Outcomes of pediatric cancer patients enrolled in phase 1 clinical trials designed for adults: Experience from a major cancer center.

e21024 Background: Phase I trials play a key role in the early evaluation of novel targeted therapies for patients with advanced cancer. Pharmaceutical industry sponsored trials exclude patients less than 18 years of age for phase I clinical trials even in the era of targeted therapy. Some investigator-initiated phase 1 trials combining US FDA approved agents allow patients less than 18 years. We sought to determine the relationship between clinical characteristics and survival outcomes of pediatric patients enrolled in adult-based phase I trials at MD Anderson Cancer Center and to validate the Royal Marsden Hospital (RMH) score and the MD Anderson Cancer Center (MDACC) score as survival predictor tools in this population. Methods: Medical records of 40 patients younger than 18 years who were treated in at least 1 phase I trial at MDACC between 2005-2013 were reviewed. Univariate and multivariate analyses were used to determine which baseline clinicopathologic characteristics were associated with overall ...