Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial

Gotlib, Jason; Reiter, Andreas; Radia, Deepti; W. Deininger, Michael; I. George, Tracy; Panse, Jens; M. Vannucchi, Alessandro; Platzbecker, Uwe; Álvarez‐Twöse, Iván; Mital, Andrzej; Hermine, Olivier; Dybedal, Ingunn; O. Hexner, Elizabeth; K. Hicks, Lisa; F. R. Span, Lambert; A. Mesa, Ruben; Bose, Prithviraj; Pettit, Kristen; Heaney, Mark; T. Oh, Stephen; Sen, Jayita; Lin, Huimin; G. Mar, Brenton; J. DeAngelo, Daniel

doi:10.1038/s41591-021-01539-8

Public

Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial

Published in Nature Medicine • Dec 1, 2021

Authors:

Jason Gotlib

Andreas Reiter

Deepti Radia

Abstract

Abstract Advanced systemic mastocytosis (AdvSM) is a rare, KIT D816V-driven hematologic neoplasm characterized by mast cell infiltration and shortened survival. We report the results of a prespecified interim analysis of an ongoing pivotal single-arm phase 2 trial (no. NCT03580655 ) of avapritinib, ...

Subject

Systemic mastocytosis

Medicine

Clinical endpoint

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