{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T20:08:46Z","timestamp":1791576526897,"version":"4.3.4"},"reference-count":16,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2022,2,22]],"date-time":"2022-02-22T00:00:00Z","timestamp":1645488000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2022,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>We retrospectively examined our experience with midostaurin therapy in 33 consecutive patients (median age 68\u2009years; 58% females) with advanced systemic mastocytosis (adv\u2010SM): aggressive SM (ASM; <jats:italic>n<\/jats:italic>\u00a0=\u00a017), SM associated with another hematologic neoplasm (SM\u2010AHN; <jats:italic>n<\/jats:italic>\u00a0=\u00a014) and mast cell leukemia (MCL; <jats:italic>n<\/jats:italic>\u00a0=\u00a02). <jats:italic>KIT<\/jats:italic>D816V mutation was detected in 84% of the patients and C findings in 91%. Eleven (33%) patients were previously treated with other cytoreductive drugs, including cladribine (<jats:italic>n<\/jats:italic>\u00a0=\u00a04) and imatinib (<jats:italic>n<\/jats:italic>\u00a0=\u00a03). Median time from diagnosis to initiation of midostaurin therapy was 2.2\u2009months (range 0.3\u201341). Using modified valent criteria, overall response was 42% (53% ASM, 29% SM\u2010AHN, 50% MCL; <jats:italic>p<\/jats:italic>\u00a0=\u00a0.22), all classified as being major. Responses included \u226550% reduction in bone marrow mast cells in 40% and normalization of serum tryptase in 29%, of evaluated cases. After a median follow\u2010up of 14.6\u00a0months from initiation of midostaurin therapy, 7 (21%) deaths, 1 (3%) leukemic progression, and 18 (55%) treatment discontinuations were documented; median duration of midostaurin treatment was 7.9\u00a0months (range 0.5\u2013123) and response duration 21.5\u00a0months (range 2.9\u2013123). Most frequent side effect was gastrointestinal (51%) while grade 3\/4 neutropenia or thrombocytopenia occurred in 12% of patients. Response to treatment was not predicted by <jats:italic>KIT<\/jats:italic> mutation (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.67) or exposure to prior cytoreductive therapy (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.44). Median survival was longer in midostaurin responders but not significantly (median 26.5 vs. 16\u2009months; <jats:italic>p<\/jats:italic>\u00a0=\u00a0.15). Findings from the current study are broadly consistent with previously published clinical trial observations.<\/jats:p>","DOI":"10.1002\/ajh.26498","type":"journal-article","created":{"date-parts":[[2022,2,14]],"date-time":"2022-02-14T09:36:43Z","timestamp":1644831403000},"page":"630-637","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":20,"title":["Midostaurin therapy for advanced systemic mastocytosis: Mayo Clinic experience in 33 consecutive cases"],"prefix":"10.1002","volume":"97","author":[{"given":"Amritpal","family":"Singh","sequence":"first","affiliation":[{"name":"Division of Hematology Mayo Clinic  Rochester Minnesota USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aref","family":"Al\u2010Kali","sequence":"additional","affiliation":[{"name":"Division of Hematology Mayo Clinic  Rochester Minnesota 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