Clinical Utility of Azacitidine in the Management of Acute Myeloid Leukemia: Update on Patient Selection and Reported Outcomes

Abstract

Acute myeloid leukemia (AML) is predominantly a disease of the elderly, and a significant proportion of these patients are not candidates for intensive, curative-intent therapies. Epigenetic dysregulation resulting in abnormal DNA hypermethylation is one of the hallmarks of AML pathogenesis. For the past two decades, hypomethylating agents including azacitidine (AZA) have been the mainstay of treatment for AML patients who are ineligible to receive intensive chemotherapies. As our understanding of AML disease biology has improved, several novel treatment combinations have been developed to improve the outcome of AML patients, with remarkable success. A considerable proportion of these novel combinations have utilized AZA as the backbone of their treatment scheme. In this review, we have highlighted the evolution of AML treatment, focusing on novel AZA-based treatment combinations and their clinical efficacy.