Avapritinib monotherapy induces rapid and deep remission of heavily treated, KIT D816H-mutated t(8;21) acute myeloid leukemia

Acute myeloid leukemia (AML) with t(8;21) is a subset of core binding factor AML and is considered to be favorable risk disease in patients receiving intensive cytarabine based chemotherapy. However, relapse remains a significant clinical challenge. Mutations in KIT , which frequently co-occur in t(8;21) AML, have been associated with worse relapse free and overall survival. Avapritinib is a novel tyrosine kinase inhibitor targeting KIT mutations that is currently approved for systemic mastocytosis but doesn’t currently have an established role in the treatment of AML. Here we report the case of a patient with heavily treated KIT D816H-mutated t(8;21) AML that relapsed following allogeneic hematopoietic stem cell transplant who received avapritinib monotherapy with rapid induction of a deep remission. This case highlights the potential role of avapritinib as a targeted therapy for relapsed t(8;21) AML with KIT mutations, warranting further clinical investigation.