Randomized Controlled Trials in Pediatric Cardiology: A Power Struggle?

Pediatric cardiology: In search for evidence

“…Various reviews looked at the quality and quantity of RCTs in pediatric cardiology over the years. [ 6 9 10 11 12 ] In an analysis of 933 pediatric cardiology RCTs done till 2018,[ 9 ] the yearly average improved to 46 RCTs/year between 2010 and 2020. Another study of 83 RCTs in pediatric cardiology[ 11 ] also confirmed a significant increase in the numbers over time.…”

“…[ 6 9 10 11 12 ] In an analysis of 933 pediatric cardiology RCTs done till 2018,[ 9 ] the yearly average improved to 46 RCTs/year between 2010 and 2020. Another study of 83 RCTs in pediatric cardiology[ 11 ] also confirmed a significant increase in the numbers over time. Despite the rise in the number of RCTs, impactful RCTs are limited in pediatric cardiology.…”

“…Most of the difficulties stem from recruiting an adequate number of subjects, randomizing to defined interventions, and choosing reliable endpoints. Most pediatric cardiology RCTs are underpowered[ 11 ] and have low generalizability, often related to the rarity of specific anatomy and pathobiology and the low occurrence of hard endpoints. Hence, a meaningful RCT usually requires extensive collaboration between the units.…”

With Adequate Power Comes Great Responsibility

“…Dear Editors, Herrera-Carrillo et al highlight concerns regarding statistical power in pediatric cardiology randomized controlled trials (RCTs) [1]. The authors conducted a systematic review of RCTs with a primary endpoint, and checked if the sample size was adequate to attain 80% power for rejecting the null hypothesis at p < 0.05, given the results presented in each article.…”

“…This post hoc power analysis should be differentiated from an a priori power analysis that might be conducted during trial design, which aims to determine whether a study will be adequately powered for an expected effect size, regardless of what the data ultimately show. On post hoc power analysis, only 45% of pediatric cardiology RCTs attained a power of 80%, leading the authors to call for reducing target power to 60%, relaxing the p value threshold for statistical significance, or both [1].…”