Wilson病治疗研究现状*

doi:10.3969/j.issn.1672-5069.2021.06.041

摘要/Abstract

摘要： Wilson病(Wilson's disease,WD)是以铜代谢异常为病变特点的常染色体隐性遗传病,可引起病变器官,主要是肝脏和大脑,的继发性损伤。目前,主要的治疗方法是应用调节体内铜负平衡的药物,如螯合剂和锌盐等,具体药物的选择需根据患者的临床表现、对药物的耐受性、医师用药经验、药物成本和可及性等进行选择。由于WD的药物治疗需终身维持,且存在副作用和依从性问题,药物治疗具有一定的局限性。肝源缺乏、费用高昂和移植后需终身免疫抑制剂治疗等因素使肝移植治疗的应用也受到限制。所以,新型治疗药物或方法,特别是安全性更好和能够永久治愈的方法,引起了越来越多的关注。本文对WD的治疗研究现状进行了系统综述。

关键词: Wilson病, 药物治疗, 神经系统症状, 细胞移植, 基因治疗

Abstract: Wilson's disease (WD) is an autosomal recessive genetic disease characterized by abnormal copper metabolism, which can cause secondary damage to pathological organs, mainly liver and brain. The current basic treatment for patients with WD is based on medicines that increase excretion of copper from the body,such as chelators or zinc salts, etc. The choices of these agents should be based on patients' clinical manifestations and tolerance to them owing to side effects, physicians' experience and the cost and availability of them. The treatment of WD remains challenging for its side effects, compliance problems and life-long treatment. The shortage of donor liver, high cost, and the need for sustained immunosuppressive therapy after operation restrict the application of liver transplantation. Therefore, new treatment or methods, especially ones with better safety and permanent cure, are also attracting more and more attention. Therefore, this article systematically reviews the current research progress in treatment of patients with WD.

Key words: Wilson disease, Therapy, Nervous system symptoms, Cell transplantation, Gene therapy

梁晨, 郑素军. Wilson病治疗研究现状^*[J]. 实用肝脏病杂志, 2021, 24(6): 930-933.

Liang Chen, Zheng Sujun. Highlights of treatments for patients with Wilson's disease[J]. Journal of Practical Hepatology, 2021, 24(6): 930-933.

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Wilson病治疗研究现状^*

Highlights of treatments for patients with Wilson's disease

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