5-Azacitidine for treating acute myelogenous leukemia

Introduction: 5-Azacytidine is a chemically synthesized nucleoside analogue that was manufactured in the 1960s. It is a DNA methyltransferase (DNMT) inhibitor that has in vitro and in vivo demethylating effects. On 19 May 2004, the US FDA approved azacitidine as injectable suspension for treatment of patients with myelodysplastic syndromes (MDS).Areas covered: Hypomethylating therapy has been increasingly used in place of standard intensive chemotherapy for the treatment of unfit elderly patients with acute myeloid leukemia (AML). In the USA, azacitidine and decitabine are the most commonly used low-intensity therapies. In this article, we review the current literature about azacitidine for the treatment of MDS, AML and in the stem cell transplant field.Expert opinion: Azacitidine is an old/new drug that gained more attention after its FDA approval for MDS. It is the only drug that showed survival benefit in MDS, in a Phase III randomized controlled trial. Azacitidine also has an important role in treating relapsed AML and MDS post-allogeneic hematopoietic stem cell transplantation. Combination therapy is promising, especially with the availability of low toxicity targeted therapies, but trials in this setting are missing and much needed to provide a tolerable, effective and personalized strategy for these patients in great need for such therapy.