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血色素沉着症和威尔逊氏病的当前及未来治疗方法

Current and future therapy in haemochromatosis and Wilson's disease.

作者信息

Murray Karen F, Lam Dickson, Kowdley Kris V

机构信息

Hepatobiliary Program, Childrens Hospital and Regional Medical Center, and University of Washington, Seattle, WA 98195, USA.

出版信息

Expert Opin Pharmacother. 2003 Dec;4(12):2239-51. doi: 10.1517/14656566.4.12.2239.

Abstract

There have been several new developments in the treatment of iron and copper overload disorders, such as haemochromatosis, thalassaemia and Wilson's disease. Clinical trials of orally administered iron chelators, both as monotherapy and in combination with deferoxamine, are in progress around the world. Several new chelators are now being introduced in clinical trials. Future therapies for iron overload may comprise of oral iron binding agents capable of preventing dietary iron absorption from the diet. The characterisation of specific iron transporters such as the divalent metallic transporter and ferroportin may hold promise for the development of 'smart' compounds capable of blocking iron transport. Several new agents are now available for the management of Wilson's disease, including trientine, zinc and tetrathiomolybdate. This review, will discuss the pathogenesis, and current and future therapies for iron and copper overload disorders.

摘要

在铁和铜过载疾病(如血色素沉着症、地中海贫血和威尔逊氏病)的治疗方面有了一些新进展。口服铁螯合剂作为单一疗法以及与去铁胺联合使用的临床试验正在全球范围内进行。几种新型螯合剂目前正在临床试验中推出。未来铁过载的治疗可能包括能够阻止饮食中铁吸收的口服铁结合剂。特定铁转运蛋白(如二价金属转运蛋白和铁转运蛋白)的特性可能为开发能够阻断铁转运的“智能”化合物带来希望。目前有几种新药可用于治疗威尔逊氏病,包括曲恩汀、锌和四硫钼酸盐。本综述将讨论铁和铜过载疾病的发病机制以及当前和未来的治疗方法。

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