Risk and surrogate benefit for pediatric Phase I trials in oncology: A systematic review with meta-analysis.
Marcin WaligoraMalgorzata M BalaMagdalena KopernyMateusz T WasylewskiKarolina StrzebonskaRafał R JaeschkeAgnieszka WozniakJan PiaseckiAgnieszka SliwkaJerzy W MitusMaciej PolakDominika NowisDean FergussonJonathan KimmelmanPublished in: PLoS medicine (2018)
Our meta-analysis suggests that, on the whole, AE and response rates in pediatric Phase I trials are similar to those in adult Phase I trials. Our findings provide an empirical basis for the refinement and review of pediatric Phase I trials, and for communication about their risk and benefit.