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Myelodysplastic syndromes (MDS) are a heterogenous group of clonal stem cell disorders commonly characterized by
a hypercellular and dysplastic bone marrow, cytopenias resulting from impaired peripheral blood cell production, and an
increased risk of leukemic transformation. Currently, azacitidine, decitabine, and lenalidomide are approved by the US
Food and Drug Administration for the treatment of MDS. The DNA methyltransferase (DNMT) inhibitors such as
azacitidine and decitabine have demonstrated the ability to alter the natural history of disease and thus prolong time to
leukemic transformation. In addition, azacitidine has shown the capacity to extend survival compared with the previous
gold standard of conventional care regimens. Recently, decitabine has been shown to be well tolerated with a toxicity
profile expected for this class of agent. Recent studies also suggest that decitabine may result in additional improvements
in response. As more is learned about the mechanism of hypomethylating agents, new roles are emerging for decitabine
in combination therapy for MDS. The third agent, lenalidomide, is a thalidomide analogue with significant activity in a
subset of patients with low-risk MDS, anemia and chromosome 5 alterations. Several other agents are being evaluated in
MDS. This review summarizes the existing clinical experience on DNMT inhibitors and other drugs for the treatment of
MDS. (Korean J Med 76:121-125, 2009) |
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