European precision medicine program for recurrent malignancies in children and adolescents

Comprehensive molecular profiling of tumors can identify molecular targets and predictive biomarkers. The prospective MAPPYACTS study in four European countries included pediatric patients with recurrent or refractory malignancies. Molecular profiling of the tumor or cell free DNA resulted in detection of at least one actionable genetic alteration in 69% of patients. Of patients with follow-up beyond 12 months, 30% received one or more matched targeted therapies. Overall, matched treatment resulted in ORR 17%. In patients with treatments considered as ‘ready for routine use’ the ORR was 38%. In patients with extra-cerebral tumors, 76% of actionable alterations detected in tumor tissue were also identified in cfDNA.