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葡萄糖-6-磷酸脱氢酶缺乏症与新生儿高胆红素血症:对疾病异质性的病理生理学、诊断及基因变异的见解

Glucose-6-Phosphate Dehydrogenase Deficiency and Neonatal Hyperbilirubinemia: Insights on Pathophysiology, Diagnosis, and Gene Variants in Disease Heterogeneity.

作者信息

Lee Heng Yang, Ithnin Azlin, Azma Raja Zahratul, Othman Ainoon, Salvador Armindo, Cheah Fook Choe

机构信息

Department of Paediatrics, Faculty of Medicine, Universiti Kebangsaan Malaysia Medical Centre, Cheras, Malaysia.

Department of Pathology, Faculty of Medicine, Universiti Kebangsaan Malaysia Medical Centre, Cheras, Malaysia.

出版信息

Front Pediatr. 2022 May 24;10:875877. doi: 10.3389/fped.2022.875877. eCollection 2022.

Abstract

Glucose-6-phosphate dehydrogenase (G6PD) deficiency is a prevalent condition worldwide and is caused by loss-of-function mutations in the G6PD gene. Individuals with deficiency are more susceptible to oxidative stress which leads to the classical, acute hemolytic anemia (favism). However, G6PD deficiency in newborn infants presents with an increased risk of hyperbilirubinemia, that may rapidly escalate to result in bilirubin induced neurologic dysfunction (BIND). Often with no overt signs of hemolysis, G6PD deficiency in the neonatal period appears to be different in the pathophysiology from favism. This review discusses and compares the mechanistic pathways involved in these two clinical presentations of this enzyme disorder. In contrast to the membrane disruption of red blood cells and Heinz bodies formation in favism, G6PD deficiency causing jaundice is perhaps attributed to the disruption of oxidant-antioxidant balance, impaired recycling of peroxiredoxin 2, thus affecting bilirubin clearance. Screening for G6PD deficiency and close monitoring of affected infants are important aspects in neonatal care to prevent kernicterus, a permanent and devastating neurological damage. WHO recommends screening for G6PD activity of all infants in countries with high prevalence of this deficiency. The traditional fluorescent spot test as a screening tool, although low in cost, misses a significant proportion of cases with moderate deficiency or the partially deficient, heterozygote females. Some newer and emerging laboratory tests and diagnostic methods will be discussed while developments in genomics and proteomics contribute to increasing studies that spatially profile genetic mutations within the protein structure that could predict their functional and structural effects. In this review, several known variants of G6PD are highlighted based on the location of the mutation and amino acid replacement. These could provide insights on why some variants may cause a higher degree of phenotypic severity compared to others. Further studies are needed to elucidate the predisposition of some variants toward certain clinical manifestations, particularly neonatal hyperbilirubinemia, and how some variants increase in severity when co-inherited with other blood- or bilirubin-related genetic disorders.

摘要

葡萄糖-6-磷酸脱氢酶(G6PD)缺乏症在全球范围内普遍存在,由G6PD基因的功能丧失突变引起。缺乏该酶的个体更容易受到氧化应激的影响,进而导致典型的急性溶血性贫血(蚕豆病)。然而,新生儿G6PD缺乏症会增加高胆红素血症的风险,可能迅速发展为胆红素诱导的神经功能障碍(BIND)。新生儿期G6PD缺乏症通常没有明显的溶血迹象,其病理生理学似乎与蚕豆病不同。本综述讨论并比较了这种酶紊乱的这两种临床表现所涉及的机制途径。与蚕豆病中红细胞膜破坏和海因茨小体形成不同,导致黄疸的G6PD缺乏症可能归因于氧化还原平衡的破坏、过氧化物酶2再循环受损,从而影响胆红素清除。筛查G6PD缺乏症并密切监测受影响的婴儿是新生儿护理中的重要方面,以预防核黄疸,这是一种永久性的、毁灭性的神经损伤。世界卫生组织建议在该缺乏症高发国家对所有婴儿进行G6PD活性筛查。传统的荧光斑点试验作为一种筛查工具,虽然成本低,但会遗漏相当一部分中度缺乏或部分缺乏的杂合子女性病例。本文将讨论一些新出现的实验室检测和诊断方法,而基因组学和蛋白质组学的发展有助于增加对蛋白质结构内基因突变进行空间分析的研究,从而预测其功能和结构效应。在本综述中,根据突变位置和氨基酸替代情况突出了几种已知的G6PD变体。这可以为为什么某些变体可能比其他变体导致更高程度的表型严重程度提供见解。需要进一步研究以阐明某些变体对某些临床表现的易感性,特别是新生儿高胆红素血症,以及某些变体在与其他血液或胆红素相关的遗传疾病共同遗传时如何加重严重程度。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/76c1/9170901/1577b29427f5/fped-10-875877-g001.jpg

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