Systemic therapy in metastatic pediatric rhabdomyosarcoma: a history of challenges and the search for promising approaches

Introduction: Metastatic rhabdomyosarcoma (RMS) is a rare and aggressive malignancy that arises from skeletal muscle precursors. It primarily affects children and adolescents. The prognosis for metastatic RMS is poor, making effective treatment strategies crucial. Chemotherapy serves as the backbone of the treatment regimen, aiming to control disease progression, shrink tumors, and improve survival outcomes. Areas covered: This paper explores the role of chemotherapy in managing metastatic RMS, examining standard therapeutic protocols, their efficacy, challenges, and future directions in treatment. Expert opinion: The treatment of metastatic RMS has evolved but remains challenging due to poor long-term survival rates, despite intensive chemotherapy and various optimization efforts. Emerging approaches, including targeted therapies, immunotherapy, and CAR-T cell therapy, offer hope, though ongoing research is crucial to improve outcomes and personalize treatment strategies.