An update on rhabdomyosarcoma risk stratification and the rationale for current and future Children’s Oncology Group clinical trials

Haduong JH, Heske CM, Allen-Rhoades W, Xue W, Teot LA, Rodeberg DA, Donaldson SS, Weiss A, Hawkins DS, Venkatramani R. An update on rhabdomyosarcoma risk stratification and the rationale for current and future Children’s Oncology Group clinical trials. Pediatr Blood Cancer. 2022 Apr;69(4):e29511. doi: 10.1002/pbc.29511. Epub 2022 Feb 7. Review. PubMed PMID: 35129294; PubMed Central PMCID: PMC8976559.

Children and adolescents with rhabdomyosarcoma (RMS) comprise a heterogeneous population with variable overall survival rates ranging between approximately 6% and 100% depending on defined risk factors. Although the risk stratification of patients has been refined across five decades of collaborative group studies, molecular prognostic biomarkers beyond FOXO1 fusion status have yet to be incorporated prospectively in upfront risk-based therapy assignments. This review describes the evolution of risk-based therapy and the current risk stratification, defines a new risk stratification incorporating novel biomarkers, and provides the rationale for the current and upcoming Children’s Oncology Group RMS studies.