Utility of DNA Methyltransferase Inhibitors for the Treatment of Myelodysplastic Syndromes
Kaoru Tohyama
Abstract
Kaoru Tohyama
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
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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
Key concepts: Decitabine, Azacitidine, Myelodysplastic syndromes, DNA methyltransferase, Methyltransferase, Epigenetics, Medicine, DNA methylation