Recent advances in understanding and managing pediatric rhabdomyosarcoma

Abstract

Rhabdomyosarcoma (RMS) is a high-grade malignant neoplasm, with a morphologic appearance mimicking that of developing skeletal muscle. Over the last 30 years, patient outcomes have improved with the incorporation of multimodal therapies, including chemotherapy, radiation therapy, and surgery. The overall cure rates exceed 70%, with patients who have low-, intermediate-, and high-risk disease experiencing long-term survival rates of >90%, 70%, and <30%, respectively. Historically, RMS was classified according to histology; however, recent advances have revealed new molecular subgroups that allow us to more accurately identify high-, intermediate-, and low-risk disease. In this review, we discuss recent advances made in understanding RMS tumor biology and propose how this understanding can drive a new classification system that can guide clinical approaches for treatment de-escalation in patients with expected favorable outcomes and escalation for those with expected poor outcomes.