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一种拉斯穆森脑炎、自身免疫性脑炎和线粒体疾病的模仿者:扩展与DNM1L相关的难治性癫痫和脑病表型。

A Rasmussen encephalitis, autoimmune encephalitis, and mitochondrial disease mimicker: expanding the DNM1L-associated intractable epilepsy and encephalopathy phenotype.

作者信息

Nolan Danielle A, Chen Baibing, Michon Anne Marie, Salatka Emily, Arndt Daniel

机构信息

Department of Pediatric Epilepsy, Beaumont Health, Royal Oak, MI 48073.

Oakland University William Beaumont School of Medicine, Rochester, MI 48309, USA.

出版信息

Epileptic Disord. 2019 Feb 1;21(1):112-116. doi: 10.1684/epd.2019.1036.

DOI:10.1684/epd.2019.1036
PMID:30767894
Abstract

Dynamin-1-like protein (DNM1L) gene variants have been linked to childhood refractory epilepsy, developmental delay, encephalopathy, microcephaly, and progressive diffuse cerebral atrophy. However, only a few cases have been reported in the literature and there is still a limited amount of information about the symptomatology and pathophysiology associated with pathogenic variants of DNM1L. We report a 10-year-old girl with a one-year history of mild learning disorder and absence seizures who presented with new-onset focal status epilepticus which progressed to severe encephalopathy and asymmetric hemispheric cerebral atrophy. Differential diagnosis included mitochondrial disease, Rasmussen's encephalitis, and autoimmune encephalitis. Disease progressed from one hemisphere to the other despite anti-seizure medications, hemispherectomy, vagus nerve stimulator, ketogenic diet, and immunomodulators. Continued cerebral atrophy and refractory seizures evolved until death four years after initial presentation. Post-mortem whole-exome sequencing revealed a pathogenic DNM1L variant. This paper presents a novel case of adolescent-onset DNM1L-related intractable epilepsy and encephalopathy.

摘要

动力蛋白1样蛋白(DNM1L)基因变异与儿童难治性癫痫、发育迟缓、脑病、小头畸形和进行性弥漫性脑萎缩有关。然而,文献中仅报道了少数病例,关于DNM1L致病变异相关的症状学和病理生理学的信息仍然有限。我们报告了一名10岁女孩,有1年轻度学习障碍和失神发作病史,出现新发局灶性癫痫持续状态,进而发展为严重脑病和不对称性半球脑萎缩。鉴别诊断包括线粒体疾病、拉斯穆森脑炎和自身免疫性脑炎。尽管使用了抗癫痫药物、半球切除术、迷走神经刺激器、生酮饮食和免疫调节剂,疾病仍从一个半球发展到另一个半球。持续的脑萎缩和难治性癫痫发作不断进展,直至初次就诊4年后死亡。尸检全外显子测序发现了一个致病的DNM1L变异。本文介绍了一例青少年期起病的DNM1L相关难治性癫痫和脑病的新病例。

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