Advancing Sarcoma Care: Emerging Data and Evolving Treatment Strategies in Desmoid Tumors

Dr. Mrinal Gounder led a comprehensive discussion with Drs. Atrayee Basu Mallick and Nam Bui on evolving treatment strategies for desmoid tumors. The program highlighted the paradigm shift from surgical intervention to multimodal approaches emphasizing active surveillance and systemic therapies. Key topics included patient selection criteria for watchful waiting versus treatment initiation, considering factors such as tumor location, size, symptoms, and growth trajectory. High-risk locations include head/neck, mesenteric areas, and proximity to neurovascular structures. The discussion emphasized moving away from surgery due to high recurrence rates and significant morbidity, particularly in challenging anatomical locations. The RINGSIDE trial presented at ASCO 2026 demonstrated impressive efficacy for varagesestat, a once-daily gamma-secretase inhibitor, with an 84% reduction in progression risk and rapid pain relief within 4 weeks. This adds to the therapeutic armamentarium alongside nirogacestat, the first FDA-approved treatment. Unmet needs include determining optimal treatment duration, understanding mechanisms of spontaneous regression in 20% of patients, managing fertility concerns in predominantly young female populations, and developing predictive biomarkers for treatment selection.