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模仿多发性硬化症的法布里杂合子。

Fabry heterozygote mimicking multiple sclerosis.

作者信息

Yau Wai Yan, Fabis-Pedrini Marzena J, Kermode Allan G

机构信息

Department of Neurology, Royal Free Hospital, Sir Charles Gairdner Hospital, London, UK.

Western Australian Neuromuscular Research Institute, Nedlands, Western Australia, Australia.

出版信息

BMJ Case Rep. 2017 Jun 2;2017:bcr-2017-220274. doi: 10.1136/bcr-2017-220274.

Abstract

Fabry's disease (FD) is a recognised mimic of multiple sclerosis (MS). It is an X-linked storage lysosomal disorder with deficiency of α-galactosidase A and enzyme replacement therapy is available. Patients with FD may satisfy modified McDonald criteria if the diagnosis of FD has not been pursued. We present a case of FD in a 65-year-old woman masquerading as benign MS for 40 years. She has recurrent posterior circulation stroke-like symptoms, hearing loss and acroparaesthesia, but typical radiological features of MS on MRI brain. Later she developed an ischaemic stroke, infiltrative cardiomyopathy and chronic renal failure. There was a missense mutation at p.R342Q in the galactodisdase alpha (GLA) gene. Neurologists need to consider FD and look for red flags in atypical MS cases and should not be over-reliant on MRI findings. Missed diagnosis of FD could lead to unnecessary immunosuppression, inappropriate disease counselling and missed treatment opportunity.

摘要

法布里病(FD)是一种已被认可的可模仿多发性硬化症(MS)的疾病。它是一种X连锁的溶酶体贮积症,缺乏α-半乳糖苷酶A,且有酶替代疗法可用。如果未对FD进行诊断,FD患者可能符合改良的麦克唐纳标准。我们报告一例65岁女性的FD病例,该病例伪装成良性MS长达40年。她有反复发作的后循环卒中样症状、听力丧失和肢端感觉异常,但脑部MRI有典型的MS影像学特征。后来她发生了缺血性卒中、浸润性心肌病和慢性肾衰竭。在半乳糖苷酶α(GLA)基因的p.R342Q位点存在错义突变。神经科医生需要考虑FD,并在非典型MS病例中寻找警示信号,不应过度依赖MRI检查结果。FD的漏诊可能导致不必要的免疫抑制、不适当的疾病咨询和错过治疗机会。

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