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中国患者的穆克-韦尔斯综合征:一项单中心病例系列研究

Muckle-Wells syndrome in Chinese patients: a single center case series.

作者信息

Wu Di, Shen Min

机构信息

Peking Union Medical College Hospital, Beijing, 100730, China.

出版信息

Clin Rheumatol. 2017 Apr;36(4):965-969. doi: 10.1007/s10067-016-3523-3. Epub 2016 Dec 27.

DOI:10.1007/s10067-016-3523-3
PMID:28028683
Abstract

Muckle-Wells syndrome (MWS) is a rare autoinflammatory disease. This study aimed to report the clinical features and gene variations of the first case series of MWS patients in Chinese population. Four Han Chinese patients were diagnosed with MWS and followed up at our adult clinic for autoinflammatory diseases. All relevant phenotypes and genotypes were collected. All patients were adult male. The median age of disease onset was 4.5 years, and one patient had adult-onset disease. No positive family history was observed. All patients had a remittent disease course. The duration of fever attacks ranged from 0.5 to 7 days. Skin rashes were present in all patients. The other manifestations included polyarthralgia/arthritis (n = 3), oral ulcers (n = 2), conjunctivitis (n = 2), myalgia (n = 2), headache (n = 2), pharyngitis (n = 1), abdominal pain (n = 1), severe sensorineural hearing loss (n = 1), and chronic meningitis with communicating hydrocephalus (n = 1). None of the patients showed evidence of renal amyloidosis. Each patient carried a heterozygous mutation in an NLRP3 gene, including D29V, V70M, T348M, and Q703K, respectively. D29V and V70M variants were novel mutations in exon 1 of NLRP3. All patients had good response to corticosteroids. Our study suggests that MWS could be identified in Chinese population. Our finding of novel mutations in NLRP3 may expand the diversity of MWS.

摘要

穆克勒-韦尔斯综合征(MWS)是一种罕见的自身炎症性疾病。本研究旨在报告中国人群中首例MWS患者系列病例的临床特征和基因变异情况。4例汉族患者被诊断为MWS,并在我们的成人自身炎症性疾病门诊接受随访。收集了所有相关的表型和基因型。所有患者均为成年男性。疾病发病的中位年龄为4.5岁,1例患者为成年发病。未观察到阳性家族史。所有患者的病程呈缓解期。发热发作持续时间为0.5至7天。所有患者均出现皮疹。其他表现包括多关节痛/关节炎(n = 3)、口腔溃疡(n = 2)、结膜炎(n = 2)、肌痛(n = 2)、头痛(n = 2)、咽炎(n = 1)、腹痛(n = 1)、重度感音神经性听力损失(n = 1)和伴有交通性脑积水的慢性脑膜炎(n = 1)。所有患者均未显示肾淀粉样变性的证据。每位患者在NLRP3基因中均携带一个杂合突变,分别为D29V、V70M、T348M和Q703K。D29V和V70M变异是NLRP3外显子1中的新突变。所有患者对皮质类固醇均有良好反应。我们的研究表明,中国人群中可识别出MWS。我们在NLRP3中发现的新突变可能会扩大MWS的多样性。

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Identification of a variant in NLRP3 gene in a patient with Muckle-Wells syndrome: a case report and review of literature.

本文引用的文献

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Recommendations for the management of autoinflammatory diseases.自身炎症性疾病管理建议。
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Diagnosis of cryopyrin-associated periodic syndrome: challenges, recommendations and emerging concepts.
NLRP3 基因变异致 Muckle-Wells 综合征 1 例报告并文献复习
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Cutaneous Manifestations of Autoinflammatory Diseases.自身炎症性疾病的皮肤表现
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Rare mutations in NLRP3 and NLRP12 associated with familial cold autoinflammatory syndrome: two Chinese pedigrees.与家族性寒冷性自身炎症综合征相关的NLRP3和NLRP12罕见突变:两个中国家系
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Monogenic Adult-Onset Inborn Errors of Immunity.单基因成人起病的先天性免疫缺陷病
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Ocular manifestations in Chinese adult patients with NLRP3-associated autoinflammatory disease.中国成年 NLRP3 相关性自身炎症性疾病患者的眼部表现。
Sci Rep. 2021 Jun 7;11(1):11904. doi: 10.1038/s41598-021-91315-y.
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Computational Modeling of NLRP3 Identifies Enhanced ATP Binding and Multimerization in Cryopyrin-Associated Periodic Syndromes.计算建模鉴定 NOD 样受体热蛋白结构域 3(NLRP3)在冷炎蛋白相关周期性综合征中的增强型 ATP 结合和多聚化。
Front Immunol. 2020 Nov 19;11:584364. doi: 10.3389/fimmu.2020.584364. eCollection 2020.
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Development and Validation of a Targeted Next-Generation Sequencing Gene Panel for Children With Neuroinflammation.针对神经炎症儿童的靶向下一代测序基因 panel 的开发和验证。
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冷吡啉相关周期性综合征的诊断:挑战、建议及新观点
Expert Rev Clin Immunol. 2015;11(7):827-35. doi: 10.1586/1744666X.2015.1047765. Epub 2015 May 15.
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Ann Rheum Dis. 2015 Nov;74(11):2043-9. doi: 10.1136/annrheumdis-2013-204991. Epub 2014 Jul 18.
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Somatic NLRP3 mosaicism in Muckle-Wells syndrome. A genetic mechanism shared by different phenotypes of cryopyrin-associated periodic syndromes.Muckle-Wells 综合征中的体细胞 NLRP3 镶嵌现象。一种与 cryopyrin 相关周期性综合征不同表型共享的遗传机制。
Ann Rheum Dis. 2015 Mar;74(3):603-10. doi: 10.1136/annrheumdis-2013-204361. Epub 2013 Dec 10.
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Clin Exp Rheumatol. 2012 Nov-Dec;30(6):943-6. Epub 2012 Dec 17.
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Efficacy and safety of anakinra therapy in pediatric and adult patients with the autoinflammatory Muckle-Wells syndrome.阿那白滞素治疗儿童和成人自身炎症性穆-韦综合征的疗效与安全性。
Arthritis Rheum. 2011 Mar;63(3):840-9. doi: 10.1002/art.30149.