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

ABSTRACT: 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 th

SUBMITTER: Cochius-den Otter S 

PROVIDER: S-EPMC9051320 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature