Suppr超能文献

单羧酸转运蛋白 1 缺乏症的神经影像学表现。

The neuroimaging findings of monocarboxylate transporter 1 deficiency.

机构信息

Division of Neuroradiology, Department of Radiology, King Faisal Specialist Hospital and Research Centre, Al Zahrawi Street, Riyadh, 11211, Saudi Arabia.

Department of Endocrinology, Hotel-Dieu de France Hospital, Alfred Naccache Boulevard, Beirut, Lebanon.

出版信息

Neuroradiology. 2020 Jul;62(7):891-894. doi: 10.1007/s00234-020-02435-7. Epub 2020 Apr 21.

Abstract

Monocarboxylate transporter 1 (MCT1) deficiency was first described in 2014 by Hasselt et al. as a novel genetic cause of recurrent ketoacidosis. Patients present in the first year of life with acute episodes of ketoacidosis triggered by fasting or infections. Patients with homozygous mutations are known to have a more severe phenotype with mild to moderate developmental delay and an increased prevalence of epilepsy. There is only one recent report of the neuroimaging findings of this disorder as reported by Al-Khawaga et al. (Front Pediatr. 7:299, 2019). We report the neuroimaging abnormalities in two siblings with similar clinical presentation of recurrent ketoacidosis, seizures, and developmental delay. Whole exome sequencing in the younger sibling confirmed a known pathogenic homozygous mutation in MCT1, also known as SLC16A1 gene. Brain MRI showed a similar very distinctive pattern of signal abnormality at the gray-white matter junction, basal ganglia, and thalami in both patients. Both siblings had agenesis of the corpus callosum. Knowledge of this pattern of brain involvement might contribute to an earlier diagnosis and timely management of this rare and under recognized disorder.

摘要

单羧酸转运蛋白 1(MCT1)缺乏症于 2014 年由 Hasselt 等人首次描述为复发性酮症酸中毒的一种新的遗传病因。患者在生命的第一年出现急性酮症酸中毒发作,由禁食或感染引发。已知纯合突变患者的表型更为严重,伴有轻度至中度发育迟缓,癫痫患病率增加。最近只有一篇关于这种疾病神经影像学表现的报道,由 Al-Khawaga 等人报道(Front Pediatr. 7:299, 2019)。我们报告了两名具有相似复发性酮症酸中毒、癫痫发作和发育迟缓临床表现的兄弟姐妹的神经影像学异常。较年轻的兄弟姐妹进行全外显子组测序证实存在已知的致病性纯合突变,该突变也称为 SLC16A1 基因。脑 MRI 显示两名患者在灰质-白质交界处、基底节和丘脑均存在相似的非常独特的信号异常模式。两名患者均存在胼胝体发育不全。了解这种脑受累模式可能有助于更早诊断和及时治疗这种罕见且认识不足的疾病。

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验