Efficacy and safety of midostaurin in advanced systemic mastocytosis
The multi-kinase inhibitor midostaurin shrank the mast cell burden and reversed organ damage in six in ten patients with advanced systemic mastocytosis, becoming the first approved targeted drug for the disease.
Overview
Open-label phase 2 study of 116 patients with advanced systemic mastocytosis (aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm, or mast cell leukaemia) treated with midostaurin 100 mg twice daily; 89 were evaluable for response.
The overall response rate was 60 percent with major responses in 45 percent, median overall survival 28.7 months and median progression-free survival 14.1 months; responses occurred regardless of KIT D816V status. Nausea, vomiting and cytopenias were the main toxicities. The FDA and EMA approved midostaurin for advanced systemic mastocytosis in 2017.
- Overall response 60 percent, major response 45 percent.
- Median overall survival 28.7 months; median progression-free survival 14.1 months.
Midostaurin was the first effective targeted therapy for advanced systemic mastocytosis and remains an option, particularly where avapritinib is unsuitable or unavailable.
- Single-arm study with response criteria that predate the current consensus.
- Gastrointestinal toxicity limits tolerance in many patients.
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