Response and progression on midostaurin in advanced systemic mastocytosis: KIT D816V and other molecular markers.

Abstract

In advanced systemic mastocytosis (advSM), disease evolution is often triggered by KIT mutations (D816V in >80% of cases) and by additional mutations (eg, in SRSF2, ASXL1, and/or RUNX1 [S/A/Rpos in >60% of cases]). In a recently reported phase 2 study, midostaurin, a multikinase/KIT inhibitor, demonstrated an overall response rate (ORR) of 60% in advSM but… (More)
DOI: 10.1182/blood-2017-01-764423

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