Article
Response and progression on midostaurin in advanced systemic mastocytosis: KIT D816V and other molecular markers.
Blood - 13 Jul 2017
Jawhar Mohamad, Schwaab Juliana, Naumann Nicole, Horny Hans-Peter, Sotlar Karl, Haferlach Torsten, Metzgeroth Georgia, Fabarius Alice, Valent Peter, Hofmann Wolf-Karsten, Cross Nicholas C P, Meggendorfer Manja, Reiter Andreas
Abstract excerpt
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 biomarkers predictive of response are lacking. We...
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