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The Landscape of Pediatric Precision Oncology: Program Design, Actionable Alterations, and Clinical Trial Development.


ABSTRACT: Over the last years, various precision medicine programs have been developed for pediatric patients with high-risk, relapsed, or refractory malignancies, selecting patients for targeted treatment through comprehensive molecular profiling. In this review, we describe characteristics of these initiatives, demonstrating the feasibility and potential of molecular-driven precision medicine. Actionable events are identified in a significant subset of patients, although comparing results is complicated due to the lack of a standardized definition of actionable alterations and the different molecular profiling strategies used. The first biomarker-driven trials for childhood cancer have been initiated, but until now the effect of precision medicine on clinical outcome has only been reported for a s

SUBMITTER: Langenberg KPS 

PROVIDER: S-EPMC8431194 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature