Patients with myelodysplastic syndromes (MDS) are challenging to treat, given the advanced median age and comorbidities of the population. For most patients, the standard therapy is supportive care, including broad-spectrum antibiotics, red blood cell/platelet transfusions, and growth factors. Decitabine, a hypomethylating agent that allows for the re-expression of tumor suppressor genes, represents an exciting new treatment option for MDS patients. In phase 2 and 3 studies, decitabine has been associated with durable responses in MDS patients and delayed time to acute myeloid leukemia (AML) transformation or death compared with supportive care. Decitabine has been shown to be well tolerated with a toxicity profile expected for this class of agent. Recent studies also suggest that lower dose schedules of 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 and in other hematologic malignancies such as AML.
Keywords: DNA methyltransferase inhibitor; decitabine; gene silencing; hypomethylating agent; myelodysplastic syndromes.