Suppr超能文献

WAS 相关 SARS-CoV-2 相关儿童多系统炎症综合征的先天错误。

Inborn Error of WAS Presenting with SARS-CoV-2-Related Multisystem Inflammatory Syndrome in Children.

机构信息

Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), Università degli Studi di Genova, Genova, Italy.

UOC Reumatologia e Malattie Autoinfiammatorie, IRCCS Istituto Giannina Gaslini, Genova, Italy.

出版信息

J Clin Immunol. 2024 Nov 25;45(1):49. doi: 10.1007/s10875-024-01840-4.

Abstract

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.

摘要

儿童多系统炎症综合征(MIS-C)已在固有免疫缺陷(IEI)患者中报道,为疾病发病机制提供了深入了解。在这里,我们报告了首例由 Wiskott-Aldrich 综合征(WAS)基因突变引起的 MIS-C 病例,阐明了潜在的易患因素和涉及的炎症途径。基因分析显示 WAS 基因发生框移移码突变,导致 WAS 蛋白表达处于轻度和重度之间,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 LPS 刺激的患者外周血单核细胞中 IL-1β 的分泌较低,但在随访期间增加。相反,在急性期患者循环单核细胞中 ASC(含 CARD 的凋亡相关斑点样蛋白)斑点的百分比高于健康受试者。MIS-C 期间的 I 型干扰素(IFN)特征正常,与远离急性事件测量的升高 IFN 特征相反。该病例支持 IEI 与 MIS-C 的关联,可能与 SARS-CoV-2 后延迟的免疫反应有关。XLT 表型潜在地涉及 NLRP3 炎症小体和 I 型 IFN 反应的亚临床免疫失调。

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验