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Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):216-21. doi: 10.1182/asheducation-2014.1.216. Epub 2014 Nov 18.

Iron age: novel targets for iron overload.

Author information

1
Department of Pediatrics, Division of Hematology-Oncology, and.
2
Department of Pediatrics, Division of Hematology-Oncology, and Department of Cell and Developmental Biology, Weill Medical College, Cornell University, New York, NY.

Abstract

Excess iron deposition in vital organs is the main cause of morbidity and mortality in patients affected by β-thalassemia and hereditary hemochromatosis. In both disorders, inappropriately low levels of the liver hormone hepcidin are responsible for the increased iron absorption, leading to toxic iron accumulation in many organs. Several studies have shown that targeting iron absorption could be beneficial in reducing or preventing iron overload in these 2 disorders, with promising preclinical data. New approaches target Tmprss6, the main suppressor of hepcidin expression, or use minihepcidins, small peptide hepcidin agonists. Additional strategies in β-thalassemia are showing beneficial effects in ameliorating ineffective erythropoiesis and anemia. Due to the suppressive nature of the erythropoiesis on hepcidin expression, these approaches are also showing beneficial effects on iron metabolism. The goal of this review is to discuss the major factors controlling iron metabolism and erythropoiesis and to discuss potential novel therapeutic approaches to reduce or prevent iron overload in these 2 disorders and ameliorate anemia in β-thalassemia.

PMID:
25696858
PMCID:
PMC5292264
DOI:
10.1182/asheducation-2014.1.216
[Indexed for MEDLINE]
Free PMC Article

Conflict of interest statement

Conflict-of-interest disclosures: S.S. has received research funding from Bayer Pharmaceuticals, Isis Pharmaceuticals, and Merganser Pharmaceuticals; has consulted for Bayer Pharmaceuticals and Isis Pharmaceuticals; and has equity ownership in Merganser Pharmaceuticals. C.C. has received research funding from Isis Pharmaceuticals and Merganser Biotech LLC. Off-label drug use: None disclosed.

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