vanderbilt evidence-based practice center katherine e. hartmann, md, phd lucius e. burch chair,...

VANDERBILT EVIDENCE-BASED PRACTICE CENTER

Katherine E. Hartmann, MD, PhDLucius E. Burch Chair, Obstetrics and Gynecology

Deputy Director, Institute for Medicine and Public HealthDirector, Vanderbilt Evidence-based Practice Center

Evidence Limited to

Inform Decisions about Maternal-Fetal Surgery


Technical Brief IntentSummarizing the state of the field including:Content of the researchAccess to fetal surgeryTraining of providersEthical aspectsCoverage of proceduresOngoing studies


Selection of target conditions Cardiac malformations Congenital diaphragmatic hernia Myelomeningocele Obstructive uropathy Sacrococcygeal teratoma Thoracic lesions (Congenital Pulmonary

Airway Malformations) Twin-twin transfusion syndrome


Review Team & Expert Panel Selection Fetal sonographers/echocardiographers

Maternal-fetal medicine specialists Perinatal geneticists Pediatric surgeons “Fetal” surgeons Neonatologists Biomedical ethicists Generalists prenatal care providers Familiarity with perinatal hospice


State of the Evidence Methods PubMed, MEDLINE (no date limits)

English, ≥ 2 cases, condition of interest, fetal intervention, training information, outcomes including quality of life (for mother or fetus)

Included RCT, cohorts with comparison, case-control, and case series


State of the Evidence Review Methods Dual review of abstracts Dual review of articles Extraction of descriptive data into

standardized forms:– study design and setting– fetal and maternal inclusion criteria – training of providers– outcomes measured – length of follow up


Disposition of the Literature Abstracts: 1382 Articles: 620 Relevant studies: 166 Study designs • RCTS: 3• Cohort studies: 47• Case series: 116

Studies from US: 74


Cardiac MalformationsStudies (n=9)• Pulmonary atresia

with intact ventricular septum—2 case series

• Aortic stenosis—8 case series

• Hypoplastic left heart syndrome and intact atrial septum—3 case series

State of the Science• 90 pregnancies in literature• 1 study provided infant

outcomes beyond hospital stay

• Maternal inclusion criteria poorly reported

• No data on long term survival or neurologic outcomes


Congenital Diaphragmatic Hernia

Studies (n=21)• 1 RCT • 3 prospective

cohorts• 5 retrospective

cohorts• 2 prospective case

series • 10 retrospective

case series

State of the Science • Fetal inclusion criteria well-

reported; 9 of 21 reported maternal criteria

• Few measured outcomes beyond hospital stay (8/21)

• Longest reported infant follow up: 24-48 months

• Long term maternal outcomes addressed in 2 studies


Overview


ChildDevelopmental

Status

Any Maternal

Outcomes

Cardiac malformations 0/9 4/9CDH 7/21 11/21Myelomeningocele 3/11 4/11Obstructive uropathy 5/25 2/25Sacrococcygeal teratoma 0 3/5Thoracic lesions 2/11 1/11Twin-twin transfusion syndrome 13/84 4/84

Outcomes reported


Challenges for the field include:• Rare conditions with no method to consolidate new cases• Cases accrue slowly - innovations change the benefit

equation• Clinical and research classifications of fetal anomalies vary• Spontaneous regression/stabilization occurs • Prognosis cannot be determined with high accuracy• Changes in the spectrum of disease reduces comparability

to prior natural history studies of anticipated outcomes• Intervention techniques change rapidly• Higher SES patients are over-represented• Regionalization of care needs consideration• No formal plan for certified training programs exists


Strategies to Reduce Evidence Gaps • Continue to develop consensus operational

definitions• Enhance attention to common data elements• Use valid and reliable tools for outcomes• Support platforms for cooperative data sharing• Use analyses to help define equipoise for trials• Develop new methods for funding and cost sharing• Establish a consistent set of ethical standards for

research in the field


Challenges for Technical Brief Process Content of the research: organizing classification, defining families,

resisting urge to cross into outcomes Access to fetal surgery: identifying pool of experts - able to

establish via direct communication with those who publish Training of providers: identifying sites - able to establish Ethical aspects: balancing priorities for the report Coverage: identifying accurate, available sources Ongoing studies: documenting trials more achievable than other

studies


Technical Brief Conclusions• Limited RCT evidence• Literature dominated by case series (70% of

studies)• Lack of clearly described maternal inclusion

criteria and long term follow-up for infant or mother

• Limited outcomes reported:

• Literature not typical level of quality of studies or aggregate strength of evidence sufficient to guide care

vanderbilt evidence-based practice center katherine e. hartmann, md, phd lucius e. burch chair,...

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