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Master Thesis

Impact thérapeutique de la midostaurin dans la prise en charge des mastocytoses systémiques avancées

Abstract : Patients with advanced systemic mastocytosis have a poor prognosis and limited treatment options. The primary and the most common oncogenetic driver of systemic mastocytosis pathogenesis is D816V-mutated c-KIT tyrosine kinase receptor. Midostaurin bloked this cell pathway. We analyzed 46 patients, with a median age of 66 years (36 to 84 years) and an advanced systemic mastocytosis (ASM (n=17), ASM-AHD (n=25), et MCL (n=4)). 83% of patients were treated by Midostaurin in first line and 17% in second line at least. Midostaurin was given at 100 mg twice daily for at least 3 cycles of 28 days. We report an overall response rate of 80% (39% with partial remission and 41% with clinical remission). Median overall survival for the whole cohort was 69,3 months, 44,6 months for ASM, 80,4 months for ASM-AHD and 69,3 months for the 4 MCL. The most frequent grade 3/4 nonhematologic side effects were vomiting (17%), headache (11%), fatigue (9%) and constipation (9%). The most frequent grade 3/4 hematologic toxicitie was anemia (9%). These data illustrate the sustainable efficacy of Midostaurin in the therapeutic management of advanced systemic mastocytosis.
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Master Thesis
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Mathieu Bellal. Impact thérapeutique de la midostaurin dans la prise en charge des mastocytoses systémiques avancées. Médecine humaine et pathologie. 2019. ⟨dumas-02467506⟩



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