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The present study aimed to directly compare the efficacy and safety of azacitidine and decitabine in patients with myelodysplastic syndromes (MDS). We compared the overall response rate (ORR) (complete responses, partial responses, marrow complete responses, and haematological improvements), overall survival (OS), event-free survival (EFS), time to leukaemic transformation, and adverse outcomes between azacitidine and decitabine. To minimize the effects of treatment selection bias in this observational study, adjustments were made using the propensity-score matching method. Among 300 patients, 203 were treated with azacitidine and 97 with decitabine. Propensity-score matching yielded 97 patient pairs. In the propensity-matched cohort, there were no significant differences between the azacitidine and decitabine groups regarding ORR (44% vs. 52%), OS (26 vs. 22.9 months), EFS (7.7 vs. 7.0 months), and rate of leukaemic transformation (16% vs. 22% at 1 year). In patients ≥ 65 years of age, survival was significantly better in the azacitidine group (P = 0.017). Patients who received decitabine experienced more frequent episodes of grade 3 or 4 cytopenia and infectious episodes. We found that azacitidine and decitabine showed comparable efficacy. Among patients ≥ 65 years of age, survival was significantly better in the azacitidine group ( Identifier: NCT01409070). © 2013 Blackwell Publishing Ltd.


Yun-Gyoo Lee, Inho Kim, Sung-Soo Yoon, Seonyang Park, June Won Cheong, Yoo Hong Min, Jeong-Ok Lee, Soo-Mee Bang, Hyeon Gyu Yi, Chul Soo Kim, Yong Park, Byung-Soo Kim, Yeung-Chul Mun, Chu-Myoung Seong, Jinny Park, Jae Hoon Lee, Sung-Yong Kim, Hong Ghi Lee, Yeo-Kyeoung Kim, Hyeoung-Joon Kim, Korean Society of Haematology AML/MDS working party. Comparative analysis between azacitidine and decitabine for the treatment of myelodysplastic syndromes. British journal of haematology. 2013 May;161(3):339-47

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PMID: 23432512

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