breakthroughTreatment update
Avapritinib FDA-approved for indolent systemic mastocytosis (PIONEER trial)
Systemic Mastocytosis →Summary
In May 2023, avapritinib became the first and only FDA-approved targeted therapy for ISM, based on PIONEER trial data showing significant symptom reduction and mast cell burden decrease vs placebo. This marks a paradigm shift from purely symptomatic management to targeted disease modification in non-advanced SM.
Source
Grade ARCT
Avapritinib versus Placebo in Indolent Systemic Mastocytosis
Gotlib J, Castells M, Engelbrecht ME, et al. · NEJM Evidence · 2023
- •PIONEER trial: avapritinib superior to placebo in ISM
- •TSS decrease: -15.6 vs -9.2 points (P<0.003)
- •54% achieved >=50% tryptase reduction vs 0% placebo
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ID: systemic-mastocytosis-update-0Type: treatment_updateImpact: breakthrough