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Avapritinib for the treatment of AdvSM: Latest updates from the PATHFINDER study

Jul 5, 2024
Learning objective: After reading this article, learners will be able to cite a new clinical development in advanced systemic mastocytosis.

During the European Hematology Association 2024 Hybrid Congress, Andreas Reiter shared 3-year follow up data from the phase II PATHFINDER study of avapritinib for the treatment of patients with advanced systemic mastocytosis (AdvSM)

Key learnings

The overall response and complete response rates were numerically higher in the treatment-naïve setting (87% and 43%, respectively) vs in patients with ≥1 prior systemic therapy (66% and 21%, respectively). 

Median overall survival (OS) was not reached, regardless of AdvSM subtype or treatment history. 

The 36-month OS rate was 75%, with more favorable data for the treatment-naïve vs pre-treated cohort.

No new safety concerns were identified during the follow up period, with most adverse events manageable with dose reduction or interruption.  

Data from PATHFINDER suggest avapritinib as a feasible treatment option in AdvSM, particularly in early lines of therapy. 

  1. Reiter A. Oral Abstract #S224. Avapritinib in patients with advanced systemic mastocytosis (AdvSM): efficacy and safety analysis from the phase 2 pathfinder study with 3-year follow-up. Presented at: EHA 2024 Hybrid Congress; Jun 15, 2024; Madrid, ES. 


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