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Opinion|Videos|May 12, 2025

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

Author(s)Johannes Lübke, MD

Long-term data show avapritinib cuts systemic mastocytosis burden, delivers lasting responses, manageable safety, and boosts bone health markers.

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.

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