{"entity":{"id":"paper-gotlib-midostaurin-advanced-systemic-mastocytosis-nejm-2016","kind":"paper","name":"Efficacy and safety of midostaurin in advanced systemic mastocytosis","aka":[],"tldr":"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.","summary":"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.\n\nThe 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.","asOf":"2026-09-18","links":[{"label":"N Engl J Med 2016","url":"https://doi.org/10.1056/NEJMoa1513098"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/27355533/"}],"tags":[],"related":[],"cancers":["advanced-systemic-mastocytosis"],"sections":[],"technologies":[],"targets":[],"drugs":["midostaurin"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["nejm"],"dependsOn":[],"notes":[],"journal":"New England Journal of Medicine","year":2016,"doi":"10.1056/NEJMoa1513098","pmid":"27355533","authors":"Gotlib J, Kluin-Nelemans HC, George TI, et al.","paperType":"observational","findings":["Overall response 60 percent, major response 45 percent.","Median overall survival 28.7 months; median progression-free survival 14.1 months."],"whatItMeans":"Midostaurin was the first effective targeted therapy for advanced systemic mastocytosis and remains an option, particularly where avapritinib is unsuitable or unavailable.","caveats":["Single-arm study with response criteria that predate the current consensus.","Gastrointestinal toxicity limits tolerance in many patients."],"changedPractice":true,"participants":116},"route":"/key-papers/paper-gotlib-midostaurin-advanced-systemic-mastocytosis-nejm-2016/","neighbours":{"cancer":[{"id":"advanced-systemic-mastocytosis","kind":"cancer","name":"Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia)","route":"/cancers/advanced-systemic-mastocytosis/"}],"drug":[{"id":"midostaurin","kind":"drug","name":"Midostaurin","route":"/drugs/midostaurin/"}],"journal":[{"id":"nejm","kind":"journal","name":"New England Journal of Medicine","route":"/journals/nejm/"}]}}