Challenges and Pitfalls: Performing Clinical ...
Document type :
Article dans une revue scientifique: Article de synthèse/Review paper
DOI :
PMID :
Permalink :
Title :
Challenges and Pitfalls: Performing Clinical Trials in Patients With Congenital Diaphragmatic Hernia.
Author(s) :
Cochius-Den Otter, Susan [Auteur]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]
Deprest, Jan A. [Auteur]
University Hospitals Leuven [Leuven]
University College of London [London] [UCL]
Catholic University of Leuven = Katholieke Universiteit Leuven [KU Leuven]
Storme, Laurent [Auteur]
METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Hôpital Jeanne de Flandre [Lille]
Greenough, Anne [Auteur]
King's College Hospital [KCH]
Tibboel, Dick [Auteur]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]
Deprest, Jan A. [Auteur]
University Hospitals Leuven [Leuven]
University College of London [London] [UCL]
Catholic University of Leuven = Katholieke Universiteit Leuven [KU Leuven]
Storme, Laurent [Auteur]

METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Hôpital Jeanne de Flandre [Lille]
Greenough, Anne [Auteur]
King's College Hospital [KCH]
Tibboel, Dick [Auteur]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]
Journal title :
Frontiers in Pediatrics
Abbreviated title :
Front Pediatr
Volume number :
10
Pages :
852843
Publication date :
2022-05-05
ISSN :
2296-2360
English keyword(s) :
congenital diaphragmatic hernia
clinical trials
congenital anomaly
prenatal therapy
postnatal therapy
clinical trials
congenital anomaly
prenatal therapy
postnatal therapy
HAL domain(s) :
Sciences du Vivant [q-bio]
English abstract : [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 ...
Show more >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.Show less >
Show more >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.Show less >
Language :
Anglais
Audience :
Internationale
Popular science :
Non
Administrative institution(s) :
Université de Lille
CHU Lille
CHU Lille
Submission date :
2023-11-15T04:14:46Z
2024-01-18T13:28:00Z
2024-01-18T13:28:00Z
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