Deacetylase inhibitors for the treatment of myelodysplastic syndromes

Abstract

Myelodysplastic syndromes (MDS) are a diverse group of myeloid disorders, with patients being at risk for cytopenias or progression to acute myeloid leukemia. Several classification and prognostic scoring systems have been developed. High-intensity treatments are not appropriate for all patients. Two demethylating agents, azacitidine and decitabine, are approved for the treatment of MDS, although many patients do not derive long-term benefit and eventually progress. Deacetylase inhibitors have emerged as novel treatment candidates based on mechanistic rationale and preliminary data. This article reviews existing data on MDS treatment and discusses the rationale and potential for combination with deacetylase inhibitors.

Keywords::

• Deacetylase inhibitor
• myelodysplastic syndromes
• acute myeloid leukemia

Acknowledgements

Financial support for medical, editorial and graphic design assistance was provided by Novartis Pharmaceuticals.

We thank William Fazzone, PhD, Andrea Hammons, PhD and Amanda Kauffman, PhD for their assistance with this manuscript.

Potential conflict of interest

Disclosure forms provided by the authors are available with the full text of this article at www.informahealthcare.com/lal.