Avapritinib Treatment of Patients with Advanced Systemic Mastocytosis: 4-Year Safety, Effect on Bone and First-Line Efficacy Results of the PATHFINDER Clinical Study

Avapritinib demonstrated durable clinical activity in advanced systemic mastocytosis, with sustained reductions in disease burden and consistent responses across subtypes over long-term follow-up. Treatment was generally well tolerated, with a safety profile aligned with prior experience and manageable adverse events. Improvements in bone health markers and density suggest a potential disease-modifying effect beyond hematologic control.