Next Generation Sequencing (Ngs) To Identify Targetable Recurring Mutations And Exceptional Responders In Relapsed And High-Risk Childhood And Adolescent/Young Adult (Aya) Malignancies.

11011 Background: NGS has been rapidly introduced into routine clinical care for select adult malignancies, and the National Cancer Institute genomic precision medicine initiatives (e.g. M-PACT, NCI-MATCH) seek to use genetic sequencing-based treatment to improve response rates in select adult populations. However, the applicability of this technology in pediatric and AYA tumors has yet to be established. Methods: In an IRB-approved retrospective analysis of focused exome sequencing of 190 relapsed and high risk pediatric and AYA malignancies, 67 hematologic malignancies, 38 sarcomas, 34 neuroblastomas, 21 CNS, 7 liver, 6 renal, and 17 other tumors were analyzed. DNA was sequenced to high, uniform coverage (Illumina HiSeq) and analyzed for genomic alterations (GAs) (Foundation Medicine, Cambridge, MA). Results: Population included 156 patients with relapsed/refractory cancer and 34 with de novo high-risk disease (median age 7 yrs; range 3 mos to 34 yrs). GAs were identified in 82% of patients (median of...