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Drugs. 2005;65(13):1781-9; discussion 1790-1.

Azacitidine: in myelodysplastic syndromes.

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  • 1Adis International Limited, Auckland, New Zealand. demail@adis.co.nz


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<or=0.015) improvements in several measures of health-related quality of life, including those assessing fatigue and physical functioning, compared with those in supportive care recipients. Given the grim prognosis of MDS patients, azacitidine was generally well tolerated, with common, but transient, myelotoxicity. Adverse events did not increase in severity or frequency during the course of the treatment.

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