decitabine + midostaurin

Decitabine + midostaurin is a combination of two small molecule drugs used in investigational regimens for the treatment of acute myeloid leukemia (AML), particularly in older or unfit patients. Decitabine is a hypomethylating agent that incorporates into DNA and inhibits DNA methyltransferases, leading to global DNA hypomethylation, reactivation of tumor suppressor genes, and decreased neoplastic cell proliferation. Midostaurin is a multikinase inhibitor targeting FLT3, KIT (CD117), PDGFR, VEGFR2, and protein kinase C alpha; it induces apoptosis in leukemia cells expressing these kinases. While both agents are approved individually for hematologic malignancies—decitabine for myelodysplastic syndromes and AML, and midostaurin (Rydapt) for FLT3-mutated AML—their combination has been studied in clinical trials but has not demonstrated clear additional benefit over monotherapy with decitabine in unfit AML patients[4][5][8]. The combination remains investigational.

Other names

5-aza-2'-deoxycytidinePKC412PKC-412PKC 412