Objective
To develop a core set of prenatal and neonatal outcomes for clinical studies evaluating perinatal interventions for congenital diaphragmatic hernia, using a validated consensus-building method.
Methods
An international steering group comprising 13 leading maternal–fetal medicine specialists, neonatologists, pediatric surgeons, patient representatives, researchers and methodologists guided the development of this core outcome set. Potential outcomes were collected through a systematic review of the literature and entered into a two-round online Delphi survey. A call was made for stakeholders with experience of congenital diaphragmatic hernia to review the list and score outcomes based on their perceived relevance. Outcomes that fulfilled the consensus criteria defined a priori were discussed subsequently in online breakout meetings. Results were reviewed in a consensus meeting, during which the core outcome set was defined. Finally, the definitions, measurement methods and aspirational outcomes were defined in online and in-person definition meetings by a selection of 45 stakeholders.
Results
Overall, 221 stakeholders participated in the Delphi survey and 198 completed both rounds. Fifty outcomes met the consensus criteria and were discussed and rescored by 78 stakeholders in the breakout meetings. During the consensus meeting, 93 stakeholders agreed eventually on eight outcomes, which constituted the core outcome set. Maternal and obstetric outcomes included maternal morbidity related to the intervention and gestational age at delivery. Fetal outcomes included intrauterine demise, interval between intervention and delivery and change in lung size in utero around the time of the intervention. Neonatal outcomes included neonatal mortality, pulmonary hypertension and use of extracorporeal membrane oxygenation. Definitions and measurement methods were formulated by 45 stakeholders, who also added three aspirational outcomes: duration of invasive ventilation, duration of oxygen supplementation and use of pulmonary vasodilators at discharge.
Conclusions
We developed with relevant stakeholders a core outcome set for studies evaluating perinatal interventions in congenital diaphragmatic hernia. Its implementation should facilitate the comparison and combination of trial results, enabling future research to better guide clinical practice.
Protocol: https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-021-05120-z
S. Vergote, F. R. De Bie, J. M. N. Duffy, J. Bosteels, A. Benachi, B. Power, F. Meijer, H. L. Hedrick, C. J. Fernandes, I. K. M. Reiss, P. De Coppi, K. P. Lally, J. A. Deprest
Disease Category: Pregnancy & childbirth
Disease Name: Congenital diaphragmatic hernia
Age Range: 0 - 100
Sex: Either
Nature of Intervention: Other
- Clinical experts
- Patient/ support group representatives
- Researchers
- COS for clinical trials or clinical research
- 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