{"title":"重叠aicardii - gouti<e:1>综合征和Singleton-Merten综合征伴IFIH1杂合功能获得突变,模拟幼年特发性关节炎。","authors":"Susumu Yamazaki, Shuya Kaneko, Asami Shimbo, Hitoshi Irabu, Ryo Ogino, Takayuki Miyamoto, Kazushi Izawa, Yuko Segawa, Jun Kakizaki, Masaaki Mori, Masaki Shimizu","doi":"10.1080/25785826.2025.2479148","DOIUrl":null,"url":null,"abstract":"<p><p>Aicardi-Goutières syndrome (AGS) and Singleton-Merten syndrome (SMS) are associated with heterozygous gain-of-function mutations in the interferon induced with helicase C domain 1 (IFIH1) gene. Recent reports describe patients exhibiting overlapping clinical features of AGS and SMS, along with marked type I interferon (IFN) overproduction. However, the clinical characteristics and optimal treatment strategies remain unclear. Herein, we present a patient with overlapping clinical features of AGS and SMS who was initially misdiagnosed with juvenile idiopathic arthritis. Surgical soft tissue release of the hip and knee joints improved joint deformities and spastic paraparesis. Baricitinib effectively treated refractory chilblains and skin ulcers while reducing IFN-stimulated gene overexpression in peripheral blood. These findings indicate that baricitinib may be a safe and effective treatment for AGS-SMS overlap, and surgical intervention may be a viable option for refractory joint deformities with spastic paraparesis.</p>","PeriodicalId":37286,"journal":{"name":"Immunological Medicine","volume":" ","pages":"1-5"},"PeriodicalIF":2.7000,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Overlapping Aicardi-Goutières and Singleton-Merten syndromes with a heterozygous gain-of-function mutation in <i>IFIH1</i> mimicking juvenile idiopathic arthritis.\",\"authors\":\"Susumu Yamazaki, Shuya Kaneko, Asami Shimbo, Hitoshi Irabu, Ryo Ogino, Takayuki Miyamoto, Kazushi Izawa, Yuko Segawa, Jun Kakizaki, Masaaki Mori, Masaki Shimizu\",\"doi\":\"10.1080/25785826.2025.2479148\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Aicardi-Goutières syndrome (AGS) and Singleton-Merten syndrome (SMS) are associated with heterozygous gain-of-function mutations in the interferon induced with helicase C domain 1 (IFIH1) gene. Recent reports describe patients exhibiting overlapping clinical features of AGS and SMS, along with marked type I interferon (IFN) overproduction. However, the clinical characteristics and optimal treatment strategies remain unclear. Herein, we present a patient with overlapping clinical features of AGS and SMS who was initially misdiagnosed with juvenile idiopathic arthritis. Surgical soft tissue release of the hip and knee joints improved joint deformities and spastic paraparesis. Baricitinib effectively treated refractory chilblains and skin ulcers while reducing IFN-stimulated gene overexpression in peripheral blood. These findings indicate that baricitinib may be a safe and effective treatment for AGS-SMS overlap, and surgical intervention may be a viable option for refractory joint deformities with spastic paraparesis.</p>\",\"PeriodicalId\":37286,\"journal\":{\"name\":\"Immunological Medicine\",\"volume\":\" \",\"pages\":\"1-5\"},\"PeriodicalIF\":2.7000,\"publicationDate\":\"2025-03-21\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Immunological Medicine\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1080/25785826.2025.2479148\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"IMMUNOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Immunological Medicine","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1080/25785826.2025.2479148","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"IMMUNOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
艾卡迪-古铁雷斯综合征(AGS)和辛格顿-默顿综合征(SMS)与干扰素诱导螺旋酶 C 结构域 1(IFIH1)基因的杂合功能增益突变有关。最近的报告描述,患者表现出 AGS 和 SMS 的重叠临床特征,并伴有明显的 I 型干扰素(IFN)过度分泌。然而,其临床特征和最佳治疗策略仍不明确。在本文中,我们介绍了一名具有 AGS 和 SMS 重叠临床特征的患者,他最初被误诊为幼年特发性关节炎。髋关节和膝关节软组织松解手术改善了关节畸形和痉挛性截瘫。巴利昔尼有效治疗了难治性皲裂和皮肤溃疡,同时降低了外周血中IFN刺激基因的过度表达。这些研究结果表明,巴利昔尼可能是治疗AGS-SMS重叠症的一种安全有效的方法,对于难治性关节畸形和痉挛性截瘫,手术干预可能是一种可行的选择。
Overlapping Aicardi-Goutières and Singleton-Merten syndromes with a heterozygous gain-of-function mutation in IFIH1 mimicking juvenile idiopathic arthritis.
Aicardi-Goutières syndrome (AGS) and Singleton-Merten syndrome (SMS) are associated with heterozygous gain-of-function mutations in the interferon induced with helicase C domain 1 (IFIH1) gene. Recent reports describe patients exhibiting overlapping clinical features of AGS and SMS, along with marked type I interferon (IFN) overproduction. However, the clinical characteristics and optimal treatment strategies remain unclear. Herein, we present a patient with overlapping clinical features of AGS and SMS who was initially misdiagnosed with juvenile idiopathic arthritis. Surgical soft tissue release of the hip and knee joints improved joint deformities and spastic paraparesis. Baricitinib effectively treated refractory chilblains and skin ulcers while reducing IFN-stimulated gene overexpression in peripheral blood. These findings indicate that baricitinib may be a safe and effective treatment for AGS-SMS overlap, and surgical intervention may be a viable option for refractory joint deformities with spastic paraparesis.