Department of Clinical Biochemistry, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran.
Bioinformatics Research Center, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran.
Endocr Metab Immune Disord Drug Targets. 2024;24(2):194-207. doi: 10.2174/1871530323666230623120935.
Inborn errors of metabolism (IEMs) are a vast array of inherited/congenital disorders, affecting a wide variety of metabolic pathways and/or biochemical processes inside the cells. Although IEMs are usually rare, they can be represented as serious health problems. During the neonatal period, these inherited defects can give rise to almost all key signs of liver malfunction, including jaundice, coagulopathy, hepato- and splenomegaly, ascites, etc. Since the liver is a vital organ with multiple synthetic, metabolic, and excretory functions, IEM-related hepatic dysfunction could seriously be considered life-threatening. In this context, the identification of those hepatic manifestations and their associated characteristics may promote the differential diagnosis of IEMs immediately after birth, making therapeutic strategies more successful in preventing the occurrence of subsequent events. Among all possible liver defects caused by IEMs, cholestatic jaundice, hepatosplenomegaly, and liver failure have been shown to be manifested more frequently. Therefore, the current study aims to review substantial IEMs that mostly result in the aforementioned hepatic disorders, relying on clinical principles, especially through the first years of life. In this article, a group of uncommon hepatic manifestations linked to IEMs is also discussed in brief.
先天性代谢缺陷(IEMs)是一大类遗传性/先天性疾病,影响细胞内的多种代谢途径和/或生化过程。虽然 IEMs 通常很少见,但它们可能会导致严重的健康问题。在新生儿期,这些遗传缺陷可能会引起几乎所有肝功能异常的主要体征,包括黄疸、凝血功能障碍、肝脾肿大、腹水等。由于肝脏是一个具有多种合成、代谢和排泄功能的重要器官,与 IEM 相关的肝功能障碍可能会严重危及生命。在这种情况下,识别这些肝脏表现及其相关特征可以促进 IEM 在出生后立即进行鉴别诊断,从而使治疗策略更成功地预防后续事件的发生。在 IEM 引起的所有可能的肝脏缺陷中,胆汁淤积性黄疸、肝脾肿大和肝功能衰竭更常表现出来。因此,本研究旨在根据临床原则,特别是在生命的头几年,回顾主要导致上述肝脏疾病的大量 IEM。本文还简要讨论了与 IEM 相关的一组不常见的肝脏表现。