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National Institute of Child Health and Human Development (NICHD)

Fetal Therapy: Needs Assessment and Future Directions
August 16 – 17, 2004 • Potomac, Maryland


Monday, August 16
10:00 a.m. Welcome, background
Duane Alexander
10:10 a.m. Meeting goals
Nancy Chescheir
10:15 a.m. Introductory remarks
Michael Socol
10:20 a.m. Where is the field of fetal therapy now and where are the gaps? Setting the stage for research needs.
Mike Harrison

10:45 a.m. The role of animal studies prior to human fetal interventions

Topics to be covered:
  • Is it necessary to do animal studies before human fetal interventions are done?
  • Would this apply to both studying the pathophysiology and mechanisms of repair/recovery and also for new techniques? If so, when is it acceptable to move from an animal model to human applications?
  • Assuming that the animal model has been published and shows a potential benefit, is it important that a group evaluating a procedure clinically be the one that did the animal work?
  • Without reviewing the research data itself, which animals have proven most useful for prior research (e.g.: Are there interventions in which subhuman primate models are necessary or others where rodent models are sufficient?)
  • Is it impossible to have a credible clinical fetal therapy program without an active animal lab?
  • What differences might be present, if any, when comparing animal models for structural (such as meningocele) versus functional fetal diseases (such as SCID)?
  Is it necessary to do animal studies before all human studies? When is it acceptable to move from animal to human for fetal interventions?
Alan Flake
11:00 a.m. Is it possible for a fetal treatment center to be credible clinically without an active animal laboratory?
Michael Greene
11:15 a.m. Which animal models have been most useful? How to select which model to use?
Michael Harrison

11:30 a.m. Discussion
11:45 a.m. Summary and recommendations for future actions
Mike Socol
1:00 p.m. Lessons learned, guiding principles and oversight

Topics to be covered:
  • The "no back door" policy for the MOMS trial, TTTS trials with other sites offering the therapy for TTTS.
  • Should there be some sort of overt collective conscience on the issue of cooperation for fetal therapy? If so, what would that look like and how would it be enforced?
  • Should fetal therapy remain largely self-governed?
  • What would be the impact on academic freedom and entrepreneurship?
Tim Cromblehome
1:15 p.m. MOMS
Scott Adzick
1:30 p.m. Potential models for cooperation v competition in fetal treatment
Mary D’Alton
2:00 p.m. Discussion
2:15 p.m. Summary and recommendations for future actions
Mike Socol

2:30 p.m. Public health significance of fetal therapy research

Topics to be covered:
  • Are there unintended consequences or opportunity costs to this approach to disease treatment?
  • Is there a diversion of resources from prevention or post-natal treatments that would likely involve many more patients?
  • Consideration of costs of a program like the folic acid campaign to prevent Spina Bifida and the benefits, verses dollars spent on fetal treatment.
  • Third party payment for fetal treatment.
  Jonelle C. Rowe
2:50 p.m. Discussion and summary
Mike Socol
3:00 p.m. Structure of fetal therapy programs

Topics to be covered:
  • Description of the structure of sample programs
  • Challenges and opportunities at individual institutions
  • Are there necessary components of a fetal therapy program?
  • What is the institutional support for the program that is required?
  • Are there turf issues and how have those been successfully resolved?
  • Should there be specific components in place to be a "fetal therapy program"?
Louise Wilkins-Hague
3:15 p.m. UCSF
Diana Farmer
3:30 p.m. Brown
Francoise Luks
3:45 p.m. Discussion
4:00 p.m. Summary and recommendations for future actions
Mike Socol

4:30 p.m. Informed consent and human subjects protection

Topics to be covered:
  • Federal guidelines regarding fetal research.
  • How does informed consent differ with fetal interventions versus with interventions that effect only one patient at a time?
  • What are the informed consent implications of moving from doing fetal therapy in a trial setting to offering the procedure clinically?
  • IRB approval for fetal therapy outside of protocol for innovative versus experimental interventions
  Informed consent with fetal therapies
Sue Estroff
4:45 p.m. Federal guidelines for fetal therapy: Distinction between innovative versus experimental
Carol Greene
5:00 p.m. Discussion
5:10 p.m. Summary and recommendations for future actions
Mike Socol

5:25 p.m. Study and trial design for maternal-fetal surgery

Topics to be covered:
  • What is the best design of study to evaluate fetal therapy?
  • Standardization of maternal-fetal surgical trials and surgeries
  • What should be considered evidence of success for a fetal therapy trial?
  • Given the limited number of procedures, would it be appropriate to suggest a core set of research questions? If so, what might those include?
  • How could such questions guide the development of an acceptable case controlled or cohort studies?
  • What are the pitfalls of an RCT?
  • What should be the status of the fetal therapy once the trial is done, but being analyzed?
  Best designs to evaluate fetal therapies/pitfalls of RCT
Elizabeth Thom
5:40 p.m. How should study outcomes be developed so that the investigators know when the trial has been a success? What should the status of the studied therapy be while the data is being analyzed?
Rusty Jennings
Joshua Copel
6:10 p.m. Discussion
6:20 p.m. Summary and recommendations for future actions
Mike Socol

Tuesday, August 17
8:00 a.m. Research needs and priorities in fetal therapy/maternal-fetal surgery
Disease-oriented discussions of the research questions and priority setting

(15 minute presentations + 5 minutes discussion)

Topics to be covered:
  • Instrumentation needs
  • Surgical approach issues such as robotics, minimally invasive surgery, open surgery, etc.
  • Are there animal studies to support these as reasonable research needs and if not, are they needed?
  Cardiac disease
Wayne Tworetzky
8:20 a.m. Genetic disorders
Diana Bianchi
8:40 a.m. Neurologic disorders
Scott Adzick
9:00 a.m. Gastroschisis
Joe Bruner
9:20 a.m. TTTS, complicated twins
Tim Crombleholme
9:40 a.m. Amniotic Bands
Tony Johnson
10:00 a.m. Summary and recommendations for future actions
Mike Socol
10:25 a.m. Fetal monitoring, complications and maternal effects
  Common complications of maternal-fetal interventions: Research for prevention of treatment

Topics to be covered:
  • Premature rupture of the membranes, Oligohydramnios Chorio-amniotic separation, infection (15 min)
    Ken Moise
  • Preterm birth (10 min)
    Michael Speer
10:50 a.m. Intra- and post-operative monitoring of the fetus
Diana Farmer
11:10 a.m. Effects of maternal-fetal interventions on maternal health, reproductive health, and on the family
Nancy Chescheir
11:25 a.m. Discussion
11:40 a.m. Summary and recommendations for future actions
Mike Socol
12:00 p.m. Summation and integration
Mike Socol

Topics to be covered:
  • Consensus of the research needs and priorities in fetal therapy/maternal-fetal surgery
  • Did we achieve the objective? Develop a plan for the surgical, obstetrical, neonatal, and maternal-fetal and surgery fields to develop a scientific plan for the valuation and dissemination of maternal-fetal surgical innovations and further the scientific evaluation of fetal surgery
  • How can we encourage cooperation among the diverse integral groups (fetal surgeons, MFM’s, neonatologist, etc.) to develop an oversight for the field to monitor fetal interventions and assure the evidence base for clinically offered fetal interventions?
  • Plan for dissemination of the workshop information
  • Future steps

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Last Reviewed: November 30, 2004
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