Decitabine (5-aza-2′-deoxycytidine) is a hypomethylating agent (HMA) used in the treatment of acute myeloid leukemia (AML)

Decitabine is indicated in the EU for the treatment of adult patients with newly diagnosed de novo or secondary AML who are not eligible for standard induction chemotherapy

In the registration trial, decitabine significantly improved overall survival, event-free survival, progression-free survival, and response rate compared with standard care, and was generally well tolerated

Similar results were seen in real-world studies

In contrast to standard chemotherapy, the presence of adverse-risk karyotypes or TP53 mutations does not negatively impact sensitivity to hypomethylating therapy

Future areas of research are necessary to identify biomarkers of response and resistance, and to investigate the use of decitabine-based combination to further improve outcomes in patients with AML