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脑腱黄瘤病:一种具有特征性影像学表现的罕见神经退行性疾病。

Cerebrotendinous xanthomatosis: A rare neurodegenerative disorder with characteristic imaging findings.

作者信息

Khan Hamza Ghazali, Raj Gaurav, Yadav Eshita, Gupta Kaustubh, Kulshreshtha Dinkar, Panda Anshumalini, Kumar Yogesh

机构信息

Department of Radio-Daignosis, Dr Rmlims, Lucknow, Uttar Pradesh, India.

Department of Neurology, Dr Rmlims, Lucknow, Uttar Pradesh, India.

出版信息

Radiol Case Rep. 2025 Jun 12;20(9):4347-4351. doi: 10.1016/j.radcr.2025.05.040. eCollection 2025 Sep.

DOI:10.1016/j.radcr.2025.05.040
PMID:40583979
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12205547/
Abstract

Cerebrotendinous Xanthomatosis (CTX) is a rare, autosomal recessive lipid storage disorder caused by mutations in the CYP27A1 gene, leading to defective bile acid synthesis and systemic accumulation of cholestanol with characteristic findings on imaging. We present a case of a patient with juvenile-onset bilateral cataract, progressive cognitive decline, spastic paraparesis, behavioral abnormalities and seizures. MRI brain revealed symmetrical T2 hyperintensity and T1 hypointensity in the bilateral dentate nuclei, fronto-parietal atrophy, and nonspecific periventricular white matter changes. Ultrasound and MRI of the Achilles tendon demonstrated xanthomas bilaterally. Based on clinical and imaging features, a diagnosis of CTX was considered and later confirmed by genetic testing. This case highlights the role of neuroimaging in the early diagnosis of CTX, enabling timely initiation of chenodeoxycholic acid (CDCA) therapy, which may arrest or reverse disease progression.

摘要

脑腱黄瘤病(CTX)是一种罕见的常染色体隐性脂质贮积病,由CYP27A1基因突变引起,导致胆汁酸合成缺陷和胆甾烷醇在体内蓄积,影像学上有特征性表现。我们报告一例患者,有青少年期双侧白内障、进行性认知衰退、痉挛性截瘫、行为异常和癫痫发作。脑部MRI显示双侧齿状核T2高信号和T1低信号、额顶叶萎缩以及脑室周围白质非特异性改变。跟腱超声和MRI显示双侧有黄瘤。基于临床和影像学特征,考虑诊断为CTX,后来经基因检测得以证实。该病例突出了神经影像学在CTX早期诊断中的作用,能够及时开始鹅去氧胆酸(CDCA)治疗,这可能会阻止或逆转疾病进展。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/8e801ebac985/gr4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/3bd652907bbb/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/95dcc2a944ca/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/f39621c9571c/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/8e801ebac985/gr4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/3bd652907bbb/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/95dcc2a944ca/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/f39621c9571c/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7e1a/12205547/8e801ebac985/gr4.jpg

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本文引用的文献

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Cerebrotendinous xanthomatosis: A complex interplay between a clinically and genetically heterogeneous condition.脑腱黄瘤病:一种临床和基因均具有异质性的病症之间的复杂相互作用。
Eur J Neurol. 2025 Jan;32(1):e70006. doi: 10.1111/ene.70006.
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Cerebrotendinous xanthomatosis: a literature review and case study.脑腱性黄瘤病:文献综述与病例研究
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脑腱黄瘤病患者的临床和影像学特征——来自印度的视频病例系列。
Tremor Other Hyperkinet Mov (N Y). 2024 Mar 6;14:10. doi: 10.5334/tohm.851. eCollection 2024.
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Cerebrotendinous Xanthomatosis, a Treatable Disorder Often Missed: Case Series of Three Patients Confirmed by Genetic Testing.脑腱黄瘤病,一种常被漏诊的可治疗疾病:经基因检测确诊的三例患者的病例系列。
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Cerebrotendinous xanthomatosis: clinical and imaging clues of a rare treatable cause of ataxia.脑腱黄瘤病:一种罕见的可治疗性共济失调病因的临床及影像学线索
BMJ Case Rep. 2022 Jul 20;15(7):e250714. doi: 10.1136/bcr-2022-250714.
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Achilles swelling and ataxia in an adolescent: A case report of cerebrotendinous xanthomatosis.一名青少年的跟腱肿胀和共济失调:脑腱性黄瘤病病例报告
Radiol Case Rep. 2022 Jan 13;17(3):898-901. doi: 10.1016/j.radcr.2021.12.043. eCollection 2022 Mar.
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Achilles Tendon Xanthoma and Cholestanol Revealing Cerebrotendinous Xanthomatosis: A New Case Report.跟腱黄瘤和胆甾烷醇揭示脑腱性黄瘤病:一例新病例报告
Case Rep Rheumatol. 2021 May 20;2021:6656584. doi: 10.1155/2021/6656584. eCollection 2021.
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The clinical and imaging features of cerebrotendinous xanthomatosis: A case report and review of the literature.脑腱黄瘤病的临床和影像学特征:病例报告及文献复习。
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