Epigenetic approaches in the treatment of myelodysplastic syndromes: clinical utility of azacitidine

Steven E McCormack, Erica D Warlick

Department of Medicine, Division of Hematology, Oncology, and Transplantation, University of Minnesota, Minneapolis, Minnesota, USA

Abstract: Myelodysplastic syndromes (MDS) are a varied group of diseases leading to ­significant morbidity and mortality. Therapy of MDS has been difficult, with supportive cares used to ameliorate symptoms, and hematopoietic stem cell transplantation the only curative option. Agents, such as the cytidine analog azacitidine, exert an effect on DNA methyltransferase leading to a reduction in DNA methylation, a process thought to be key to the pathogenesis of MDS. Recently, azacitidine has been shown to prolong survival and improve quality of life in patients with MDS, while maintaining a favorable adverse effect profile. This review highlights the scientific rationale for the use of azacitidine in addition to its application in current clinical practice for patients with MDS.

Keywords: hypomethylation, epigenetics, myelodysplastic syndromes, azacitidine