Tao Zhuang, Zhou Jiafeng, Jiang Zhenzhen, Hu Ya, Jia Shupei, Wang Meixia
Encephalopathy Center, The First Affiliated Hospital of Anhui University of Chinese Medicine, Hefei, China.
Graduate School, Anhui University of Chinese Medicine, Hefei, China.
Front Med (Lausanne). 2025 May 14;12:1599283. doi: 10.3389/fmed.2025.1599283. eCollection 2025.
Hepatolenticular Degeneration (HLD) is a rare condition caused by a genetic copper metabolism disorder and a basal ganglia-dominated degenerative brain disease. Its characteristic clinical features include progressive extrapyramidal symptoms, psychiatric manifestations, cirrhosis, renal impairment, and the Kayser-Fleischer ring. Furthermore, its key diagnostic bases include the ceruloplasmin level, copper oxidase activity, trace copper in the human body, brain Magnetic Resonance Imaging (MRI), and genetic testing. Here, we present an HLD case with atypical clinical manifestations. A 43-year-old male HLD patient presented to our hospital with normal copper oxidase activity and serum copper levels, as well as results of ceruloplasmin testing, slit-lamp examination, and histopathological examination of the liver, which showed no typical manifestations. On the other hand, the genetic testing results showed new mutation sites. To improve our clinical understanding of HLD and reduce the probability of misdiagnosis and missed diagnosis, we discussed and clarified the clinical manifestations, pathogenesis, and diagnosis and treatment of the disease, all based on existing literature.
肝豆状核变性(HLD)是一种由遗传性铜代谢紊乱引起的罕见疾病,是一种以基底节为主的退行性脑病。其特征性临床症状包括进行性锥体外系症状、精神症状、肝硬化、肾功能损害以及Kayser-Fleischer环。此外,其关键诊断依据包括铜蓝蛋白水平、铜氧化酶活性、人体微量铜、脑磁共振成像(MRI)和基因检测。在此,我们报告一例具有非典型临床表现的HLD病例。一名43岁男性HLD患者因铜氧化酶活性、血清铜水平正常,以及铜蓝蛋白检测、裂隙灯检查和肝脏组织病理学检查结果均无典型表现而前来我院就诊。另一方面,基因检测结果显示有新的突变位点。为提高我们对HLD的临床认识并降低误诊和漏诊的概率,我们根据现有文献对该疾病的临床表现、发病机制以及诊断和治疗进行了讨论和阐述。