Available and emerging therapies for bona fide advanced systemic mastocytosis and primary eosinophilic neoplasms.

ABSTRACT: The historically poor prognosis of patients with advanced systemic mastocytosis (AdvSM) and primary eosinophilic neoplasms has shifted to increasingly favorable outcomes with the discovery of druggable targets. The multikinase/KIT inhibitor midostaurin and the highly selective KIT D816V inhibitor avapritinib can elicit marked improvements in measures of mast cell (MC) burden as well as reversion of MC-mediated organ damage (C-findings) and disease symptoms. With avapritinib, the achievement of molecular remission of KIT D816V and improved survival compared with historical therapy suggests a potential to affect disease natural history. BLU-263 and bezuclastinib are KIT D816V inhibitors currently being tested in trials of AdvSM. In the new World Health Organization and International Consensu

SUBMITTER: Gotlib J 

PROVIDER: S-EPMC9821059 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature