Medicine, Maulana Azad Medical College, New Delhi, Delhi, India.
Medicine, Maulana Azad Medical College, New Delhi, Delhi, India
BMJ Case Rep. 2024 Jul 29;17(7):e259598. doi: 10.1136/bcr-2023-259598.
A female adolescent presented with a 9 month history of progressive involuntary movements, initially manifesting as finger tremors and evolving into flinging motions of the extremities, resulting in an inability to walk over the last 4 months. Concurrently, she developed dysarthria. Neurologically, she exhibited normal power, rigidity and brisk deep tendon reflexes, with a downgoing plantar reflex. Contrast-enhanced MRI revealed hyperintensity in bilateral caudate lobes, basal ganglia and pons, indicative of Wilson's disease. Liver function tests and ultrasound were normal while Kayser-Fleischer rings were confirmed by slit lamp examination. Serum ceruloplasmin was low, 24-hour urine copper was elevated (125.5 mcg) and whole exome sequencing identified a heterozygous ATP7B mutation, confirming the diagnosis. Isolated neurological involvement without hepatic involvement is an extremely rare presentation and needs clinical expertise to delineate Wilson's disease as a possible aetiology.
一位女性青少年出现了 9 个月的进行性不自主运动病史,最初表现为手指震颤,随后发展为四肢投掷运动,导致过去 4 个月无法行走。同时,她出现构音障碍。神经学检查显示四肢肌力正常,僵直和深腱反射活跃,足跖反射下降。增强 MRI 显示双侧尾状核、基底节和脑桥存在高信号,提示Wilson 病。肝功能检查和超声正常,而裂隙灯检查证实了 Kayser-Fleischer 环。血清铜蓝蛋白降低,24 小时尿铜升高(125.5 mcg),全外显子组测序发现杂合性 ATP7B 突变,确诊为该病。无肝受累的单纯神经系统受累是一种极其罕见的表现,需要临床专业知识来明确Wilson 病作为可能病因。