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Developments in the treatment of transfusion-dependent anemia in patients with myelodysplastic syndromes: epidemiology, etiology, genetics, and targeted therapies

Authors Raza A, Iverson N, Ali AM

Received 25 March 2014

Accepted for publication 8 April 2014

Published 14 July 2014 Volume 2014:4 Pages 95—106

DOI https://doi.org/10.2147/AGG.S54184

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Azra Raza, Nicholas Iverson, Abdullah M Ali

The MDS Center, Columbia University, New York, NY, USA

Abstract: Myelodysplastic syndromes are malignant hematopoietic stem cell disorders that present with variable cytopenias and predominantly affect the elderly. Treatment options are limited, with allogeneic transplant being the only potentially curative strategy. Recent mutational profiling studies have led to cataloguing of driver and passenger mutations most commonly affecting the epigenetic regulators and genes involved in RNA splicing. Despite improved understanding of the disease biology, these emerging molecular insights have not led to identification of novel therapeutic strategies. Although several drugs approved in the last decade improve the cytopenias, the relief is temporary, most likely due to the sequential activation of clones. Future advances depend upon identification of signaling pathways in dominant clones and targeting these with agents that might be known but need to be matched to suit the needs of individual patients in a longitudinal, dynamic fashion. Myelodysplastic syndromes are ideally suited for the development of such personalized medicine.

Keywords: cancer, epigenetics, iron, MDS, myelodysplasia, splicing

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