Decitabine for the treatment of older patients with myelodysplastic syndrome and acute myelogenous leukemia

The azanucleoside analog decitabine inhibits DNA methyltransferases, leding to hypomethylation of DNA and altered gene expression. Phase II and III trials have reported the efficacy of decitabine in patients with high-risk myelodysplastic syndromes (MDS), including those with 20-30% bone-marrow blasts, who would now be classified as having acute myeloid leukemia (AML). These studies have used a variety of dosing schedules; however, only two schedules are currently US FDA approved for the treatment of MDS. Clinical trials have also explored the use of decitabine for the treatment of elderly patients with AML, with promising results. Current research focuses on establishing the activity of decitabine as a single agent in AML and combining decitabine with other active agents for the treatment of both MDS and AML.