Lessons learned from a pediatric clinical trial: the Pediatric Heart Network XXXX inhibition in @entity339 study.

@entity1 @entity4688 @entity339 @entity1289 @entity474 @entity65 @entity1284
BACKGROUND: @entity339 is the most common indication for reoperation in @entity1 following repair of @entity474 ( @entity474 ). We hypothesized that @entity1289 inhibitor therapy would decrease the severity of @entity339 and limit left ventricular @entity1284 in @entity1 following @entity474 repair. METHODS: The Pediatric Heart Network designed a placebo-controlled randomized trial of @entity4688 in this population. The primary aim was to test the effect of @entity4688 on the change in left ventricular end-diastolic dimension body surface area-adjusted z score. Before the launch of the trial, a feasibility study was performed to estimate the number of @entity1 with at least moderate @entity339 following @entity474 repair. TRIAL EXPERIENCE: Seventeen months after the start of the study, 349 @entity1 were screened, 8 were trial eligible, and only 5 were enrolled. The study was subsequently terminated because of low @entity1 accrual. Several factors led to the problems with @entity1 accrual, including (1) the use of criteria to assess disease severity in the feasibility study that were not identical to those used in the trial, (2) failure to achieve equipoise for the study among clinicians and referring physicians, (3)  reliance on methodology developed in adult populations with different disease mechanisms, and (4) absence of adequate data to define the natural history of the disease process under study. Progress in the treatment of @entity1 with @entity65 will depend on the future of multicenter collaborative clinical trials. The lessons learned from this study may contribute to improvements in this research.
