrow failure diseases. AA&MDSIF provides answers, support, and hope to thousands of patients and their families around the world.

VisionWe are a patient-focused, patient-centered organization, serving patients and families throughout the three phases of bone marrow failure diseases:

• the life changing phase of diagnosis

• the life threatening phase of treatment

• the life-long phase of living with a chronic disease

Values• accountability and commitment to the good of patients, caregivers and

families dealing with bone marrow failure diseases

• respect for the worth and dignity of all individuals

• dedication to inclusiveness and diversity

• commitment to transparency, integrity, and honesty

• responsible stewardship of resources

AA&MDSIF received Charity Navigator’s 4-Star rating for the ninth consecutive year, achieved by only the top 1% of the charities in the United States. The Guidestar Gold Level designation is the highest rating a nonpro�t organization can receive for mission, governance, and transparency to donors.

REVENUE

EXPENSES

UnrestrictedIndividualDonations

Unrestricted Workplace Giving from Individuals

UnrestrictedDonationsfromCorporations,NonprofitOrganizationsandCommunityFoundations

Grant&DonationsRestrictedforMedical Symposiums & Professional Education

Grant&DonationsRestrictedforResearch&Scholarship

Grants&DonationsRestrictedforClinicalResearchConsortium

Grants&DonationsRestrictedforPatientProgramsandConferences

Miscellaneous & Interest

2013 FINANCIAL HIGHLIGHTS

PatientEducation&Support

ProfessionalEducation

MedicalResearch

ClinicalResearchConsortium

Advocacy

Awareness

Scholarship

Management/General

Fundraising

36%

21%

7% 6%

1%

1%

12%

16%

33%

7%

16%

29%

7%6%

1%

1%

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Dear Friends,

Annual reports are often about programs, numbers, dollars, facts, and �gures. That’s �ne, but organizations like ours are about people. This year, our Annual Report tells you about our programs and services through the eyes and words of the people we’ve served, helped, or supported as well as the people who helped us.

The stories are examples –big and small—of what we do, how we serve, and what you have helped make possible through your con�dence in us and your partnership in our work.

Thank you for all you’ve helped us to accomplish in 2013. With your continued support, we plan to do even more in the years ahead.

Stay well,

John M. Huber Kevin Lyons-Tarr Executive Director Chairman, Board of Directors

A MESSAGE FROM THE EXECUTIVE DIRECTOR AND CHAIRMAN

OfficersChairman, Kevin Lyons-Tarr

Vice Chairman, Harsha Murthy

Secretary, Stephen King

Treasurer, Tony San�lippo

Executive Director, John Huber

Deborah Zi� Cook

Bart Fisher

Stephanie Dillon Hamm

Neil Horikoshi

Melanie Marquez

Judy Paulette

William Shulevitz

John Huber, Executive Director

Stephanie Chisolm, Senior Director of Programs and Services

Benita Marcus, Senior Director of Information/Management

Ellen J. Salkeld, Senior Director of Research and Health Professional Programs

Pamela Spears, Senior Director of Resource Development and Marketing

Catrell Harris, Director of Patient Education

Alice Houk, Director of Health Professional Programs

Robert Burleson, Publications Manager

Ramona Crawford, Grants and Industry Relations O�cer

Martha Crews, Community Development O�cer

Desiree Ruiters, Database Manager

Nila Vehar, Annual Campaign O�cer

Teresa A. Witt, Major Gift O�cer

Leigh Clark, Patient Educator

Mike Breuer, Development Support

Angie Onofre, Program Support

Elise Harper, Administrative Support

Paul Stehlin, Program Support - Web

AA&MDSIF Staff

Board Members

Every e�ort has been made to accurately acknowledge all contributors who gave $500 or more cumulatively between January 1, 2013 and December 31, 2013. If you would like to report an error or omission, please contact Pamela Spears at (301) 279-7202, x 122 or email spears@aamds.org. We apologize for any inadvertent errors and thank each of you for your generosity.

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AA&MDSIF LEADERSHIP

Pamela Becker, MD, PhD University of Washington School of Medicine

Monica Bessler, MD, PhD Children’s Hospital of Philadelphia

Carlos M. deCastro, III, MD Duke University Medical Center

H. Joachim Deeg, MD Fred Hutchinson Cancer Research Center

Amy E DeZern, MD, MHS Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Benjamin L. Ebert, MD, PhD Dana-Farber/Harvard Cancer Center

Guillermo Garcia-Manero, MD MD Anderson Center

Aristoteles Giagounidis, MD, PhD Marien hospital Dusseldorf, Germany

Steven Gore, MD Yale School of Medicine

Timothy Graubert, MD MGH Cancer Center

Alan List, MD H. Lee Mo�tt Cancer Center & Research Institute

Jaroslaw Maciejewski, MD, PhD Cleveland Clinic Taussig Cancer Center

David Margolis, MD Medical College of Wisconsin

Judith Marsh, MD Kings College Hospital

Stephen Nimer, MD Sylvester Comprehensive Cancer Center, University of Miami

Olatoyosi Odenike, MD University of Chicago

Ronald Paquette, MD UCLA Medical Center

Gail Roboz, MD Weill Medical College of Cornell University

Valeria Santini, MD University of Florence

B. Douglas Smith, MD Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Neal Young, MD National Institutes of Health

David J. Araten, MDEyal Attar, MD Maria Baer, MDRobin BrannonSusan Carson, RN, MSN, CPNPKenneth Cooke, MDAmanda FoisyIlene Galinsky, MSN, APRN-BCNorma GoodSteven Gore, MDLisa GruberEric HodiesPaige Jamieson Joan Latsko, MSN, CRNP, OCN, AOCNPRachael OttoLynda PayneOlga (Nunez) Rios RN, BSNLarry SaugerIsabel Schuermeyer, MDDavid Steensma, MDValerie SunJim ThomasMelissa TigheBarbara Weinstein, RN, BSN

Patient Education CouncilChair, David Margolis, MD

Members

Medical Advisory BoardChair Richard Stone, MD Co-Chair Mikkael Sekkeres, MD, MS Dana-Farber Cancer Center Cleveland Clinic Taussig Cancer Center

Members

Program PartnersNational Heart, Lung, and Blood Institute of the National Institutes of Health

O�ce of Rare Diseases of the National Institutes of Health

National Organization for Rare Diseases

PNH Research and Support Foundation

Continuing Education Credentialing InstitutionsAlabama State Nurses Association

American Academy of Nurse Practitioners

Cleveland Clinic Center for Continuing Education

Oncology Nursing Society

University of Nebraska

National Association of Social Workers

Association of Oncology Social Workers

Educational PartnersHealio.com

MPN Research Foundation

Primary Care Education

University of Alabama Birmingham

MD Anderson Cancer Center

University of Maryland

Weill Cornell Medical College of Cornell University

Medical College of Wisconsin

Cleveland Clinic

Pennsylvania Presbyterian Medical Center

University of Michigan Medical Center

North Carolina Central University

Robert H. Lurie Comprehensive Cancer Center

Rush University Medical Center

University of Utah

Sidney Kimmel Comprehensive Cancer Center at John Hopkins

Sylvester Comprehensive Cancer Center

Children’s Hospital of Wisconsin

University of California, San Diego

NYU School of Medicine

Harvard Dana-Farber Cancer Center

MDS Clinical Research Consortium PartnersCleveland Clinic Taussig Cancer Institute

Dana-Farber Cancer Institute

MD Anderson Cancer Center

H. Lee Mo�tt Cancer Center and Research Institute

Weill Medical College of Cornell University

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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DONOR HONOR ROLL

AA&MDSIF 2013 Honor Roll of Donors

Our thanks to those individuals, corporations, foundations, and organizations making contributions to fund AA&MDSIF’s programs and services during 2013. We honor all of our generous supporters, with additional thanks here to those who have donated $500 or more during 2013.

CHAIRMAN’S CIRCLE ($100,000+)Edward P. Evans Foundation

VISIONARIES ($50,000 - $99,999)

INNOVATORS ($25,000 - $49,999)Shizue Spielberg

CHAMPIONS ($10,000 - $24,999)Craig Dunlap Gary Gibson and Timothy HenryJames and Shirley O’BrienMia Hamm FoundationRhona’s Place FoundationWilliam and Marion Shulevitz PATRONS ($5,000 - $9,999)Kathi ColbyCongregation Agudath AchimMarta and Miguel GonzalezMike HenleyHerman Goldman FoundationGunther and Frances HeussmanNational Marrow Donor ProgramOld World IndustriesJe� SamuelsCrawford SandefurFred and Nancy SchumacherJohn SchwartzLesley SigallPeter and Colleen Skewes-Cox Jr.Matthew SmithCarol StewartThe Henry and Marilyn Taub FoundationStephanie Vendig

BENEFACTORS ($2,500 - $4,999) Bonnie BargstedtFred DamavandiEastman School of MusicCarl and Florence GolubKohl’sAlan LarsonKevin and Jenny Lyons-TarrSusan Morse-PoynterRon NicholsDavid PrimmerReep Family FoundationDouglas and Linda RobinsonThe Charles and Marion Weber FoundationValero Services, Inc.

LEADERS ($1,500- $2,499)Mima BairdBank of America Matching Gifts ProgramClyde BianchiBiogen-IdecDr. Morton and Mrs. Tommie BosniakDeborah and John CookFrank DamavandiMose DunningTammy Lee DuntonScott and Dawn FaurieMark FieldingEdward and Shelley GordonJohn and Mary Jane Gri�thDean and Jo He�el�ngerIntuit FoundationDr. Abraham and Kimberly KaderKass Family FoundationChristine and David LimbertMetzger Law GroupJohn MinelliTrisha MinorDr. Lisa MinterDoris MorelandMark and Luanne MosherChris PettyDeborah RathkeAnthony and Briget San�lippoDr. Mark and Lori SchneiderColleen and Elton ShuknechtJohn and Gina SteinerThe University of TexasAlex and Elyse VaysbergAllen and Susan WeaverGloria and Paul Winter

PARTNERS ($1,000- $1,499)AA & MDS Association of CanadaTamara and Peter AllenAllentown Inc.AnonymousBabak AzizzadehBeatrice and Leon Friedman Charitable FoundationDavid BiroAnn Bortscheller and Bill EliopoulosChecking for CharityGrace and Edmond ChengMr. and Mrs. Mark CoeDouglas and Maureen CohnJonathan Cohn, MDMichael Coiro, Sr.

Conserve, Inc.John and Anne CurlisJohn and Lili DamavandiElden and Sandra DellaninaDavid DerberRita and Johan Du RandSusan EkholmBart and Margaret FisherMary GeorgatsosSpencer and Penelope GreggRalph and Sally HamakerBrent and Maureen HansonDale HarrisHEB Supermarkets Inc.Mr. and Mrs. Gail HendricksNeil and Allyn HorikoshiCurtis and Sally JacksonJohnson & Johnson, Inc.Donald and Susan JosephsonSusan KassPhilip KenneyStephen KingRita KunofskyCindy LewallenSteve LewineJennifer LipseyVivian and Raymond LockeJohan and Rondi LotterThe Honorable William and Mary Ellen MaddenMargaret McCarthyMary Meeusen, RNNik MendrygalSusan NasonRobert and Sandra OglesbyJean OpatrnyNancy OrdwayDaryl and Ti�anie PappThomas ReepLarry and Michelle SaugerJudy SchnurmanPhyllis ScroggieDean and Carrie SharpJenelle and Rod SnowMeryl and Robert SobelChristina StileStoneturn Group LLPShirley SutherlandThe Shadden Group at Morgan StanleyMary Elaine and Javier ValenzuelaMargaret and Jon Wake�eldJames WalkerJessica WinchenbachEdward Winn

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INVESTORS ($500 - $999) Janet AarnioAmerican Power CorporationTim Andrews and Valerie CummingsAnonymous (6)Barbara Banks and David RootsLeann BednarczykAmy and Douglas BergnerBerne Ready MixBig Dog Gravel ProductsCharlcie BlackRonald and Anna BlaessigBriarcli� Congregational ChurchElsa BriggsHui CaoCarolyn CarlsonMarie CarrollAsuncion ChangChevron Humankind Matching Gift ProgramChurchville Moose Lodge #2244Thomas ClarkSuzanne and Tim CollinsColumbus Claims AssociationChristian ContiJe� and Ellen ConybearDavid and Paula CoulombeDr. William and Rachel DaviesDavid and Karen DevinoChristine DickinsonLloyd and Darlene DolbyAline DunnChris and Maureen DurackCharles and Torena EhlertDiane EngelExxonMobil FoundationRebecca FairchildSteve Filardo, MDRita FilowickFirst Ticket Travel, Inc.Gayle Flanagan and Ken CoxRichard and Linda FoisyDan and Pamela GallagherKyle and Jennifer Garza

Jess and Christine GasparPatricia and Robert GawneGE Foundation Matching Gifts ProgramArthur and Marie GodfreyGold’ s Holding CorpHoward and Susan GoldbergThomas and Lisa GrybaBill and Stephanie HammSusan HansonJean HopemanPatrick and Rochelle HultonIllinois Tool Works FoundationZachariah JenningsPatrick JonesRobert KaplanKitty and Neil KaplanGreg and Alba KeelingDorothy KelsoPaul LackJessica LeeBrian LevineTimothy LindleyKathy LoebNancy LoebBryan LoewenthalRobert and Rosalia MartinezSheila McCloskeyMary McEwanMcGladrey Inc.Jodi McKonicCarol Ann and Robert McMillanPaul MiskellyVincent MorrissetteLorry MurphyNational Metal Stampings, Inc.Ohio Chapter of IASIUStuart and Jo Anne OliverBerend OzceriCatherine PolcenFatima ReepWilliam RelyeaRainer and Fangyun RichterChris and Jane RiversGerald Roberts

Ronald and Carol RoweLouis RudgersNancy Rutledge, RNSan Antonio Chapter of Oncology Nursing SocietyPatricia SchleuningTyler SchwartzJim SchwartzHeather SellsAaron and Pamela SharpNanci SheltonGayle SlackEaton SmithMatthew and Cyndi SmithJohn, Pamela and Rachel SpearsScott StoddardPatricia StohlerDouglas and Candida StoutenboroughTom TheilerWilliam TherouxJack and Janet TreadwayCharles ViersPatricia WaltonPat WatsonChanel WhitacreJerry and Lois WilsonStephanie Ziegler

PNH FOUNDATION DONORSThe PNH Research and Support Foundation is a patient partner organization with AA&MDSIF.Amos and Ruth Wilnai FoundationLauren BoyerJoe CusanelliJohn and Leslie FavicchiaRobert GoldenBill and Linda HalvorsenCatherine PrainoWintek CorporationYotam Yemini

DONOR HONOR ROLL continued

Deanhasaplasticanemiabutit’snotonlyhisdisease,it’sourdisease,andwe’retakingthisjourneywithAA&MDSIF.ThankstoeachofyouwhohelpsupportAA&MDSIF.

Dean and Jo He�el�nger

DorothyKelsoisalong-timefriendtoAA&MDSIF.Hergrandson,ScottPaulWestin,wasbornin1979anddiagnosedwithAplasticAnemiain1986atage7½.“Whatinspiresmetocontinuetosupportthework(ofAA&MDSIF)iswhatIhavesaidmany,manytimes:Ihavefivegreat-grandchildren,soIstillhaveavestedinterestindoingwhateverIcantohelpwipeoutthishorribledisease,”sheadded.“Icontinue,tothebestofmyfinancialability,tomakemymonthlycontributions(toAA&MDSIF)inmemoryofmyhusband,inmemoryofmyolderdaughter,andofcourse,inmemoryofmydarlinggrandson,ScottPaulWestin.”

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CORPORATE LEADERSHIP CIRCLE

CHAIRMAN’S CIRCLE ($100,000 +)Celgene CorporationAlexion Pharmaceuticals VISIONARIES ($50,000 - $99,999)Onconova Therapeutics, Inc.Amgen, Inc.Novartis INNOVATORS ($25,000 - $49,999) CHAMPIONS ($10,000 - $24,999)Incyte Corporation PATRONS ($5,000 - $9,999)4ImprintMEI Pharma, Inc. BENEFACTORS ($2,500 - $4,999) Otsuka America Pharmaceutical, Inc.

GUARDIANS OF HOPEMembers of the Guardians of Hope have created legacies through bequests or by naming AA&MDSIF as a bene�ciary of a trust, IRA, or insurance policy. Their planned gifts will strengthen the Foundation’s e�orts for generations to come. We are grateful to the following individuals for remembering AA&MDSIF in their estates. AnonymousJane and James AndersonAlice Lucille BlakeMargaret “Maggie” A. BrownFlorentine CamenischMelvin ChristiansonFrancis ClayDeloris CopeRichard E. DavisKaren FisherFaithe E. FurnasVincent and Patricia GeczikHelen L. GrahamMichael I. Greenberg, MDJoanne M. HansonDavid W. HutchisonCarolyn IsaacsRobert O. JohnEdward KandelGeorge F. KappeltMihchael KarnacewiczBetty G. KeelPage C. Keel, Sr.Ernest and Mary Ellen KnappMary J. KronmanLisa LancasterW. Homer Lindahl

Arnold M. MackinnonSally L. McInnisEileen M. MichaelyMarica Cok MillerDoris N. MorelandShirley and Patrick OldsSusan M. PaxJeanne RaislerCarmine F. RicciPhillip L. Schre�erPamela SpearsLois Walter

ENDURING FRIENDSAA&MDSIF thanks the following individuals for their consistent giving for 10 or more years.Anonymous Je�rey AbtsSara BakerShelly and Patricia BarrisLois BendaMary Bischo�Ronald and Anna BlaessigBob and Emma BorlandVirginia BrittinghamThomas BryantFrances Car�Marie CarrollGertrude and Eugene CataniaHarriet and Mel ChaifetzMarie CharlesCatherine DunlopMary FarringtonGeorge and Eleanor FilowickRichard and Pam GrossRalph and Sally HamakerBill and Stephanie HammJoanna HenkelJean InnauratoArnold and Judith IsaacsDonald and Susan JosephsonRobert KaplanDorothy KelsoPaula KempchinskyThomas KnowltonJanine KowackMike and Laurie Ann KulagaRita KunofskyRobert LarrabeeLeonard and Harriet LevineSteve and Beverly MarceWilliam and Jennifer MasonDoris MorelandRobert Nahabedian and Claudine LouisHerbert and Barbara NoblesRobert and Sandra OglesbyIdo and Orlandina PuglieseFred and Jan ReeseElsa Roberts

Margaret and Louis RomanoAnthony RussoJack and Judith RyderNancy SandAnthony and Briget San�lippoSusan SattlerRobert and Lois SchultzJack SchwartzSuzanne and Darryl SjostromEaton and Lucy SmithLynne SowersShizue SpielbergCarol StewartHelene ValentiMary Elaine and Javier ValenzuelaStephanie VendigHerbert and Yvette VinePatricia and Jim WardAllen and Susan WeaverRudi WehmschulteRichard, Annette, and Kathryn WeiserRoger WestmanJack and Rebecca WilsonScott and Sarahjane Wisthu�Yahn Family

PatriciaandVincentGeczikLegacyGifttoFundPNHResearch

“Momalwaysjokedabout‘loavesandfishes’,”saidDanaConsoli.“Sheneverturnedanyoneaway.Everyonewaswelcomeatourtable.”Itwasthatessentialcommitmenttohelpingothersthatcreatedalegacyforpatientsandfamilieswithbonemarrowfailurediseases.

DanaConsoli,isthedaughterofPatriciaandVincentGeczik,whorememberedtheAplasticAnemia&MDSInternationalFoundationwithaspecialgiftintheirestate.TheirgiftwillsupportAA&MDSIF’seffortstofindbettertreatmentsandperhapsacureforparoxysmalnocturnalhemoglobinuria,PNH,Dana’sdisease.“Wewantthelegacythatmyparentscreatedtohaveapositiveandlastingimpact,”saysDana.Wedon’tknowwhatthatlookslikeyet,butthatistheawesomegoal.”

DONOR HONOR ROLL continued

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EVENTS VOLUNTEERS

2013 AA&MDSIF Volunteer Wrap-Up

AA&MDSIF volunteers made a big di�erence again this

year, helping to raise more than $246,000 while building

awareness for what bone marrow failure disease is and

what AA&MDSIF o�ers for its patients and families.

HERE IS A SNAPSHOT OF 2013 VOLUNTEER ACTIVITIES:

2013 EVENTS INCLUDED:

Annual events included: • Torry Yahn Barbeque and Auction• Judy Joyce Memorial Golf Tournament • Catherine’s Craft Fair• 5 Mile Walk• TS Gates Memorial Run/Walk• Wing Fling• Camp for a Cure• Flapjack Fundraiser• Smac Down

Other events included:• Walks/Runs (A Walk to Remember; Napa

Valley Half Marathon)• Fitness events (B4MDS, Dance of Life,

Soccer Tournament, Uplifting Athletes, Checking for Charity)

• Chips for Charity and Mom Prom • Denim Day at work• Flapjack Fundraiser• Coasters for a Cause• #GivingTuesday• Game Night• Awareness Week events • Bone marrow and blood drives

HOPEis action

Volunteering for AA&MDSIF is just one way to get involved

and give back. More than 700 volunteers help organize

fundraising and awareness events around the United States.

55+ fundraising/ awareness events• 32+ new fundraising/

awareness events• 300+ volunteers helping at

all events• 26 grassroots fundraisers• 18 new grassroots fundraisers

5 walks raised over $114,000• 2 new PNH walks• 595 walkers• 49 walk teams• 70+ volunteers• 2 5Ks

Over $246,000 raised from all events• 25 events raised $1,000+• 11 events raised $5,000+• 6 events raised $10,000+• 5 events raised $25,000+• 1 event raised over $40,000

Hope, Steps & A Cure Walks: 5 walks raised over $114,000:• San Antonio, TX• Los Angeles, CA• Milwaukee, WI• Oneonta, NY• New York, NY (new partnership

with PNH Foundation)

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LEADERSHIP AWARDS

The Edward P. Evans Foundation, Dr. Mikkael

Sekeres, and Neil Horikoski were named

recipients of the Foundation’s 2013 Leadership in

Philanthropy, Science, and Service Awards.

The Leadership in Philanthropy Award is presented for continued and consistent generous �nancial support of AA&MDSIF programs and services. The Edward P. Evans Foundation has been awarded the Leadership in Philanthropy award in recognition for its support of the

AA&MDSIF MDS Clinical Research Consortium.

The MDS Clinical Research Consortium, a six-institution consortium designed to undertake unique studies and trials to advance treatments and improve outcomes for patients with myelodysplastic syndromes (MDS), was established in 2012. The consortium enlists multiple MDS clinical research centers and �lls a major gap in MDS-related clinical research in the United States by providing a new “critical mass” of patients and patient data to support the evaluation of promising new compounds, epidemiological studies, and translational studies leading to new classi�cations, treatments, and procedures. In this collaborative environment, enhanced by expanded access to patient data, tissue samples, and the use of a standardized patient work-up, research leading to potentially bene�cial outcomes for patients is accelerated.

The Edward P. Evans Foundation, whose primary mission is to support transformational and collaborative medical research in the areas of myelodysplastic syndromes and acute myeloid leukemia, supported the �ve-year, $16 million initiative. This is believed to be the largest ever single award to support MDS clinical research.

Mikkael A. Sekeres, MD, MS has been presented the award for Leadership in Science. Recipients of this award are selected for their contributions to the �elds of bone marrow failure disease treatment and/or research. Nominees are considered for their impact on patients and families a�ected by aplastic anemia,

MDS, and PNH, and/or their contributions through research to increasing knowledge and understanding about these diseases. Additional consideration is given to those who have demonstrated a commitment to AA&MDSIF.

Dr. Sekeres is an associate professor of medicine and Director of the Leukemia Program, Department of Hematologic Oncology and Blood Disorders with the Cleveland Clinic Taussig Cancer Institute, Ohio. He earned a medical degree and a master’s in clinical epidemiology from the University of Pennsylvania School of Medicine. He completed his postgraduate training at Harvard University, �nishing an internal medicine residency at Massachusetts General Hospital and a fellowship in hematology-oncology at the Dana-Farber Cancer Institute in Boston. A frequent speaker and presenter for AA&MDSIF patient and family conferences and webinars, and webcasts and videos for both patients and health professionals, he is co-chair of the AA&MDSIF Medical Advisory Board (MAB), and also co-chair of the MDS Clinical Research Consortium.

Neil Horikoshi has been named the recipient of the Leadership in Service award. Recipients of this award are selected for their demonstrated commitment of time, energy, and ideas that raise awareness for AA&MDSIF and bone marrow failure diseases. Their outreach to engage the public and/

or AA&MDSIF patients and families results in new levels of achievement for AA&MDSIF.

Horikoshi joined the Asian & Paci�c Islander American Scholarship Fund (APIASF) in 2008 as the president and executive director after a distinguished 30-year career at International Business Machines Corporation (IBM), where he served in a variety of legal and executive management positions in the United States and Asia Paci�c. He is a past chairman of the AA&MDSIF Board of Directors, and led its transformation to a strategic and policy making governance Board. He is an Advisory Council member for both the Asian American Justice Center (AAJC) and the Asian Paci�c American Institute for Congressional Studies (APAICS). He also serves on the Board of Governors for the Go for Broke National Education Center and as an Advisory Council member for the Bill & Melinda Gates Foundation’s Gates Millennium Scholars Program.

2013 Leadership Award Recipients Announced

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HOPEis a voice

Global Awareness Program

AA&MDSIF has launched a Global Awareness Program. Working with existing patient advocacy organizations in Europe, Canada, the United States, and Australia, AA&MDSIF shares educational materials and programs, adapting and translating them for use in other countries, and assists program partners in developing their organizational capabilities.

MDS AllianceOriginally conceived to address

the problem of ageism in the treatment of older MDS patients, leading MDS patient advocacy organizations in North America

and Europe have formed the MDS Alliance to better serve the

MDS patient and caregiver community globally and provide optimal care for all MDS patients worldwide.

AA&MDSIF was selected to serve as the convener and the administrative secretariat of this new organization. MDS Alliance members include the Aplastic Anemia & MDS International Foundation, the MDS Foundation, the Aplastic Anemia & Myelodysplasia Association of Canada, the Asociación Linfoma Mieloma y Leukemia, Leukämiehilfe Rhein-Main-MDS, and MDS-UK.

Patients and family members tell us that the AA&MDIF is their most trusted resource for information about bone marrow failure diseases. Providing patients and their families with access to timely and high-quality information enables them to become strong advocates, and to take an active role in decisions about their care and treatment.

Online Learning CenterThe Online Learning Center provides easy access to more than 100 presentations by key opinion leaders in bone marrow failure diseases. Participants can stop, review, or watch repeatedly, as well as share with other family members involved in the patient’s care.

MDS ConnectionThe MDS Connection is an important resource for MDS patients. Each issue includes interviews with experts, positive stories about MDS patients and their families managing their disease and; information about AA&MDSIF direct support services for patients and caregivers. The MDS Connection has a circulation of 13,000 and is also distributed to physician’s o�ces, at conferences, and via patient information packets. Six regular editions and one special edition were published in 2013.

Treatment Tracker and What is MDS? AppsThe Treatment Tracker enables patients to track treatment, document and display in graph form any symptom/side e�ects experienced, as well as create a record and monitor lab results. There were nearly 300 users of the online Treatment Tracker in 2013. Also in 2013, a What Is MDS? iPad App was developed as a resource for both clinicians and patients.

Research UpdatesResearch Updates for Patients provide the most up-to-date information about signi�cant research in bone marrow failure diseases diagnosis, prognosis, and treatment. Members of the AA&MDSIF Medical Advisory Board identify the most recent top scienti�c studies presented through the American Society of Hematology (ASH), National Heart Lung & Blood Institute (NHLBI), National Cancer Institute (NCI), MDS Clinical Research Consortium, European Hematology Association (EHA), American Society of Clinical Oncology (ASCO), and other notable resources. This year, 3,000 copies were printed, as well as made available online and in the MDS Connection.

Standing Up for Your HealthAA&MDSIF developed Standing Up for Your Health to help people with bone marrow failure diseases become e�ective self-advocates and better navigate the healthcare system. The Center for Plain Language recognized the program in 2013 with its ClearMark Merit Award.

HOPEis understanding

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is educationAA&MDSIF has o�ered regional day-long conferences since 2011 to provide patients and families with answers from medical experts, support from other patients and caregivers, and to leave with a sense of optimism and hope. In 2013 there were more than 700 registrants attending conferences in Phoenix, Houston, Cleveland, San Francisco, Boston, and Tampa. Live international webcasts of conference sessions were added in 2013, enabling more than 530 registrants from 37 countries to join us. More than 1,700 individuals registered for educational webinars. Online learning programs are available to patients and families anytime and anywhere.

“It’s so good to be with people who understand what we’re going through, that we are not alone, and that we have all of you all in our corner. AA&MDSIF o�ers support you can’t get anywhere else.”

Martha Porter and Justin Simon

“Having both of my senior parents diagnosed with bone marrow failure disease, I attended the AAMDSIF conference with a mission in my heart- seeking understanding of this disease, how it progresses, and all of the possible treatments. I left that day with all of those questions answered. The unexpected gift that day: I found just the right blend of encouragement, expert information, personal stories, and an opportunity for on-going connections and help.”

-Jennifer

HOPE

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HOPEis strength in numbers

COMMUNITIES OF HOPE

AA&MDSIF continues to build Communities of Hope – volunteer led, community-based support groups that connect patients and families. Communities of Hope provide a local resource for support, information exchange, and education, and engage volunteers to create awareness and encourage advocacy.

As of 2013, 12 Community of Hope groups operate around the United States and eight more are in development.

• Phoenix, AZ

• San Francisco Bay Area, CA (MDS)

• Middletown, CT

• Atlanta, GA

• Chicago, IL (MDS)

• Boston, MA

• Asheville/Henderson, NC

• Central NJ

• Portland, OR (MDS)

• Houston, TX

• Seattle, WA

• Milwaukee, WI

The Support Connection is a national network of volunteers, including patients, caregivers, and family members, who o�er comfort and support to others. One-to-one support sessions take place over the phone and via email with AA&MDSIF trained volunteers who share personal experiences, coping strategies, problem solving skills, and other resources.

Support Connection

AA&MDSIF Social Media

In our social media communities, patients, families, healthcare providers, and friends learn the latest in bone marrow failure disease research, read about upcoming events, share personal stories, and connect with fellow patients and families.

By the end of 2013, our Facebook page had more than 4,400 fans, we had 400 followers on Twitter, and the AA&MDSIF YouTube channel had over 12,000 views.

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The Aplastic Anemia & MDS International Foundation was one of two recipients of a new Innovation Impact Award by Celgene Corporation. AA&MDISF received the award for its MDS Clinical Research Consortium.

Inaugurated in 2013, the Celgene Corporation awards program was created to recognize e�ective, innovative and successful initiatives of U.S.-based not-for-pro�t organizations addressing the needs of patients, caregivers and healthcare providers in today’s challenging healthcare environment in one of two therapeutic areas: hematology or oncology. Each award winner received a $100,000 cash grant. Said Celgene Vice President of Patient Advocacy, Joel W. Beetsch, PhD, “Innovation is at the core of everything we do at Celgene. The Innovation Impact Award not only recognizes the signi�cant contributions of patient and professional organizations to patient treatment and care, but also broadens the impact of the award-winning programs by helping sustain, expand, and replicate those innovative initiatives. We are delighted to announce The Aplastic Anemia & MDS International Foundation as a recipient of our �rst Innovation Impact Award.” Established in July 2012, the MDS Clinical Research Consortium is innovative in its structure, governance, funding, responsiveness, cost containment and its focus on developing future talent.

It is an unprecedented collaboration six of the largest MDS centers in the U.S. and is designed to undertake unique studies and trials to advance treatments and improve outcomes for patients with myelodysplastic syndromes (MDS). The next generation of MDS clinician researchers is being fostered through the “Edward P. Evans Fellows” program within the MDS Clinical Research Consortium.

The �ve-year, $16 million initiative is sponsored by AA&MDSIF and supported by the Edward P. Evans Foundation. The programs are located at the Cleveland Clinic Taussig Cancer Institute, the Dana-Farber Cancer Institute, the MD Anderson Cancer Center, the H. Lee Mo�tt Cancer Center and Research Institute, the Weill Medical College of Cornell University, and the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

AA&MDSIF Receives Innovation Impact Award

is the futureHOPE

Finding better treatments and working toward a cure.

ABoneMarrowFailureDiseaseCommunitySymposia was piloted in collaboration with the University of Maryland, School of Medicine in 2013. Attended by more than 100 physicians/fellows, nurses, pharmacists, physician assistants, researchers, and other healthcare professionals, the program facilitated the translation of research �ndings into clinical practice to improve patient care.

The eInsider forHealthProfessionals is a quarterly electronic newsletter distributed to more than 14,000 physicians, nurses, researchers, social workers, and other health professionals who work with patients with bone marrow failure disease. The publication includes articles on practical treatment guidance, communicating with patients, and applying research �ndings. Each issue also includes information about research updates, information on patient education resources and techniques, and live and web-based continuing medical education (CME) opportunities.

AA&MDSIF initiated the MDS/MPNRounds program in 2011 in partnership with the MPN Research Foundation and collaborating with �ve medical institutions in Chicago. Participants share best practices and learn about new therapies, clinical trials, and advances in the management of myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN) through interactive case studies presented by experts in the greater Chicago area. To date, approximately 150 physicians have attended this series.

AA&MDSIF o�ers a variety of continuing education programs and resources for nurses to learn more about the latest treatment for patients with bone marrow failure disease and to learn how to educate these patients and their caregivers. The NursingNetwork provides a link to experts who have a vast array of experience caring for patients with bone marrow failure diseases. Community Symposia provide updates on the most recent research related to the biology and clinical management of bone marrow failure diseases. 138 nurses and 66 patients attended three Treating MDS: Nurse and Patient Learning Events, while 350 nurses attended the Oncology Nursing Society Satellite Symposium.

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Dr. Parinda A. Mehta received a grant through the AA&MDSIF Trinity Ewert Research Fund which allowed her to conduct a pilot study into quercetin in patients with fanconi anemia. Quercetin is a naturally occurring �avonoid

that Mehta hypothesized had the potential to prevent progressive bone marrow failure in children with fanconi anemia.

Mehta completed her pilot project, and preliminary results suggested that quercetin is well tolerated in the pediatric fanconi anemia population, and has the potential to produce therapeutically relevant levels of the �avonoid in the blood stream. She was then able to demonstrate the feasibility of conducting a study with children who have such a rare disease. Her pilot study provided enough data to apply for a R01 grant through the FDA, which received federal funding in 2013 totaling $600,000 over two years to continue her work.

“The seed money received from AA&MDSIF provided the stepping stone for this study. It really made a di�erence to have an organization like AA&MDSIF support this study, and that we had generated preliminary data using the AA&MDSIF grant. A study like this, which is �rst of its kind in a rare disease like FA, raises a lot of questions regarding logistics and actual feasibility,” said Mehta. Mehta is Professor, Division of Bone Marrow Transplantation and Immune De�ciency at Cincinnati Children’s Hospital.

Grantee Profile: Parinda A. Mehta, MD

Named Research FundsLiviya Anderson Jack Byrne Holly Cataldo Michael Fernandes Earl Goldberg Stephen and Joanna Janowiak Sam Jordan Emily Matilda Kass Caitlyn Langley Mary Pat Madden-Grieshaber

Lindsay Marshall Minelli Mary Lou and Manuel Palacio PNH FoundationProviding New HopePursuing New HopeResearch is Hope for AA, MDS, and PNHBradley Richard Harold Spielberg Amber Lynn Wake�eld Torry Yahn

RESEARCH GRANTSAA&MDSIF provides patients and families with answers, support, and hope. And, while each of our programs and services touch on all three, our research grants inspire the most hope. For more than 20 years, AA&MDSIF has provided �nancial support for research that leads to new insights into the causes of bone marrow failure disease and the development of new therapeutic approaches. As of 2013, AA&MDSIF has awarded more than $3.5 million in research grants to 61 researchers through individually named research funds, and with the contributions of many families and individuals toward the Research is Hope Fund.

Andrew Dancis, MD Research Title: SF3B1 mitochondrial phenotype in myelodysplasia as a therapeutic target Funded By: Research is Hope Fund

Eirini Papapetrou, MD, PhD Research Title: Modeling 7q-MDS with human induced pluripotent stem cells Funded By: Research is Hope Fund

Akiko Shimamura, MD, PhD Research Title: Genetic predictors of response to immunosuppressive therapy for aplastic anemia Funded By: Torry Yahn Research Fund

Hideki Makishima, MD, PhD Research Title: Clonal Architecture in PNH: Somatic genetic defects facilitating clonal expansion Funded By: PNH Research and Support Foundation

Rosario Notaro, MD Research Title: The role of GPI-reactive T cells in the pathogenesis of acquired aplastic anemia Funded By: Amber Lynn Wake�eld Research Fund

Harold Spielberg Research Fund2007 AA&MDSIF Leadership in Philanthropy Award recipient, Shizue Spielberg, and her husband, Harold, started funding MDS research through AA&MDSIF in 1998 after he was diagnosed with MDS. In 1999, following his death, Mrs. Spielberg decided to honor him by establishing the Harold Spielberg MDS Research Fund. Her goal in establishing this named research fund is to better understand how to treat and cure MDS. Thanks to the generosity of Mrs. Spielberg and her company, Vibratex, Inc., the Harold Spielberg Research Fund has supported �ve investigators for more than 10 years of vital research projects.

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SCHOLARSHIP RECIPIENT PROFILEDerek Cope – Impossible Has Never Been An Option For Me

Derek lives life like there’s not a moment to waste. His diagnosis of aplastic anemia �ve years ago has had a lot to do with that. In 2009, Derek had begun his studies in pre-med at Stony Brook University

but had to drop out of college. After missing a year of school, Derek returned to Stony Brook, determined to continue his studies.

Awarded Matthew Debono scholarships in both 2012 and 2013, Derek was accepted into a one-year accelerated degree program in nursing. He became involved in student government, volunteered at the Stony Brook Pediatric Cancer Center, and was also part of the �rst student-run bone marrow drive at his university, which succeeded in adding 841 people to the registry. He was also a founding board member of the Stony Brook Chapter of the National Student Nurses’ Association. This year, he was awarded the State University of New York award for Student Excellence and was invited to join the international nursing honor society after graduating Summa Cum Laude.

“I navigated through this disease with the support of my family and friends, and the belief that I was stronger than the disease I was battling,” says Derek. “At times, this program seemed impossible. However, I would remember my battle with aplastic anemia and know that impossible has never been an option for me. This is just one example of how my experience with my disease has changed me. It has given me the ability to �nd strength within myself to face my challenges head on and persevere to overcome them.”

Derek hopes to work at the Stony Brook University Hospital and build his nursing skills to work on a critical care �oor in an intensive care unit. He eventually wants to earn an MBA and work in hospital administration.

“Being a two-time recipient of the Matthew Debono Memorial Scholarship will be something I will always be thankful for,” he says. “My survival of this disease has taught me many things. The best advice I can give to anyone with aplastic anemia is not to think ‘Why me’, but to think, ‘You can’t beat me!”

MatthewDebono was diagnosed with aplastic anemia when he was a freshman at Wabash College in Indiana. He was unable to complete his college education, and his family established the Matthew Debono Memorial Scholarship Fund in his memory in 1986. Two years ago, the Debono family partnered with AA&MDSIF to expand Matt’s legacy through scholarships. Thanks to the Debono family and friend, students from 13 states were awarded scholarships ranging from $1000 to $2000 in the 2013-2014 academic year. The scholarships are awarded to students impacted by bone marrow failure disease. Since 2008, AA&MDSIF has awarded scholarships to students who have had to interrupt their studies, extracurricular activities or jobs due to their health condition and treatments, and whose families �nd it �nancially di�cult to a�ord higher education.

Kyle AndersonStevensville, MTUniversity of Montana - Missoula

Kristen CavalleriNorth Tonawanda, NYNiagara University

Derek CopeJessup, MDStony Brook University

Kellie DayHixon, TNCarson-Newman University

Bailey HildebrandCottage Grove, OROregon Institute of Technology

Kayla IlesElsmere, KYChrist College of Nursing/Health Sciences

Christian LaChanceDumfries, VAVirginia Tech University

Catherine MacLeanSouth Hamilton, MAWesleyan University

Tim McCarthyBrush Prairie, WASt. Olaf College

Chris NeinChesapeake, VAEastern Virginia Medical School

Kylene OgbornIsland Lake, ILElmhurst College

Nicholas PipitoneHarrison, NYUniversity at Albany/State University of NY Haley RamirezAustin, TXTexas State University

Evelyn SteinStatesville, NCLenoir-Rhyne University

Jennifer UllmanSonoma, CASan Jose State University

2013 – 2014 Scholarship Recipients

DEBONO SCHOLARSHIPS

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The Aplastic Anemia & MDS International Foundation

(800) 747-2820 | (301) 279-7202

100 Park Avenue, Suite 108

Rockville, Maryland 20850 U.S.A.

www.AAMDS.org

HOPE