Oral decitabine in acute myeloid leukemia: assessing efficacy, safety, and future implications for older patients

Older patients with acute myeloid leukemia (AML) are often unsuitable for standard treatments and traditionally have a dismal prognosis. For 20 years, hypomethylating agents (HMAs), as single agents and recently as a backbone with venetoclax, have been used in this setting. The oral combination of decitabine and cedazuridine (C-DEC), which is therapeutically and pharmacologically equivalent to the intravenous (IV) formulation (IV-DEC), has expanded the therapeutic arsenal for AML, allowing for better convenience of administration. This review provides an overview of C-DEC, current clinical applications, and ongoing clinical studies, highlighting its potential role in managing AML in older patients.

For the aim of this review, the authors searched the current English literature on C-DEC in AML patients through PubMed, using several pertinent keywords. To summarize the most recent developments as up-to-date as possible, we considered, with a few exceptions, only papers published over the last three years until March 2025.

Compared to IV-DEC, C-DEC provides similar therapeutic exposure and efficacy with an optional safety profile, enhanced treatment adherence, reduced treatment burden, and required healthcare facilities compared to those associated with IV-DEC, providing a precious tool to manage older and unfit AML patients.