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Blood Rev. 2012 Apr;26 Suppl 1:S12-5. doi: 10.1016/S0268-960X(12)70005-X.

The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia.

Author information

1
Weill Medical College of Cornell University, New York, NY, USA. str2010@med.cornell.edu

Abstract

Ineffective erythropoiesis is the hallmark of beta-thalassemia that triggers a cascade of compensatory mechanisms resulting in clinical sequelae such as erythroid marrow expansion, extramedullary hematopoiesis, splenomegaly, and increased gastrointestinal iron absorption. Recent studies have begun to shed light on the complex molecular mechanisms underlying ineffective erythropoiesis and the associated compensatory pathways; this new understanding may lead to the development of novel therapies. Increased or excessive activation of the Jak2/STAT5 pathway promotes unnecessary disproportionate proliferation of erythroid progenitors, while other factors suppress serum hepcidin levels leading to dysregulation of iron metabolism. Preclinical studies suggest that Jak inhibitors, hepcidin agonists, and exogenous transferrin may help to restore normal erythropoiesis and iron metabolism and reduce splenomegaly; however, further research is needed.

PMID:
22631035
PMCID:
PMC3697110
DOI:
10.1016/S0268-960X(12)70005-X
[Indexed for MEDLINE]
Free PMC Article

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