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表现为严重新生儿脑病的USP18缺乏症:一例病例报告及伪TORCH综合征综述

USP18 Deficiency Presenting as Severe Neonatal Encephalopathy: A Case Report and Review of Pseudo-TORCH Syndromes.

作者信息

Alattas Amjed, Alsamnan Malak

机构信息

Neonatology, Prince Sultan Military Medical City, Riyadh, SAU.

出版信息

Cureus. 2025 Sep 10;17(9):e91952. doi: 10.7759/cureus.91952. eCollection 2025 Sep.

DOI:10.7759/cureus.91952
PMID:40937018
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12421291/
Abstract

Pseudo-TORCH (Toxoplasma, "Other" agents, Rubella, Cytomegalovirus, Herpes simplex, etc.) syndromes are rare genetic disorders that mimic congenital TORCH infections but lack an infectious cause. Affected infants present with encephalopathy, intracranial calcifications, and congenital anomalies. We report a term male infant who developed catastrophic encephalopathy and coagulopathy on day eight of life, with neuroimaging showing hemorrhages, calcifications, and cortical malformations. Whole-exome sequencing revealed a homozygous splice-site mutation in USP18, confirming pseudo-TORCH syndrome type 2. Despite maximal intensive care, the infant succumbed at one month of age. This case illustrates the diagnostic challenges and rapid progression of USP18 deficiency, highlights the value of early genetic confirmation for family counseling, and reviews the spectrum of pseudo-TORCH syndromes. Emerging therapies targeting interferon pathways may offer future hope. Our literature review highlights the differential diagnoses and genetic variability among pseudo-TORCH conditions, which include mutations in OCLN, USP18, STAT2, and JAM3. This emphasizes the importance of early genetic confirmation for better prognosis and family counseling. Emerging therapies targeting the interferon pathway, such as Janus kinase (JAK) inhibitors, are currently under investigation and may offer hope for these previously untreatable disorders. This case underscores the necessity of considering pseudo-TORCH syndromes in neonates who present with unexplained encephalopathy and brain calcifications after infectious causes have been ruled out.

摘要

假TORCH(弓形虫、“其他”病原体、风疹、巨细胞病毒、单纯疱疹等)综合征是罕见的遗传性疾病,其症状类似于先天性TORCH感染,但并非由感染引起。患病婴儿表现为脑病、颅内钙化和先天性异常。我们报告了一名足月儿男婴,其在出生后第8天出现了灾难性脑病和凝血病,神经影像学检查显示有出血、钙化和皮质畸形。全外显子测序揭示了USP18基因存在纯合剪接位点突变,确诊为2型假TORCH综合征。尽管给予了最大程度的重症监护,该婴儿仍在1个月大时死亡。本病例说明了USP18缺乏症的诊断挑战和快速进展,强调了早期基因确诊对家庭咨询的价值,并回顾了假TORCH综合征的谱系。针对干扰素途径的新兴疗法可能带来未来的希望。我们的文献综述强调了假TORCH病症之间的鉴别诊断和基因变异性,其中包括OCLN、USP18、STAT2和JAM3基因的突变。这强调了早期基因确诊对于改善预后和家庭咨询的重要性。目前正在研究针对干扰素途径的新兴疗法,如 Janus激酶(JAK)抑制剂,可能为这些以前无法治疗的疾病带来希望。 本病例强调了对于出现不明原因脑病和脑钙化且已排除感染原因的新生儿,考虑假TORCH综合征的必要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b5a9/12421291/80aef050bd5f/cureus-0017-00000091952-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b5a9/12421291/80aef050bd5f/cureus-0017-00000091952-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b5a9/12421291/80aef050bd5f/cureus-0017-00000091952-i01.jpg

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本文引用的文献

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A novel homozygous variant in JAM3 gene causing hemorrhagic destruction of the brain, subependymal calcification, and congenital cataracts (HDBSCC) with neonatal onset.一个新的 JAM3 基因纯合变异导致脑内出血性破坏、室管膜下钙化和先天性白内障(HDBSCC),并伴有新生儿发病。
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