Multicenter Analysis of Genomically Targeted Single Patient Use Requests for Pediatric Neoplasms.
Himalee S SabnisDavid S ShulmanBenjamin E MizukawaNancy BouvierAhmet ZehirJason R FangusaroVanessa A FabrizioChanta WhitlowMarilyn WinchesterLaura AgrestaBrian TurpinDaniel S WechslerSteven G DuBoisJulia L Glade BenderSharon M CastellinoNeerav N ShuklaPublished in: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (2021)
SPU requests remain an important mechanism for pediatric access to genomically targeted agents given the limited availability of targeted clinical trials for children with high-risk neoplasms. Furthermore, this subset of SPUs resulted in a substantial number of objective tumor responses. The development of a multi-institutional data registry of SPUs may enable systematic review of toxicity and clinical outcomes and provide evidence-based access to new drugs in rare pediatric cancers.