5-Azacytidine for the treatment of myelodysplastic syndromes

Janusz Krawczyk, Niamh Keane, Ciara Louise Freeman, Ronan Swords, Michael O'Dwyer, Francis J. Giles

Research output: Contribution to journalArticlepeer-review

11 Scopus citations


Introduction: 5-Azacytidine is a pyrimidine nucleoside analog of cytidine that undergoes incorporation into DNA and blocks DNA methyltransferase leading to hypomethylation and potentially beneficial re-expression of abnormally silenced genes. It is the first agent approved for use in patients with myelodysplastic syndromes (MDSs) based on its improvement in overall survival as monotherapy. Evidence of efficacy in combination with other agents is also accumulating. Areas covered: Key information on mechanisms of action is presented. Development, synthesis, and pharmacokinetics are also outlined. Key safety, tolerability, and efficacy data from clinical trials of 5-azacytidine as monotherapy as well as in combination are also presented. Expert opinion: Our understanding of the molecular basis and pathogenesis of MDS continues to evolve rapidly. 5-Azacytidine has been shown to improve both overall survival and quality of life in patients with high-risk MDS. Currently, the oral route of administration is undergoing evaluation in clinical trials. Used as a monotherapy and also in novel combinations, 5-azacytidine has the potential to further improve the prognosis of some patients with MDS.

Original languageEnglish (US)
Pages (from-to)1255-1268
Number of pages14
JournalExpert Opinion on Pharmacotherapy
Issue number9
StatePublished - Jun 2013
Externally publishedYes


  • 5-azacytidine
  • Epigenetic therapy
  • Histone deacetylase inhibitors
  • Hypomethylation
  • Myelodysplastic syndrome

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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