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一种导致新生儿肝性脑病鉴别诊断困难的DNA聚合酶γ亚基(POLG)突变:病例报告

A DNA Polymerase Subunit Gamma (POLG) Mutation Imposing a Difficult Differential Diagnosis of Hepatic Encephalopathy in a Newborn: A Case Report.

作者信息

Cheyne Ithamar, Boryszewski Bartosz, Chang Wyven, Mikaszewska-Sokolewicz Małgorzata

机构信息

Anesthesiology and Critical Care Scientific Circle English Division (ANKONA ED), Medical University of Warsaw, Warsaw, POL.

Anesthesiology and Intensive Care, Children's Memorial Health Institute, Warsaw, POL.

出版信息

Cureus. 2024 Jul 24;16(7):e65239. doi: 10.7759/cureus.65239. eCollection 2024 Jul.

DOI:10.7759/cureus.65239
PMID:39184802
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11342063/
Abstract

Hepatic encephalopathy (HE) is a condition connected with neuropsychiatric alteration during hepatic failure. The differential diagnosis of HE is challenging due to overlapping symptoms with other conditions. Polymerase subunit gamma (POLG) is a mitochondrial gene, and an infant POLG mutation can manifest with severe and progressive hepatic failure and encephalopathy, imposing a difficult differential diagnosis due to similarities to other conditions. The lack of curative treatment leads to a poor prognosis.  An 11-month-old boy was admitted to the intensive care unit (ICU) due to altered consciousness and increasing edema due to acute hepatic failure of unknown etiology. After extensive multidisciplinary discussions and a lack of response to treatment for more than three weeks, a mitochondrial disease was suspected, and a genetic test was taken. The patient's condition continued to deteriorate. The patient died on the 25th day of hospitalization in the ICU. After death, a genetic test confirmed a rare POLG mutation NM_002693.3(POLG):c.3104+2T>A (Variation ID: 422378 Accession: VCV000422378.8).  We suggest that a screen test for POLG mutations be considered early in the diagnostic process and that clinicians consider mitochondrial genetic mutations, such as POLG mutations, more often. This article is the first to describe a patient with this specific mutation.

摘要

肝性脑病(HE)是一种与肝衰竭期间神经精神改变相关的病症。由于与其他病症症状重叠,HE的鉴别诊断具有挑战性。聚合酶亚基γ(POLG)是一种线粒体基因,婴儿POLG突变可表现为严重且进行性的肝衰竭和脑病,因其与其他病症相似而导致鉴别诊断困难。缺乏治愈性治疗导致预后不良。一名11个月大的男孩因意识改变和病因不明的急性肝衰竭导致水肿加重而入住重症监护病房(ICU)。经过广泛的多学科讨论且治疗三周多无反应后,怀疑有线粒体疾病并进行了基因检测。患者病情持续恶化。患者在ICU住院第25天死亡。死后基因检测证实存在罕见的POLG突变NM_002693.3(POLG):c.3104+2T>A(变异ID:422378登录号:VCV000422378.8)。我们建议在诊断过程早期考虑进行POLG突变筛查试验,临床医生应更频繁地考虑线粒体基因突变,如POLG突变。本文首次描述了一名具有这种特定突变的患者。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/0e552d88d13c/cureus-0016-00000065239-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/05776094faea/cureus-0016-00000065239-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/fbc88719ac8a/cureus-0016-00000065239-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/0e552d88d13c/cureus-0016-00000065239-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/05776094faea/cureus-0016-00000065239-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/fbc88719ac8a/cureus-0016-00000065239-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c90e/11342063/0e552d88d13c/cureus-0016-00000065239-i03.jpg

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本文引用的文献

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Mitochondrial Epilepsy, a Challenge for Neurologists.线粒体性癫痫:神经科医生面临的挑战。
Int J Mol Sci. 2022 Oct 30;23(21):13216. doi: 10.3390/ijms232113216.
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North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition Position Paper on the Diagnosis and Management of Pediatric Acute Liver Failure.北美儿童胃肠病学、肝病学和营养学学会关于儿童急性肝衰竭的诊断和管理的立场文件。
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Acute liver failure in newborns.新生儿急性肝衰竭
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POLG1-Related Epilepsy: Review of Diagnostic and Therapeutic Findings.POLG1相关癫痫:诊断与治疗结果综述
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