What is the efficacy of azacitidine and decitabine for the treatment of myelodysplastic syndrome (MDS)?

Updated: Jul 24, 2018
  • Author: Emmanuel C Besa, MD; Chief Editor: Koyamangalath Krishnan, MD, FRCP, FACP  more...
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Azacitidine and decitabine are approved by the US Food and Drug Administration for treatment of all 5 MDS subtypes. They are considered standard therapy for both low-risk cases without 5q-, as well as intermediate and high-risk MDS. Although the two drugs were thought to be similar, only azacytidine has additional RNA and DNA activity compared with decitabine.

In a pivotal trial that included patients in all stages of MDS, patients treated with azacitidine showed a 37% response (7% complete response, 16% partial response) versus a 5% response in the control arm, with an improved median time to transformation or death (21 mo for azacitidine vs 13 mo for controls) and transformation to leukemia (15% for azacitidine vs 38% for controls). [30]

Compared with supportive care, both agents show an overall response (60% with azacytidine vs 5% with decitabine) and a longer time to progression to AML or death, and improvement of quality of life but no overall survival advantage. In a phase III trial involving 358 patients with an IPSS classification of intermediate-2 or high risk, treatment with azacytidine (75 mg/m2/d for 7 d q28d) significantly increased survival. At 2 years, 50.8% of patients in the azacitidine group were alive compared with 26.2% in the patients who received conventional care. [31]

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