{"title":"托法替尼单药治疗青少年皮肌炎维持治疗1例。","authors":"Delia Argüelles Balas, Estefanía Barral Mena, M Ángeles Martín Díaz, Enrique Calvo-Aranda","doi":"10.1136/rmdopen-2025-005651","DOIUrl":null,"url":null,"abstract":"<p><p>Juvenile dermatomyositis (JDM) is a multisystem autoimmune disease associated with considerable morbidity and mortality, having a significant impact on patient health. Early diagnosis and appropriate treatment play a key role in optimising outcomes. JDM management remains challenging, particularly in severe or refractory cases, due to the limited evidence regarding therapeutic options. Given these challenges, Janus kinase inhibitors, including tofacitinib, have emerged as promising alternatives, yet evidence in paediatric populations is still scarce. We present the case of a child with JDM who achieved sustained remission with tofacitinib monotherapy following the failure of previous therapies. While its use is currently restricted to refractory cases, these findings suggest its potential for earlier integration into treatment algorithms. This work aims to provide valuable insights to guide therapeutic strategies in JDM.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"11 2","pages":""},"PeriodicalIF":5.1000,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12097033/pdf/","citationCount":"0","resultStr":"{\"title\":\"Tofacitinib monotherapy as maintenance treatment in juvenile dermatomyositis: a case report.\",\"authors\":\"Delia Argüelles Balas, Estefanía Barral Mena, M Ángeles Martín Díaz, Enrique Calvo-Aranda\",\"doi\":\"10.1136/rmdopen-2025-005651\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Juvenile dermatomyositis (JDM) is a multisystem autoimmune disease associated with considerable morbidity and mortality, having a significant impact on patient health. Early diagnosis and appropriate treatment play a key role in optimising outcomes. JDM management remains challenging, particularly in severe or refractory cases, due to the limited evidence regarding therapeutic options. Given these challenges, Janus kinase inhibitors, including tofacitinib, have emerged as promising alternatives, yet evidence in paediatric populations is still scarce. We present the case of a child with JDM who achieved sustained remission with tofacitinib monotherapy following the failure of previous therapies. While its use is currently restricted to refractory cases, these findings suggest its potential for earlier integration into treatment algorithms. This work aims to provide valuable insights to guide therapeutic strategies in JDM.</p>\",\"PeriodicalId\":21396,\"journal\":{\"name\":\"RMD Open\",\"volume\":\"11 2\",\"pages\":\"\"},\"PeriodicalIF\":5.1000,\"publicationDate\":\"2025-05-21\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12097033/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"RMD Open\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1136/rmdopen-2025-005651\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"RHEUMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"RMD Open","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1136/rmdopen-2025-005651","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"RHEUMATOLOGY","Score":null,"Total":0}
Tofacitinib monotherapy as maintenance treatment in juvenile dermatomyositis: a case report.
Juvenile dermatomyositis (JDM) is a multisystem autoimmune disease associated with considerable morbidity and mortality, having a significant impact on patient health. Early diagnosis and appropriate treatment play a key role in optimising outcomes. JDM management remains challenging, particularly in severe or refractory cases, due to the limited evidence regarding therapeutic options. Given these challenges, Janus kinase inhibitors, including tofacitinib, have emerged as promising alternatives, yet evidence in paediatric populations is still scarce. We present the case of a child with JDM who achieved sustained remission with tofacitinib monotherapy following the failure of previous therapies. While its use is currently restricted to refractory cases, these findings suggest its potential for earlier integration into treatment algorithms. This work aims to provide valuable insights to guide therapeutic strategies in JDM.
期刊介绍:
RMD Open publishes high quality peer-reviewed original research covering the full spectrum of musculoskeletal disorders, rheumatism and connective tissue diseases, including osteoporosis, spine and rehabilitation. Clinical and epidemiological research, basic and translational medicine, interesting clinical cases, and smaller studies that add to the literature are all considered.