Unique Challenges of Randomised Controlled Trials in Pediatric Cardiology

ABSTRACT Pediatric cardiology has evolved over time with reductions in childhood mortality due to congenital heart disease. Surgical innovation drove early changes in care. Increasingly, the need for more robust evidence provided by randomised controlled trials (RCTs) has been recognised. Although the number of RCTs has increased, there remains a relative paucity of truly impactful trials in the field. However, those trials that have changed practice have demonstrated the potential and importance of this work. Examples include the PRIMACORP trial, which established the safety and efficacy of milrinone after cardiac surgery, and the Single Ventricle Reconstruction trial, which was the first multicentre pediatric cardiac surgical RCT. The successful conduct and important findings emanating from these trials serve as beacons as clinicians strive to improve the evidence base in this field. The establishment of national and international networks such as the Pediatric Heart Network and the Canadian Pediatric Cardiology Research Network provide a strong foundation for future collaborative work. Despite this progress, there remain important challenges to designing and executing RCTs in pediatric cardiology. These include issues of greater disease and patient heterogeneity and increased costs. The use of innovative study designs and analytic methods and the establishment of core outcome measures have the potential to overcome some of the issues related to the smaller patient numbers compared with adult disciplines. As pediatric cardiologists look to the future, it is imperative that we work together to derive the maximum benefit from the considerable efforts directed toward conducting impactful clinical trials in pediatric cardiology.