Iron age: novel targets for iron overload.

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.

• Authors: Carla Casu, Stefano Rivella
• Journal: Hematology. American Society of Hematology. Education Program (Hematology Am Soc Hematol Educ Program) Vol. 2014 Issue 1 Pg. 216-21 (Dec 05 2014) ISSN: 1520-4383 [Electronic] United States
• PMID: 25696858 (Publication Type: Journal Article, Review)
• Copyright: © 2014 by The American Society of Hematology. All rights reserved.

Chemical References

• Iron

Topics

• Absorption, Physiological
• Animals
• Disease Models, Animal
• Erythropoiesis
• Humans
• Iron (metabolism)
• Iron Overload (metabolism)
• Models, Biological