Event-free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children’s Oncology Group

Metts J, Xue W, Gao Z, Oberoi S, Weiss AR, Venkatramani R, Harrison DJ. Event-free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children’s Oncology Group. Pediatr Blood Cancer. 2024 Jul;71(7):e31009. doi: 10.1002/pbc.31009. Epub 2024 Apr 16. PMID: 38627882; PMCID: PMC11180298.

Novel therapies are needed for relapsed and refractory rhabdomyosarcoma (RRMS). Phase II clinical trials in RRMS have typically utilized radiologic response as the primary activity endpoint, an approach which poses several limitations in RRMS. In this analysis, we aimed to estimate an event-free survival (EFS) endpoint for RRMS that could be used as a benchmark for future studies.