Comparative analysis between azacitidine and decitabine for the treatment of myelodysplastic syndromes

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

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74 Citations (Scopus)


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).

Original languageEnglish
Pages (from-to)339-347
Number of pages9
JournalBritish Journal of Haematology
Issue number3
Publication statusPublished - 2013 May

Bibliographical note

Funding Information:
The CUORE experiment (Cryogenic Underground Observatory for Rare Events, see [1, 2]) is a nuclear physics experiment funded by several agencies, among which the National Italian Institute of Nuclear Physics (INFN), the US Department of Energy (DOE) and the National Science Foundation (NSF, USA). The experiment is set up in the Gran Sasso National (Italian) Laboratory (LNGS), an underground facility in a highly seismic zone. In fact, the laboratory is located in a gallery beneath the Gran Sasso mountain, not far from the city of L’Aquila: that zone has been hit by a destructive earthquake in 2009 (MMS 6.3) and is currently involved in the earthquake swarm that follows the Amatrice earthquake (August 2016, MMS 6.2). In spite of the fact that the underground gallery seems to mitigate the seismic effects [3, 4], there is a major concern on the structural safety of all the experiments hosted by LNGS. The complexity of the structures, along with the strict requirements from the point of view of experimental physics, calls for the application of computational tools at the cutting-edge of technology.


  • Azacitidine
  • Comparison
  • Decitabine
  • Myelodysplastic syndromes

ASJC Scopus subject areas

  • Hematology


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