Molecular screening for cancer treatment optimization (MOSCATO 01) in pediatric patients: First feasibility results of a prospective molecular stratification trial.

10050 Background: This feasibility study intends to characterize genomic alterations in recurrent tumors of an individual pediatric patient in order to select a targeted therapy approach. Methods: Pediatric patients with recurrent or refractory solid tumor were proposed to undergo on-purpose tumor biopsy or surgical resection for molecular characterization. Biopsies were obtained using 18G needles under ultrasound, CT or MRI control from primary tumor or metastatic sites. DNA extracted from fresh samples was analyzed by CGHarray 180K (if ≥ 50% tumor cells in the sample) and by sequencing for 74 target genes (if ≥ 30% tumor cells). A panel of scientists and clinicians reviewed results to determine biological signification of the alteration and match patients to the most relevant targeted therapy available (mainly in early clinical trials). Results: From December 2012 to January 2014, 12 patients were consented; 3 were screening failures. Nine enrolled patients with a median age of 7 years (range, 3-18) and...