Rhabdomyosarcoma (RMS): Insights into Epidemiology, Molecular Classification, Therapeutic Advances, and Future Directions
Abstract:
Rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in children, represents a formidable challenge in pediatric oncology. This review article delves into the multifaceted landscape of RMS, combining clinical insights with molecular intricacies to provide a comprehensive overview. RMS manifests in two major subtypes, alveolar RMS (ARMS) and embryonal RMS (ERMS), each propelled by distinct molecular mechanisms. While initial classification was based on histological features, fusion status now plays a pivotal role. Fusion-positive (FP) RMS, characterized by PAX3–FOXO1 or PAX7–FOXO1 translocations, presents unique clinical challenges, while fusion-negative (FN) RMS shares similarities with ERMS. Accurate risk stratification, encompassing clinical, pathological, and molecular attributes, is imperative in tailoring therapeutic strategies. Current treatment modalities encompass surgical resection, ionizing radiation, and cytotoxic chemotherapy. These multidimensional approaches have significantly improved survival rates over the past three decades. RMS continues to pose obstacles, particularly for patients with metastatic or recurrent disease. Treatment-related toxicities and long-term late effects further underscore the need for innovative therapeutic avenues and comprehensive survivorship care. This review article provides a detailed portrait of RMS, from epidemiology to molecular subtypes, risk stratification, and treatment modalities. As research unveils RMS's biological intricacies through next-generation sequencing and advanced disease models, the path toward improved clinical outcomes becomes increasingly discernible.
