Abstract

Unprecedented progress continues to be made in the treatment strategies for the myelodysplastic syndromes (MDS). This review provides an introduction to DNA methyltransferase inhibitors. These agents include 5-azacytidine (azacitidine; AZA) and 5-aza-2’- deoxycytidine (decitabine; DAC). In particular, AZA was shown for the first time to significantly prolong the life expectancy of high-risk MDS patients in international multicenter clinical studies. The selection of responders and assessment of the long-term efficacy warrant further study.

Keywords: MDS, DNA hypomethylation, epigenetic therapy, DNA methyltransferase inhibitors, azacitidine, decitabine, long-term efficacy, hematopoietic stem cell disorders, solid tumors, cytotoxic