Shimizu Norikazu
Department of Pediatrics, Toho University School of Medicine, Ohashi Medical Center.
Rinsho Shinkeigaku. 2019 Sep 25;59(9):565-569. doi: 10.5692/clinicalneurol.cn-001241. Epub 2019 Aug 30.
Wilson disease is an autosomal recessive disorder based on inborn error of copper metabolism. The copper accumulates in the liver, brain, cornea, kidney, and other organs. This disease should be considered any individual with liver abnormality except infant, any patient older than teenage with neurological (especially for extra pyramidal signs) or neuropsychiatric disorder with or without liver disease and sibling of Wilson disease patient. Typically, a combination of low serum ceruloplasmine levels and high levels of urinary copper contents is sufficient to establish a diagnosis. As other diagnostic tests, measurement of hepatic copper content and ATP7B gene analysis are available. The key strategy of treatment is to reduce the amount of copper in the liver and other tissues by administering both copper-chelating agents, such as D-penicillamine or Trientine, and/or zinc acetate. The author recommend zinc acetate monotherapy for mild to moderate hepatic disorder, Trientine mono therapy for mild to moderate neurologic disorder and combination therapy of Trientine and zinc acetate for sever hepatic or neurologic disorder.
威尔逊病是一种基于铜代谢先天性缺陷的常染色体隐性疾病。铜在肝脏、大脑、角膜、肾脏和其他器官中蓄积。对于任何有肝脏异常的个体(婴儿除外)、任何青少年以上患有神经疾病(尤其是锥体外系体征)或神经精神疾病且有或无肝脏疾病的患者以及威尔逊病患者的兄弟姐妹,均应考虑此病。通常,血清铜蓝蛋白水平低和尿铜含量高的组合足以确诊。作为其他诊断测试,可进行肝铜含量测定和ATP7B基因分析。治疗的关键策略是通过给予铜螯合剂(如D-青霉胺或曲恩汀)和/或醋酸锌来减少肝脏和其他组织中的铜含量。作者推荐对于轻度至中度肝脏疾病采用醋酸锌单一疗法,对于轻度至中度神经疾病采用曲恩汀单一疗法,对于严重肝脏或神经疾病采用曲恩汀和醋酸锌联合疗法。