avapritinib + azacitidine + venetoclax

This is a combination therapy consisting of three small molecule drugs: avapritinib, azacitidine, and venetoclax. Avapritinib is a selective inhibitor of KIT and PDGFRA tyrosine kinases, primarily developed for the treatment of systemic mastocytosis and gastrointestinal stromal tumors with specific mutations. Azacitidine is a hypomethylating agent that incorporates into DNA and RNA, leading to DNA hypomethylation and cytotoxicity in abnormal hematopoietic cells; it is widely used in myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Venetoclax is a BCL-2 inhibitor that promotes apoptosis in cancer cells by disrupting anti-apoptotic signaling. The combination has been investigated as an induction regimen for relapsed acute myeloid leukemia (AML) after allogeneic hematopoietic stem cell transplantation, particularly in patients with RUNX1::RUNX1T1 or CBFB::MYH11 with C-KIT mutation[2][4]. This regimen leverages complementary mechanisms—targeted kinase inhibition (avapritinib), epigenetic modulation (azacitidine), and pro-apoptotic activity (venetoclax)—to enhance antileukemic efficacy.