Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children and young adults representing 5% of all childhood cancers. There are approximately 350 new cases per year in the United States. RMS has a bimodal age of presentation, with a younger peak between 2 to 6 years of age and an older peak at 10 to 18 years1, 2, 3, 4. A small subset of RMS are associated with cancer predisposition syndromes, including Li- Fraumeni syndrome and neurofibromatosis type 1. Embryonal RMS of the genitourinary tract can also be associated with DICER1 germline mutations5, 6, 7, 8, 9.

RMS has traditionally been classified into embryonal (ERMS) and alveolar (ARMS) histology, with ERMS being more predominant in younger children as well as head and neck and genitourinary locations. ARMS occurs more commonly in older children and extremities, trunk, perineum/perianal locations10,11. However, classification has transitioned away from histology and instead focuses on fusion status. PAX-FOXO1 fusion is a critical prognostic biomarker second only to the presence of metastatic disease. Approximately 80% of ARMS carry a FOXO1 fusion, whereas >95% of ERMS do not express the FOXO1 fusion protein12,13. This FOXO1 fusion gene is a potent prognostic factor for RMS and has replaced histology in current risk stratification guidelines14,15.

Treatment for RMS is always multimodal, including chemotherapy and local control with surgical resection and/or radiation therapy. The choice of local treatment is dependent on many factors including tumor location, type, and size, proximity to critical structures, patient age, tumor biology, response to neoadjuvant chemotherapy and surgical expertise. Since RMS is of mesenchymal origin, it can arise from nearly any anatomic site. This creates unique challenges in determining the best local control and regional lymph node evaluation strategies and requires multidisciplinary decision-making. Each tumor site has unique nuances associated with differences in anatomy, biology, and clinical behavior.

This article discusses the general principles of surgical management of rhabdomyosarcoma for the most common RMS cared for by the pediatric surgeon including surgical decision making and technical aspects.