Midostaurin in daily clinical practice of patients with advanced systemic mastocytosis.

ABSTRACT: Midostaurin, a multikinase/KIT inhibitor, is approved for the treatment of advanced systemic mastocytosis (AdvSM). We evaluated dosing regimens, safety, efficacy and overall survival (OS) of 79 patients from the 'German Registry on Disorders of Eosinophils and Mast Cells'. Midostaurin was initiated at 200 and ≤150 mg daily in 63/79 (80%) and 16/79 (20%) and continued at month 12 in 44/63 (70%) and 10/16 (63%) patients respectively. Over a cumulative observation period of 146 patient-years, 96 adverse events (AE) led to dose adjustment, most commonly nausea/emesis (n = 22, 23%) and neutropenia (n = 8, 8%). Responses were achieved in 60% (modified Valent criteria), 29% (IWG-MRT-ECNM, International Working Group-Myeloproliferative Neoplasms Research and Treatment & ECNM criteria) and 13% (pur

SUBMITTER: Lubke J 

PROVIDER: S-EPMC12512096 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature