Abstract

Knowledge of the basic mechanisms involved in iron metabolism has increased greatly in recent years, improving our ability to deal with the huge global public health problems of iron deficiency and overload. Several million people worldwide suffer iron overload with serious clinical implications. Iron overload has many different causes, both genetic and environmental. The two most common iron overload disorders are hereditary haemochromatosis and transfusional siderosis, which occurs in thalassaemias and other refractory anaemias. The two most important treatment options for iron overload are phlebotomy and chelation. Phlebotomy is the initial treatment of choice in haemochromatosis, while chelation is a mainstay in the treatment of transfusional siderosis. The classical iron chelator is deferoxamine (Desferal), but due to poor gastrointestinal absorption it has to be administered intravenously or subcutaneously, mostly on a daily basis. Thus, there is an obvious need to find and develop new effective iron chelators for oral use. In later years, particularly two such oral iron chelators have shown promise and have been approved for clinical use, namely deferiprone (Ferriprox) and deferasirox (Exjade). Combined subcutaneous (deferoxamine) and oral (deferiprone) treatment seems to hold particular promise.

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