Hypomethylating agents

Two randomized prospective clinical trials compared the hypomethylating agent, azacitidine, with best supportive care in patients with myelodysplastic syndromes (MDS). The trials involved large numbers of patients with MDS but also included small numbers of patients (fewer than 25) with CMML.[30,31] The overall response rates exceeded 60% for all patients who received azacitidine, but the data did not allow an assessment specifically for patients with CMML.[30,31][Level of evidence C3] Several phase II trials reported response rates of 30% to 60% for patients with CMML who received hypomethylating agents.[32-36] Azacitidine and decitabine may reverse cytopenias, cytoreduce elevated WBC counts, reduce splenic size, and improve clinical symptoms (like decreased appetite or itching).