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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.

DOI:10.1007/s00234-020-02435-7
PMID:32318771
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。

相似文献

1
The neuroimaging findings of monocarboxylate transporter 1 deficiency.单羧酸转运蛋白 1 缺乏症的神经影像学表现。
Neuroradiology. 2020 Jul;62(7):891-894. doi: 10.1007/s00234-020-02435-7. Epub 2020 Apr 21.
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Monocarboxylate transporter 1 deficiency and ketone utilization.单羧酸转运蛋白 1 缺乏与酮体利用。
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Rare cause of ketolysis: Monocarboxylate transporter 1 deficiency.罕见的酮体分解原因:单羧酸转运蛋白 1 缺乏症。
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Mitochondrial bioenergetic is impaired in Monocarboxylate transporter 1 deficiency: a new clinical case and review of the literature.单羧酸转运蛋白 1 缺乏症导致线粒体生物能量受损:一个新的临床病例及文献复习。
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Monocarboxylate transporter 1 deficiency and ketone utilization.单羧酸转运蛋白1缺乏与酮体利用
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Monocarboxylate transporter 1 deficiency and ketone utilization.单羧酸转运蛋白1缺乏与酮利用
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Monocarboxylate transporter 1 deficiency and ketone utilization.单羧酸转运蛋白1缺乏与酮利用
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Monocarboxylate transporter-1 deficiency results in severe metabolic acidosis with ketogenic diet in early onset absence epilepsy: Case report.单羧酸转运体-1缺乏导致早发性失神癫痫患者采用生酮饮食时出现严重代谢性酸中毒:病例报告。
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Monocarboxylate transport matters.单羧酸转运很重要。
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A SLC16A1 Mutation in an Infant With Ketoacidosis and Neuroimaging Assessment: Expanding the Clinical Spectrum of MCT1 Deficiency.一名患有酮症酸中毒婴儿的SLC16A1突变及神经影像学评估:扩展单羧酸转运蛋白1缺乏症的临床谱
Front Pediatr. 2019 Jul 18;7:299. doi: 10.3389/fped.2019.00299. eCollection 2019.

引用本文的文献

1
Mitochondrial bioenergetic is impaired in Monocarboxylate transporter 1 deficiency: a new clinical case and review of the literature.单羧酸转运蛋白 1 缺乏症导致线粒体生物能量受损:一个新的临床病例及文献复习。
Orphanet J Rare Dis. 2022 Jun 21;17(1):243. doi: 10.1186/s13023-022-02389-4.
2
SLC gene mutations and pediatric neurological disorders: diverse clinical phenotypes in a Saudi Arabian population.SLC 基因突变与儿科神经发育障碍:沙特阿拉伯人群中的多种临床表型。
Hum Genet. 2022 Jan;141(1):81-99. doi: 10.1007/s00439-021-02404-x. Epub 2021 Nov 19.