Azacitidine: in myelodysplastic syndromes

Azacitidine, a pyrimidine analogue, is an antineoplastic agent that acts mainly by causing hypomethylation of cytosine residues in newly replicated DNA and has shown efficacy in the treatment of myelodysplatic syndromes (MDS). In a randomised controlled trial in patients with MDS (n=191), subcutaneous azacitidine 75-100 mg/m2/day in 7-day cycles every 28 days with continuing supportive care produced a significantly higher response rate (including reductions in rate of transformation to acute myeloid leukaemia and transfusion requirements) than that seen with supportive care alone (60% vs 5%; p<0.001). Patients (n=49) who were switched from supportive care to azacitidine after 4 months also showed a 47% response rate. The clinical response in patients receiving azacitidine was associated with significant (p