一个患有 3-羟基异丁酰-CoA水解酶缺乏症并伴有严重高阴离子差酸中毒的足月婴儿死亡病例及文献综述。

Case Reports in Genetics Pub Date : 2024-06-30 eCollection Date: 2024-01-01 DOI:10.1155/2024/8099373
Surasak Puvabanditsin, Ian Lee, Natasha Cordero, Keisha Target, Su Young Park, Rajeev Mehta
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引用次数: 0

摘要

3-hydroxy isobutyl-CoA hydrolase (HIBCH) 缺乏症是最近描述的一种罕见的缬氨酸代谢先天性错误,与利综合征(Leigh Syndrome)样表型和神经退行性症状相关,由 HIBCH 基因的隐性突变引起。我们报告了一个迄今为止最严重的病例:一名宫内生长受限的足月男婴出生后不久即出现严重酸中毒和高阴离子间隙。这种表现是致命的,导致患者在出生后36小时内死亡。诊断是在出生后通过全基因组测序(WGS)得出的。我们报告了一起新生儿快速致命的 HIBCN 事件,并回顾了相关文献。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Fatal Case of 3-Hydroxyisobutyryl-CoA Hydrolase Deficiency in a Term Infant with Severe High Anion Gap Acidosis and Review of the Literature.

3-hydroxy isobutyl-CoA hydrolase (HIBCH) deficiency is a recently described, rare inborn error of valine metabolism associated with a Leigh syndrome-like phenotype, neurodegenerative symptoms, and caused by recessive mutations in the HIBCH gene. We report the most severe case to date of an intrauterine growth-restricted term male who presented with severe acidosis and a high anion gap soon after birth. The manifestation was fatal that led to death within 36 hours of life. The diagnosis was made postnatally by Whole Genome Sequencing (WGS). We report a rapid and fatal event of HIBCN in a neonate and review of the literature.

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