The role of prospective randomized clinical trials in pediatric surgery: State of the art?

Purpose: This study sought to determine the role of randomized controlled t rials (RCT) in the evolution of pediatric surgical practice. Methods: The authors used a computer-assisted literature search to identify all clinical trials related to pediatric surgery published in the English- language literature from 1966 through 1999. Each article was reviewed in de tail for purpose, content, conduct, and quality of the trial. The authors a ssessed quality with a previously validated instrument (Chalmers Qualitativ e Assessment). Results: The authors identified 134 RCTs related to pediatric surgery over the past 33 years. This accounts for 0.17% of 80,377 articles published in the field. The areas of surgery studied were analgesia 65 (49%), antibiotic s 17 (13%), extracorporeal membrane oxygenation (ECMO) 9 (7%), gastrointest inal, burns, oncology, minimally invasive surgery, vascular access, congeni tal anomalies, and trauma (each <5%). Only 16 (12%) trials compared 2 surgi cal therapies, 9 (7%) compared a medical versus a surgical therapy, and 109 (81%) compared 2 medical therapies in surgical patients. Fourteen (10%) RC Ts were funded by peer-reviewed agencies. Only 17 (13%) RCTs included a bio statistician as an author or a consultant. Trial design included calculatio n of sample size and statistical power in 21 (16%) RCTs. Method of randomiz ation was reported in only 51 (38%). The test statistic and observed probab ility value was reported in 15 (11%). Conclusions: Clinical trials are used infrequently to answer questions rela ted to pediatric surgery. When RCTs are utilized, they often suffer from po or trial design, inadequate statistical analysis, and incomplete reporting. Pediatric surgery could benefit from increased expertise, funding, and par ticipation in clinical trials. J Pediatr Surg 36:1182-1186. Copyright (C) 2 001 by W.B. Saunders Company.