ASH 2020: Treatment advances in mastocytosis

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07/09/23

Daniel DeAngelo, MD, PhD, Dana-Farber Cancer Institute, Boston, MA, outlines the latest investigations in the systemic mastocytosis (SM) field that will be presented at ASH 2020. Advanced SM, comprised of aggressive SM, SM with associated hematologic neoplasm and mast cell leukemia, is a rare group of mast cell neoplasms with poor survival outcomes and very limited treatment options. Avapritinib is a KIT D816V inhibitor, the primary driver of mast cell proliferation in mastocytosis. Data to be presented at ASH 2020 from a Phase I trial (NCT02561988) shows rapid responses and tolerability with avapritinib use in the advanced setting. Additionally, details of the Phase II PIONEER study (NCT03731260) will be shared, showing significant improvements in patient-reported outcomes in indolent systemic mastocytosis after avapritinib therapy, a setting where no treatments are currently approved. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.