A Guide for Initiating and Managing Chimeric Antigen Receptor T Cell Therapy Clinical Trials in Autoimmune Rheumatic Diseases

作者：Roberto Caricchio, Stacie Bell, Sasha Bernatsky, Maria Dall’Era, David H. Goddard, Kenneth Kalunian, Alfred H.J. Kim, Fotios Koumpouras, Jose Rubio, Amit Saxena, Saira Z. Sheikh · 发表于：ACR Open Rheumatology · 年份：2025 · DOI：10.1002/acr2.70139 · 被引用次数：3 · 研究领域：CAR-T cell therapy research、Biosimilars and Bioanalytical Methods、Biomedical Ethics and Regulation

Chimeric antigen receptor (CAR) T-cell therapy, long transformative in oncology, is now rapidly emerging as a frontier in autoimmune rheumatic diseases, particularly systemic lupus erythematosus (SLE), driven by accumulating evidence of deep B-cell depletion, immune "resetting," and durable drug-free remission in early studies, yet its translation into rheumatology demands mastery of formidable logistical, regulatory, clinical, and ethical complexities that span institutional readiness, multidisciplinary team formation, stringent regulatory compliance, sophisticated operational workflows, comprehensive patient selection and education, meticulous clinical management of both classical toxicities (CRS, ICANS, ICAHT) and autoimmune-specific reactions such as LICATS, robust financial and resource planning, and long-term follow-up extending 15 years or more; successful implementation requires coordinated expertise among rheumatologists, hematologist-oncologists, cellular therapy units, pharmacists, research coordinators, and ICU-capable teams, all embedded within disciplined communication structures, harmonized SOPs, validated PROs, biorepository governance frameworks, and adherence to national and international cellular therapy standards; in parallel, investigators must anticipate bottlenecks such as apheresis access, manufacturing slot scarcity, competing trial enrollment, fluctuating SLE phenotypes, and heterogeneity-driven signal variability, while sustaining patient engagement...