Department of Chemistry, Norwegian University of Science and Technology, NO-7491 Trondheim, Norway.
J Trace Elem Med Biol. 2012 Jun;26(2-3):127-30. doi: 10.1016/j.jtemb.2012.03.009. Epub 2012 May 5.
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.
近年来，人们对铁代谢相关基本机制的认识有了很大的提高，这提高了我们应对缺铁和铁过载这两个巨大全球公共卫生问题的能力。全世界有数百万人患有具有严重临床意义的铁过载。铁过载有许多不同的原因，包括遗传和环境因素。两种最常见的铁过载疾病是遗传性血色素沉着症和输血性血色素沉着症，分别发生于地中海贫血症和其他难治性贫血症。铁过载的两种最重要的治疗选择是放血和螯合。放血是血色素沉着症的初始治疗选择，而螯合是输血性血色素沉着症治疗的主要方法。经典的铁螯合剂是去铁胺（地拉罗司），但由于胃肠道吸收不良，它必须静脉或皮下给药，主要是每天给药。因此，显然需要寻找和开发新的有效口服铁螯合剂。近年来，特别是两种这样的口服铁螯合剂显示出了希望，并已被批准用于临床，即地拉罗司（去铁酮）和去铁斯罗（曲恩汀）。联合皮下（去铁胺）和口服（去铁酮）治疗似乎具有特别的前景。
