以儿童 SARS-CoV-2 相关多系统炎症综合征为表现的先天性 WAS 错误。

IF 7.2 2区 医学 Q1 IMMUNOLOGY Journal of Clinical Immunology Pub Date : 2024-11-25 DOI:10.1007/s10875-024-01840-4
Enrico Drago, Francesca Fioredda, Federica Penco, Ignazia Prigione, Arinna Bertoni, Genny Del Zotto, Paola Bocca, Erika Massaccesi, Marina Lanciotti, Daniele Moratto, Lorenz Thurner, Roberta Caorsi, Marco Gattorno, Stefano Volpi
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引用次数: 0

摘要

儿童多系统炎症综合征(MIS-C)已在先天性免疫错误(IEI)患者中有所报道,这为我们了解疾病的发病机制提供了线索。这里,我们介绍了首例威斯科特-阿尔德里奇综合征(Wiskott-Aldrich syndrome,WAS)基因突变患儿的多系统炎症综合征(MIS-C)病例,阐明了潜在的易感因素和所涉及的炎症通路。遗传分析发现,WAS基因存在一个框移截断变异,导致WAS蛋白表达介于轻度和重度之间,尽管临床表型类似于X连锁血小板减少症(XLT)。与健康受试者相比,MIS-C期间患者外周血单核细胞在LPS刺激下分泌的IL-1β较低,但在随访期间分泌量有所增加。相反,急性期患者循环单核细胞中 ASC(凋亡相关斑点样蛋白,含 CARD)斑点的百分比高于健康人。MIS-C 期间的 I 型干扰素(IFN)特征正常,而在远离急性期的地方测得的 IFN 特征则升高。该病例证实了 IEI 与 MIS-C 的关联,这可能与对 SARS-CoV-2 的延迟免疫反应有关。XLT 表型是一种亚临床免疫调节,涉及 NLRP3 炎症小体和 I 型 IFN 反应。
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Inborn Error of WAS Presenting with SARS-CoV-2-Related Multisystem Inflammatory Syndrome in Children.

Multisystem inflammatory syndrome in children (MIS-C) has been reported in patients with inborn errors of immunity (IEI), providing insights into disease pathogenesis. Here, we present the first case of MIS-C in a child affected by Wiskott-Aldrich syndrome (WAS) gene mutation, elucidating underlying predisposing factors and the involved inflammatory pathways. Genetic analysis revealed a frameshift truncating variant in the WAS gene, resulting in WAS protein expression between mild and severe forms, despite a clinical phenotype resembling X-linked thrombocytopenia (XLT). IL-1β secretion by LPS-stimulated peripheral blood mononuclear cells from patient during MIS-C was lower compared to healthy subjects but increased during follow-up. Conversely, the percentage of ASC (apoptosis-associated speck-like protein containing a CARD) specks in the patient's circulating monocytes during the acute phase was higher than in healthy subjects. The type I interferon (IFN) signature during MIS-C was normal, in contrast to the raised IFN signature measured far from the acute event. This case supports the association of IEI with MIS-C, potentially linked to delayed immune responses to SARS-CoV-2. The XLT phenotype underlies a subclinical immunodysregulation involving the NLRP3 inflammasome and the type-I IFN response.

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来源期刊
CiteScore
12.20
自引率
9.90%
发文量
218
审稿时长
2 months
期刊介绍: The Journal of Clinical Immunology publishes impactful papers in the realm of human immunology, delving into the diagnosis, pathogenesis, prognosis, or treatment of human diseases. The journal places particular emphasis on primary immunodeficiencies and related diseases, encompassing inborn errors of immunity in a broad sense, their underlying genotypes, and diverse phenotypes. These phenotypes include infection, malignancy, allergy, auto-inflammation, and autoimmunity. We welcome a broad spectrum of studies in this domain, spanning genetic discovery, clinical description, immunologic assessment, diagnostic approaches, prognosis evaluation, and treatment interventions. Case reports are considered if they are genuinely original and accompanied by a concise review of the relevant medical literature, illustrating how the novel case study advances the field. The instructions to authors provide detailed guidance on the four categories of papers accepted by the journal.
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