Core Outcome Measures in Effectiveness Trials

Development of a core outcome set for prenatal interventions for congenital diaphragmatic hernia

General Information

Summary:
Congenital diaphragmatic hernia (CDH) is a serious developmental defect with a birth prevalence of approximately 1 in 4000. This defect results in partial or complete absence of the diaphragm resulting in liver and bowel migrating to the chest cavity and impairing lung development and function for the neonate. Current survival rates in population based studies vary between 55 and 70%. Survivors may have serious morbidity, mostly respiratory in nature as well as feeding problems and reflux, growth and orthopedic problems. The leading causes of death remain pulmonary hypoplasia and pulmonary hypertension.
Historically, this defect was treated postnatally by surgical repair of the CDH. Advances in prenatal ultrasound now mean that most cases of CDH are diagnosed in the prenatal period. Prenatal surgical interventions have been developed with the aim of improving lung size and function in utero and consequently improving lung function and neonatal outcome. Aside from new prenatal surgical techniques, new non-surgical prenatal interventions are also being developed. However, with these new developments, a wide range of outcomes are reported. This makes it difficult to compare results or combine data from individual studies, limiting the potential of research to guide clinical practice.
The aim of this study is to define a set of core-outcomes to be reported on in studies investigating prenatal for congenital diaphragmatic hernia. This core-outcome set will be developed using a validated methodology (systematic review, stakeholder interviews, Delphi questionnaires and consensus meeting).

Contributors:
Principal investigator:
Dr Simen Vergote
University Hospitals Leuven, Leuven, Belgium

Contributors:
Dr Felix De Bie
University Hospitals Leuven, Leuven, Belgium / Children’s Hospital of Philadelphia, Philadelphia, United States of America
Dr Jan Bosteels
University Hospitals Leuven, Leuven, Belgium / Imeldaziekenhuis, Bonheiden, Belgium
Professor Jan Deprest
University Hospitals Leuven, Leuven, Belgium / Institute for Women’s Health, University College London, London, United Kingdom

Further Study Information

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

Health Area

Disease Category
Pregnancy & childbirth

Disease Name
Congenital diaphragmatic hernia

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Other

Method(s)

Consensus meeting
Delphi process
Survey
Systematic review

- Prospective Registration: COMET initiative, PROSPERO
- Steering Group: international steering group comprising healthcare professionals and researchers
- Identifying potential core outcomes: systematic review
- Determining core outcomes: modified Delphi method
- Stakeholder meeting
- Determining how core outcomes should be measured


Stakeholders Involved

Clinical experts
Patient/ support group representatives
Researchers

Study Type

COS for clinical trials or clinical research

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