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病例报告:以“头部下垂”为主要症状的亚历山大病及文献综述

Case report: Alexander's disease with "head drop" as the main symptom and literature review.

作者信息

Yuan Yujun, Wu Qiong, Huo Liang, Wang Hua, Liu Xueyan

机构信息

Department of Pediatrics, Shengjing Hospital of China Medical University, Shenyang, China.

出版信息

Front Neurol. 2022 Dec 19;13:1002527. doi: 10.3389/fneur.2022.1002527. eCollection 2022.

Abstract

Alexander's disease (AxD) is a rare autosomal dominant hereditary disorder that is caused by the mutations in the GFAP gene, which encodes the glial fibrillary acidic protein (GFAP). This neurogenerative disease has many clinical manifestations, and the onset of disease spans a wide range of ages, from newborns to children, adults, and even the elderly. An overaccumulation of the expression of GFAP has a close causal relationship with the pathogenesis of Alexander's disease. Usually, the disease has severe morbidity and high mortality, and can be divided into three distinct subgroups that are based on the age of clinical presentation: infantile (0-2 years), juvenile (2-13 years), and adult (>13 years). Children often present with epilepsy, macrocephaly, and psychomotor retardation, while adolescents and adults mainly present with muscle weakness, spasticity, and bulbar symptoms. Atonic seizures are a type of epilepsy that often appears in the Lennox-Gastaut syndrome and myoclonic-astatic epilepsy in early childhood; however, the prognosis is often poor. Atonic episodes are characterized by a sudden or frequent reduction in muscle tone that can be local (such as head, neck, or limb) or generalized. Here, we report a 4-year-old girl whose main symptoms were intermittent head drop movements, which could break the frontal frame and even bleed in severe conditions. A video-encephalography (VEEG) showed that the nodding movements were atonic seizures. A head magnetic resonance imaging (MRI) revealed abnormal signals in the bilateral paraventricular and bilateral subfrontal cortex. The gene detection analyses indicated that the GFAP gene exon 1 c.262 C>T was caused by a heterozygous mutation, as both her parents were of the wild-type. The girl had no other abnormal manifestations except atonic seizures. She could communicate normally and go to kindergarten. After an oral administration of sodium valproate, there were no atonic attacks. Although epilepsy is a common symptom of Alexander's disease, atonic seizures have not been reported to date. Therefore, we report a case of Alexander's disease with atonic seizures as the main symptom and provide a review of the literature.

摘要

亚历山大病(AxD)是一种罕见的常染色体显性遗传性疾病,由编码胶质纤维酸性蛋白(GFAP)的GFAP基因突变引起。这种神经退行性疾病有许多临床表现,发病年龄范围广泛,从新生儿到儿童、成人甚至老年人。GFAP表达的过度积累与亚历山大病的发病机制有密切的因果关系。通常,该疾病发病率高、死亡率高,根据临床表现的年龄可分为三个不同的亚组:婴儿型(0 - 2岁)、青少年型(2 - 13岁)和成人型(>13岁)。儿童常表现为癫痫、巨头畸形和精神运动发育迟缓,而青少年和成人主要表现为肌肉无力、痉挛和延髓症状。失张力发作是一种癫痫类型,常出现在儿童早期的Lennox - Gastaut综合征和肌阵挛 - 失张力癫痫中;然而,预后通常较差。失张力发作的特征是肌肉张力突然或频繁降低,可局部(如头部、颈部或肢体)或全身性。在此,我们报告一名4岁女孩,其主要症状为间歇性头部下垂动作,严重时可撞破额部框架甚至出血。视频脑电图(VEEG)显示点头动作是失张力发作。头部磁共振成像(MRI)显示双侧脑室旁和双侧额叶下皮质有异常信号。基因检测分析表明,GFAP基因外显子1 c.262 C>T由杂合突变引起,其父母均为野生型。该女孩除失张力发作外无其他异常表现。她能正常交流并上幼儿园。口服丙戊酸钠后,未再出现失张力发作。虽然癫痫是亚历山大病的常见症状,但迄今为止尚未有失张力发作的报道。因此,我们报告一例以失张力发作为主要症状的亚历山大病病例并进行文献复习。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/15dd/9807021/fcae9de1145c/fneur-13-1002527-g0001.jpg

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