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沙特阿拉伯一例非典型全身性原发性肉碱缺乏症病例。

A case of atypical systemic primary carnitine deficiency in Saudi Arabia.

作者信息

Alghamdi Abdulrahman, Almalki Hani, Shawli Aiman, Waggass Rahaf, Hakami Fahad

机构信息

King Abdullah International Medical Research Center.

King Saud bin Abdulaziz University for Health Sciences.

出版信息

Pediatr Rep. 2018 Jun 27;10(2):7705. doi: 10.4081/pr.2018.7705. eCollection 2018 May 24.

Abstract

Systemic primary carnitine deficiency (SPCD) is an autosomal recessive inborn error of fatty acid metabolism caused by a defect in the transporter responsible for moving carnitine across plasma membrane. The clinical features of SPCD vary widely based on the age of onset and organs involved. During infancy, patients might show episodes of hypoketotic hypoglycemia, hepatomegaly, elevated transaminases, and hyperammonemia. Skeletal myopathy, elevated creatine kinase, and cardiomyopathy are the main manifestations in children with SPCD, while in adults, the disorder is usually manifested as cardiomyopathy, arrhythmias, or fatigability. Here, we report a 5-year-old boy with SPCD that presented as dilated cardiomyopathy with atypical features, such as anemia, respiratory distress, and proximal muscle weakness. This report supports considering carnitine deficiency treatment in the work-up of unexplained pediatric dilated cardiomyopathy.

摘要

系统性原发性肉碱缺乏症(SPCD)是一种常染色体隐性遗传的脂肪酸代谢先天性缺陷疾病,由负责将肉碱转运穿过质膜的转运体缺陷引起。SPCD的临床特征因发病年龄和受累器官的不同而有很大差异。在婴儿期,患者可能会出现低酮性低血糖、肝肿大、转氨酶升高和高氨血症发作。骨骼肌病、肌酸激酶升高和心肌病是SPCD患儿的主要表现,而在成人中,该疾病通常表现为心肌病、心律失常或易疲劳。在此,我们报告一名5岁患有SPCD的男孩,其表现为具有非典型特征的扩张型心肌病,如贫血、呼吸窘迫和近端肌无力。本报告支持在不明原因的小儿扩张型心肌病检查中考虑肉碱缺乏症的治疗。

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