Titre :

Challenges and Pitfalls: Performing Clinical Trials in Patients With Congenital Diaphragmatic Hernia.

Auteur(s) :

Cochius-Den Otter, Susan [Auteur]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]
Deprest, Jan A. [Auteur]
Catholic University of Leuven = Katholieke Universiteit Leuven [KU Leuven]
University College of London [London] [UCL]
University Hospitals Leuven [Leuven]
Storme, Laurent [Auteur]
Hôpital Jeanne de Flandre [Lille]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Greenough, Anne [Auteur]
King's College Hospital [KCH]
Tibboel, Dick [Auteur]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]

Titre de la revue :

Frontiers in Pediatrics

Nom court de la revue :

Front Pediatr

Numéro :

10

Pagination :

852843

Date de publication :

2022-05-05

ISSN :

2296-2360

Mot(s)-clé(s) en anglais :

congenital diaphragmatic hernia
clinical trials
congenital anomaly
prenatal therapy
postnatal therapy

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Congenital diaphragmatic hernia (CDH) is a rare developmental defect of the lungs and diaphragm, with substantial morbidity and mortality. Although internationally established treatment guidelines have been developed, most ...
Lire la suite >Congenital diaphragmatic hernia (CDH) is a rare developmental defect of the lungs and diaphragm, with substantial morbidity and mortality. Although internationally established treatment guidelines have been developed, most recommendations are still expert opinions. Trials in patients with CDH, more in particular randomized controlled trials, are rare. Only three multicenter trials in patients with CDH have been completed, which focused on fetoscopic tracheal occlusion and ventilation mode. Another four are currently recruiting, two with a focus on perinatal transition and two on the treatment of pulmonary hypertension. Herein, we discuss major challenges and pitfalls when performing a clinical trial in infants with CDH. It is essential to select the correct intervention and dose, select the appropriate population of CDH patients, and also define a relevant endpoint that allows a realistic duration and sample size. New statistical approaches might increase the feasibility of randomized controlled trials in patients with CDH. One should also timely perform the trial when there is still equipoise. But above all, awareness of policymakers for the relevance of investigator-initiated trials is essential for future clinical research in this rare disease.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
CHU Lille

Collections :

• METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694

Date de dépôt :

2023-11-15T04:14:46Z
2024-01-18T13:28:00Z