Author Affiliations: Nemours Cardiac Center, A. I. duPont Hospital for Children, Wilmington, Delaware; Jefferson Medical College, Philadelphia, Pennsylvania.
Experience with randomized controlled clinical trials in pediatric cardiology is limited. Perhaps the most cited article in the field had a sample size of 1, a baby with transposition of the great arteries who successfully underwent balloon dilation of a patent foramen ovale.1 When this procedure was found to improve survival from a median of less than a week to several years, the immediate challenge to clinicians was not to replicate the finding by a randomized trial but to determine how best to manage a living child with an oxygen saturation of 60% to 70% and persistent complex anatomical defects.
Gidding SS. The Importance of Randomized Controlled Trials in Pediatric Cardiology. JAMA. 2007;298(10):1214–1216. doi:10.1001/jama.298.10.1214