Core Outcome Measures in Effectiveness Trials

Development of a core outcome set for fetal interventions for myelomeningocele (spina bifida)

General Information

Summary:
Fetal myelomeningocele (spina bifida) is a rare but severe fetal condition. Incomplete closure of the neural tube during embryonic life leads to persistent exposure of the spinal cord to neurotoxic amniotic fluid during pregnancy. Moreover, a lack of protection of the spinal cord from the outside world leads to repeated (micro)trauma of the nervous tissue with fetal movements. As a consequence, fetuses with myelomeningocele will be born with loss of nerve function below the level of the spinal lesion. Clinically, this results in a loss of motor function and sensation in the lower limbs and pelvic floor area, with ensuing ambulation problems, bowel, bladder and sexual dysfunction.

Besides these distal problems, the fetal spinal defect also leads to leakage of cerebro-spinal fluid, which induces hindbrain herniation and secondary hydrocephalus. As such, most infants with myelomeningocele will require surgery at birth to relieve the intracranial pressure (ventriculo-peritoneal shunting). This procedure is associated with long-term complications such as shunt obstruction or infection, with ensuing impact on neurodevelopment.

Fetal surgery for spina bifida has been shown to improve fetal motor function, to decrease hindbrain herniation and to reduce the need for postnatal shunting. As such, it has found widespread uptake in the last decade. However, the surgery carries significant fetal and maternal risks and new, less invasive fetal procedures are currently being developed to hopefully reduce these associated risks. Unfortunately core-outcomes to be reported on in eventual future intervention trials have not been agreed upon.

The aim of this study is to define a set of core-outcomes to be reported on in studies investigating interventions for fetal myelomeningocele. This core-outcome set will be developed using a validated methodology (systematic review, stakeholder interviews, Delphi questionnaires and consensus meeting).

Contributors:
Tim Van Mieghem
Maternal Fetal Medicine Specialist
Mount Sinai Hospital, Toronto, Canada

Clare Whitehead
Maternal Fetal Medicine Fellow
Mount Sinai Hospital, Toronto, Canada

Greg Ryan
Maternal Fetal Medicine Specialist
Mount Sinai Hospital, Toronto, Canada

Jan Deprest
Maternal Fetal Medicine Specialist
University Hospitals Leuven, Leuven, Belgium

Luc Joyeux
Pediatric Surgeon
University Hospitals Leuven, Leuven, Belgium

James Drake
Neurosurgeon
Hospital For Sick Children, Toronto, Canada

Paige Church
Developmental Behavioral Physician
Holland-Bloorview clinic, Toronto, Canada

Yenge Diambomba
Neonatologist
Mount Sinai Hospital, Toronto, Canada

Jose Carvalho
Anesthetist
Mount Sinai Hospital, Toronto, Canada


Further Study Information

Current Stage:
Ongoing
Date:
September 2018 - 2020
Funding source(s):
None

Health Area

Disease Category
Pregnancy & childbirth

Disease Name
Fetal myelomeningocele
Fetal spina bifida

Target Population

Age Range
-

Sex
Either


Nature / type of Intervention
Drug
Surgery

Method(s)

Consensus meeting
Delphi process
Survey
Systematic review

Systematic literature review to identify previously reported outcomes, survey of healthcare providers and parents of infants with spina bifida who did or did not undergo fetal therapy. Relevant outcomes will be selected by consensus through Delphi process. Consensus meeting will be held to validate results.


Stakeholders Involved

Clinical experts
Consumers (caregivers)
Consumers (patients)
Families
Patient/ support group representatives
Researchers

Study Type

COS for clinical trials or clinical research
COS for practice

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