5-Azacytidine for the treatment of myelodysplastic syndromes

Janusz Krawczyk, Niamh Keane, Ciara Louise Freeman, Ronan T Swords, Michael O'Dwyer, Francis J. Giles

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

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
Pages (from-to)1255-1268
Number of pages14
JournalExpert Opinion on Pharmacotherapy
Volume14
Issue number9
DOIs
StatePublished - Jun 1 2013

Fingerprint

Azacitidine
Myelodysplastic Syndromes
Clinical Trials
Pyrimidine Nucleosides
Cytidine
Survival
DNA
Expert Testimony
Methyltransferases
Therapeutics
Oral Administration
Pharmacokinetics
Quality of Life
Safety
Genes

Keywords

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

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Pharmacology

Cite this

Krawczyk, J., Keane, N., Freeman, C. L., Swords, R. T., O'Dwyer, M., & Giles, F. J. (2013). 5-Azacytidine for the treatment of myelodysplastic syndromes. Expert Opinion on Pharmacotherapy, 14(9), 1255-1268. https://doi.org/10.1517/14656566.2013.794222

5-Azacytidine for the treatment of myelodysplastic syndromes. / Krawczyk, Janusz; Keane, Niamh; Freeman, Ciara Louise; Swords, Ronan T; O'Dwyer, Michael; Giles, Francis J.

In: Expert Opinion on Pharmacotherapy, Vol. 14, No. 9, 01.06.2013, p. 1255-1268.

Research output: Contribution to journalArticle

Krawczyk, J, Keane, N, Freeman, CL, Swords, RT, O'Dwyer, M & Giles, FJ 2013, '5-Azacytidine for the treatment of myelodysplastic syndromes', Expert Opinion on Pharmacotherapy, vol. 14, no. 9, pp. 1255-1268. https://doi.org/10.1517/14656566.2013.794222
Krawczyk J, Keane N, Freeman CL, Swords RT, O'Dwyer M, Giles FJ. 5-Azacytidine for the treatment of myelodysplastic syndromes. Expert Opinion on Pharmacotherapy. 2013 Jun 1;14(9):1255-1268. https://doi.org/10.1517/14656566.2013.794222
Krawczyk, Janusz ; Keane, Niamh ; Freeman, Ciara Louise ; Swords, Ronan T ; O'Dwyer, Michael ; Giles, Francis J. / 5-Azacytidine for the treatment of myelodysplastic syndromes. In: Expert Opinion on Pharmacotherapy. 2013 ; Vol. 14, No. 9. pp. 1255-1268.
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