Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
University Hospital Heidelberg, Department of Internal Medicine IV, Heidelberg, Germany.
Lancet Neurol. 2015 Jan;14(1):103-13. doi: 10.1016/S1474-4422(14)70190-5.
The copper metabolism disorder Wilson's disease was first defined in 1912. Wilson's disease can present with hepatic and neurological deficits, including dystonia and parkinsonism. Early-onset presentations in infancy and late-onset manifestations in adults older than 70 years of age are now well recognised. Direct genetic testing for ATP7B mutations are increasingly available to confirm the clinical diagnosis of Wilson's disease, and results from biochemical and genetic prevalence studies suggest that Wilson's disease might be much more common than previously estimated. Early diagnosis of Wilson's disease is crucial to ensure that patients can be started on adequate treatment, but uncertainty remains about the best possible choice of medication. Furthermore, Wilson's disease needs to be differentiated from other conditions that also present clinically with hepatolenticular degeneration or share biochemical abnormalities with Wilson's disease, such as reduced serum ceruloplasmin concentrations. Disordered copper metabolism is also associated with other neurological conditions, including a subtype of axonal neuropathy due to ATP7A mutations and the late-onset neurodegenerative disorders Alzheimer's disease and Parkinson's disease.
铜代谢紊乱所致威尔逊病于 1912 年首次被定义。威尔逊病可出现肝和神经功能缺损,包括肌张力障碍和帕金森病。现在已认识到婴儿期早发和 70 岁以上成年人晚发表现。ATP7B 基因突变的直接基因检测越来越多地用于确认威尔逊病的临床诊断,生化和遗传流行研究的结果表明,威尔逊病可能比以前估计的更为常见。早期诊断威尔逊病对于确保患者能够接受适当的治疗至关重要,但对于最佳药物选择仍存在不确定性。此外,威尔逊病需要与其他临床上也表现为肝豆状核变性或与威尔逊病共享生化异常的疾病相鉴别,例如血清铜蓝蛋白浓度降低。铜代谢紊乱也与其他神经疾病相关,包括由于 ATP7A 突变引起的轴索性神经病的一个亚型以及阿尔茨海默病和帕金森病等迟发性神经退行性疾病。