Purpose of review: The purpose of this review is to highlight some of the advances in the way we think about rhabdomyosarcoma (RMS). Recent outcome and biological analyses have shifted the risk stratification and treatment paradigms for pediatric RMS.
Recent findings: The presence or absence of the FOXO1 translocation is one of the most important prognostic factors in RMS. Future clinical studies will incorporate FOXO1 translocation status within risk stratification criteria. Molecular analyses have identified RAS/NF1, hedgehog, IL-4R, and ALK pathway abnormalities as potential therapeutic targets in RMS. Reductions in systemic therapy are possible, although radiation therapy remains essential to prevent local failures in most patients.
Summary: Although survival for RMS has not improved in recent years, refinement in risk stratification, further understanding of the biological drivers of the disease, and modifications in treatment intensity have set the stage for the next generation of studies in RMS.