Azacitidine in adult patients with acute myeloid leukemia

Azacitidine is recommended front-line treatment for older patients with acute myeloid leukemia (AML) who are not candidates for intensive treatment regimens, and was recently granted approval in the European Union for treatment of adult AML. Reviewed here is azacitidine experience in AML, including: mechanistic and pharmacokinetic data; safety and efficacy in controlled trials; treatment effects in AML subpopulations defined by disease characteristics; experience in unselected patients treated in the community setting; clinical outcomes relative to other approved AML therapies; and experience with azacitidine-based combination treatment regimens. Collectively, these data suggest that (a) azacitidine may prolong overall survival to a similar or greater extent than do other approved AML treatments, but with less toxicity, (b) azacitidine may be the preferred treatment option for older patients with unfavorable cytogenetics, and (c) experience and outcomes with azacitidine in the clinic are similar to those seen in clinical trials. Continued investigation of combination regimens on an azacitidine backbone is warranted.