Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #36

Abstract: The incorporation of genomic profiling into the care of pediatric, adolescent and young adult (AYA) cancer patients has resulted in identification of targetable alterations and use of molecularly targeted therapy (MTT), necessitating collection and reporting of outcomes of real-world use of MTT. Using genomic data contributed to the CCDI and our institutional cohorts, we identified a cohort of patients who received MTT outside of clinical trials. We included patients with cancer seen at the Dana-Farber/Boston Children’s, who enrolled in a clinical sequencing/banking study and had next-generation sequencing (NGS) performed (OncoPanel, Rapid Heme and Fusion Panels). Sequencing results were used to identify patients with potentially targetable alterations in a list of 66 genes. Patients who received MTT were identified, and data including dosing, toxicity and response were extracted from the medical record. Between 2013 and 2024, 2163 patients had tumor profiling via targeted NGS. 35% (760/2163) had an actionable alteration detected for which MTT could have been administered. 114 patients received at least one regimen that included an MTT (72 patients with brain tumors, 28 with solid tumors, 14 with hematologic malignancies). Alterations in BRAF, NF1, ALK, FLT3, and PIK3CA led to the most MTT use. Of the 114 patients who received MTT, 79% (90/114) received at least one targeted therapy regimen outside of a clinical trial. This project will create a registry of MTT use within the CCDI that can be expanded to include additional patients and be a resource for MTT use for the pediatric and AYA cancer community.