Anushka Aggarwal, Maha Almackenzie, Rohit Aggarwal
{"title":"Role of CD19 Chimeric Antigen Receptor T Cell Therapy in Idiopathic Inflammatory Myopathies.","authors":"Anushka Aggarwal, Maha Almackenzie, Rohit Aggarwal","doi":"10.3899/jrheum.2024-1115","DOIUrl":null,"url":null,"abstract":"<p><p>Idiopathic inflammatory myopathies (IIMs) comprise a spectrum of autoinflammatory disease characterized primarily by muscle inflammation, with secondary involvement of diverse organs including joints, skin, lungs, heart, and the gastrointestinal system. Managing these conditions poses considerable challenges, often inflicting profound distress on the afflicted individuals. Encouragingly, the deployment of chimeric antigen receptor (CAR) T cell therapy has demonstrated promising efficacy across various autoimmune diseases, extending hope for ameliorating the burden of IIM. This review provides an overview of the role of B cells in IIM pathogenesis, currently available B cell-depleting therapies, reasons for their lack of efficacy, and the application of CD19 CAR T cell therapy in the management of IIM, encompassing indications, efficacy, and tolerability profiles in these patient populations. Through this comprehensive review, we propose clinical trial design, target population, response criteria, and long-term follow-up measures for future clinical trials focusing on CD19 CAR T cell therapy in IIM. This overview aims to streamline research efforts and enhance the efficacy of therapeutic interventions in challenging cases.</p>","PeriodicalId":50064,"journal":{"name":"Journal of Rheumatology","volume":" ","pages":""},"PeriodicalIF":3.6000,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Rheumatology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3899/jrheum.2024-1115","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"RHEUMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Idiopathic inflammatory myopathies (IIMs) comprise a spectrum of autoinflammatory disease characterized primarily by muscle inflammation, with secondary involvement of diverse organs including joints, skin, lungs, heart, and the gastrointestinal system. Managing these conditions poses considerable challenges, often inflicting profound distress on the afflicted individuals. Encouragingly, the deployment of chimeric antigen receptor (CAR) T cell therapy has demonstrated promising efficacy across various autoimmune diseases, extending hope for ameliorating the burden of IIM. This review provides an overview of the role of B cells in IIM pathogenesis, currently available B cell-depleting therapies, reasons for their lack of efficacy, and the application of CD19 CAR T cell therapy in the management of IIM, encompassing indications, efficacy, and tolerability profiles in these patient populations. Through this comprehensive review, we propose clinical trial design, target population, response criteria, and long-term follow-up measures for future clinical trials focusing on CD19 CAR T cell therapy in IIM. This overview aims to streamline research efforts and enhance the efficacy of therapeutic interventions in challenging cases.
特发性炎症性肌病(IIMs)包括一系列以肌肉炎症为主要特征的自身炎症性疾病,继发累及多种器官,包括关节、皮肤、肺、心脏和胃肠道系统。管理这些情况带来了相当大的挑战,往往对受折磨的人造成深刻的痛苦。令人鼓舞的是,嵌合抗原受体(CAR) T细胞治疗在各种自身免疫性疾病中显示出有希望的疗效,为减轻IIM的负担带来了希望。本文综述了B细胞在IIM发病机制中的作用,目前可用的B细胞消耗疗法,其缺乏疗效的原因,以及CD19 CAR - T细胞疗法在IIM治疗中的应用,包括这些患者群体的适应症,疗效和耐受性。通过这篇全面的综述,我们提出了临床试验设计、目标人群、反应标准和长期随访措施,以关注CD19 CAR - T细胞治疗IIM的未来临床试验。本综述旨在简化研究工作,提高治疗干预在具有挑战性的情况下的疗效。
期刊介绍:
The Journal of Rheumatology is a monthly international serial edited by Earl D. Silverman. The Journal features research articles on clinical subjects from scientists working in rheumatology and related fields, as well as proceedings of meetings as supplements to regular issues. Highlights of our 41 years serving Rheumatology include: groundbreaking and provocative editorials such as "Inverting the Pyramid," renowned Pediatric Rheumatology, proceedings of OMERACT and the Canadian Rheumatology Association, Cochrane Musculoskeletal Reviews, and supplements on emerging therapies.