Correlates of organ damage in patients with advanced systemic mastocytosis enrolled in clinical trials of avapritinib.

ABSTRACT: Organ damage in patients with advanced systemic mastocytosis (AdvSM) at the time of enrollment in clinical trials of KIT inhibitors has not been well characterized. We describe the spectrum and clinicopathologic correlates of organ damage as defined by World Health Organization (WHO) and modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG) criteria among patients with AdvSM enrolled in the phase 1 and 2 trials of the KIT D816V inhibitor avapritinib (n = 174; systemic mastocytosis [SM] with an associated hematologic neoplasm [SM-AHN], 68%; aggressive SM and mast cell leukemia [MCL], both 16%). Notably, 47% of patients received previous midostaurin. Patients with SM-AHN had the highest absolute

SUBMITTER: Liang EC 

PROVIDER: S-EPMC12711656 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature