ASH 2020 | Avapritinib for indolent systemic mastocytosis in the PIONEER trial
Cem Akin, MD, PhD, University of Michigan, Ann Arbor, MI, discusses the ongoing Phase II PIONEER Trial (NCT03731260) of avapritinib, a selective tyrosine kinase inhibitor of activated KIT and PDGFRA, in patients with indolent systemic mastocytosis. Part 1 of the trial to determine the recommended dose showed that avapritinib reduced the total symptoms score (TSS), allelic fraction of the KIT mutation, bone marrow mast cell burden, and tryptase levels. Additionally, avapritinib showed a good safety profile with no serious grade ≥4 adverse events. The most common adverse events were mild fluid retention, nausea, diarrhea, and headaches. Part 2 of the study is now open for patient enrolment, which will randomize patients to receive 25mg of avapritinib or placebo for 6 months for safety and efficacy evaluation. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.
Disclosures
Research support: Blueprint Medicines
Honoraria: Blueprint Medicines, Novartis
