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Expert Rev Hematol. 2016;9(4):377-88. doi: 10.1586/17474086.2016.1135047. Epub 2016 Feb 15.

New Insights into the Pathogenesis of MDS and the rational therapeutic opportunities.

Author information

1
a Division of Hematology Oncology , Mount Sinai Beth Israel , New York , NY , USA.
2
b Department of Internal Medicine , Mount Sinai Beth Israel , New York , NY , USA.
3
c Division of Hematology Oncology , Lancaster General Hospital , Lancaster , PA , USA.
4
d Division of Hematology, Department of Medicine , Yale University , New Haven , CT , USA.

Abstract

Myelodysplastic syndromes (MDS) include a heterogeneous group of acquired hematopoietic malignancies characterized by ineffective hematopoiesis, peripheral cytopenias, and a varying propensity for progression to acute myeloid leukemia. The clinical heterogeneity in MDS is a reflection of its molecular heterogeneity. Better understanding of aberrant epigenetics, dysregulation of immune responses, and del(5q) MDS has provided the rationale for well-established treatments in MDS. Further understanding of abnormal signal transduction and aberrant apoptosis pathways has led to development of new rational therapies that are in advanced phases of clinical translation. This review seeks to describe recent developments in our understanding of the pathogenesis of MDS and the potential therapeutic implications of these observations.

KEYWORDS:

DNMTI; Del5q; Epigenetics; Myelodysplastic syndrome; Pathogenesis; immune dysregulation

PMID:
26734762
DOI:
10.1586/17474086.2016.1135047
[Indexed for MEDLINE]
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