The hematologic neoplasm systemic mastocytosis is marked by the clonal proliferation of mast cells in the skin, bone marrow, gastrointestinal tract, spleen, and/or liver, caused by an activating KIT mutation in the KIT gene.
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Three-quarters of patients with the rare blood disorder advanced systemic mastocytosis responded to treatment with avapritinib (Ayvakit), an interim analysis of a phase II trial found.
Among 32 patients treated with daily avapritinib in the PATHFINDER study, 19% had a complete remission (CR) with partial hematologic recovery, 31% had a partial response, and 25% achieved stable disease, with responses observed in all disease subtypes, reported Daniel DeAngelo, MD, PhD, of the Dana-Farber Cancer Institute in Boston. Avapritinib at a starting dose of 200 mg induced rapid, durable, and improving responses, DeAngelo said during his presentation at the American Association for Cancer Research virtual meeting. Reductions were seen in disease burden based on bone marrow mast cells, reduction in serum tryptase,