患有肉碱-肉碱转运酶缺乏症的新生儿因心脏受累而猝死

IF 2.3 4区 医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS
Jiayu Jing , Cui Zhang , Sihao Du, Xiaohui Tan, Xia Yue, Dongfang Qiao
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引用次数: 0

摘要

一名在胎龄 38+2 周时出生、Apgar 评分正常的女新生儿在出生后不到 30 小时内意外死亡。这种情况与她哥哥早先在产后 24 小时内死亡的情况如出一辙,这表明可能存在遗传疾病。大体检查发现,她的心室普遍发绀,并有明显的淡黄色改变。组织病理学分析显示,肝脏、心脏和肾脏中存在脂质堆积。串联质谱法检测到心脏血液中 10 种氨基酸和 14 种肉碱含量升高。三重全基因组测序(Trio-WGS)发现,SLC25A20 c.199-10T>G突变与肉碱-酰肉碱转位酶病(CACTD)有关,这是一种可能导致猝死的脂肪酸氧化紊乱病(FAODs)。基因表达的进一步验证证实了 SLC25A20 的功能缺陷,最终确诊 CACTD 是导致新生儿死亡的根本原因。该病例突出了产前代谢和遗传筛查对准父母的重要性,并强调了法医将代谢组学和基因组学调查纳入疑似遗传代谢性疾病尸检的必要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Sudden death with cardiac involvement in a neonate with carnitine-acylcarnitine translocase deficiency

A female neonate born with normal Apgar scores at 38+2 weeks of gestational age unexpectedly passed away within less than 30 hours after birth. The situation mirrored her brother's earlier demise within 24 hours post-delivery, suggesting a possible genetic disorder. Gross examination revealed widespread cyanosis and distinct yellowish changes on the cardiac ventricles. Histopathological examination disclosed lipid accumulation in the liver, heart, and kidneys. Tandem mass spectrometry detected elevated levels of 10 amino acids and 14 carnitines in cardiac blood. Trio-whole genome sequencing (Trio-WGS) identified the SLC25A20 c.199-10T>G mutation associated with carnitine-acylcarnitine translocase disease (CACTD), a type of fatty acid oxidation disorders (FAODs) with a potential for sudden death. Further validation of gene expression confirmed the functional deficiency of SLC25A20, ultimately diagnosing CACTD as the underlying cause of the neonate's demise. This case highlights the importance of prenatal metabolic and genetic screening for prospective parents and emphasizes the need for forensic doctors to integrate metabolomic and genomic investigations into autopsies for suspected inherited metabolic diseases.

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来源期刊
Cardiovascular Pathology
Cardiovascular Pathology 医学-病理学
CiteScore
7.50
自引率
2.70%
发文量
71
审稿时长
18 days
期刊介绍: Cardiovascular Pathology is a bimonthly journal that presents articles on topics covering the entire spectrum of cardiovascular disease. The Journal''s primary objective is to publish papers on disease-oriented morphology and pathogenesis from clinicians and scientists in the cardiovascular field. Subjects covered include cardiovascular biology, prosthetic devices, molecular biology and experimental models of cardiovascular disease.
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