There have been few late-phase clinical trials that included children who underwent surgery for congenital heart disease, leading authors of a new European Journal of Cardio-Thoracic Surgery review to call for “high-quality, multicenter clinical trials to provide a robust evidence base for contemporary pediatric cardiac surgical practice.”
“The surgery available for children with heart problems has improved dramatically over the past 20 years,” Nigel E. Drury, PhD, FRCS(CTh), clinical scientist at the University of Birmingham, United Kingdom, and honorary consultant in pediatric cardiac surgery at Birmingham Children’s Hospital, said in a press release. “However, by not carrying out large-scale, cutting-edge clinical trials to continually improve surgeries, we’re letting down the thousands of children born in the U.K. each year with heart problems.”
Researchers analyzed data from 23,902 children with congenital heart disease from 34 countries who underwent cardiac surgery in 333 randomized controlled trials from January 2000 to August 2016. Trials with outcomes that were not directly related to the cardiac surgery or involved invasive life support were excluded.
Most studies were phase 2 (94%) with a median of 45 patients (interquartile range, 28-82). Only 4.5% of trials were phase 3 or 4. Surgical intervention was only evaluated in 3.3% of trials. Sample size calculation was performed in 32.7% of trials, 15.3% were registered on a public trial database, 15.6% published a CONSORT flow diagram and 7.5% were analyzed by an independent data monitoring committee.
The overall risk for bias was unclear in 72.7% of trials; 20.7% were high and 6.6% were low.
“As a congenital heart disease community, we have the responsibility to provide scientific leadership and work together to conduct well-designed, rigorously conducted, multicenter clinical trials with clearly defined, clinically relevant endpoints that answer important questions to improve the outcomes of surgery for our patients and their families,” Drury and colleagues wrote. “Our colleagues in other specialties such as pediatric oncology have made the opportunity to participate in late-phase clinical trials part of the routine care pathway, and so must we.” – by Darlene Dobkowski
The authors report no relevant financial disclosures.