Wobma Holly, Ardoin Stacy P, Bonifant Challice L, Cooper Jennifer C, Kim Hanna, Sadun Rebecca E, Lewandowski Laura, Keller Michael, Colbert Robert A, Edens Cuoghi, DeQuattro Kimberly, Driest Kyla, Shalen Julia, Stojkic Ivana, Knight Andrea, Annesley Colleen, Torok Kathryn S, Elgarten Caitlin W, Onishi Toshihiro, Jackson Shaun W, Prockop Susan, Shah Nirali N, Ardalan Kaveh, Lamb Margaret
Division of Immunology, Boston Children's Hospital, Boston, MA, USA.
Department of Paediatrics, Division of Rheumatology, Ohio State University, Columbus, OH, USA.
Nat Rev Rheumatol. 2025 Jul 2. doi: 10.1038/s41584-025-01272-3.
Initial success with B cell-targeted chimeric antigen receptor (CAR) T cells for the treatment of systemic lupus erythematosus and other rheumatic diseases has generated enthusiasm for the broad application of this technology outside of the field of oncology. Paediatric patients with severe rheumatic diseases require lifelong therapy with a substantial toxicity burden and a high cost of care. Paradigm-shifting treatments, including CAR T cells, are desperately needed. Although CAR T cell therapy shows promise for paediatric rheumatic diseases, there are unique aspects of care compared with adults, which require careful consideration and expertise. In response, we established the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group, comprising international experts in the fields of paediatric rheumatology, oncology and cellular therapy, immunology and nephrology, to address the challenges of introducing cell therapies to patients with paediatric-onset autoimmune diseases. Given the possible benefits, we advocate for the study of CAR T cells in paediatric patients with rheumatic diseases who carry a lifelong risk of morbidity and mortality from chronic illness and medication toxicity. As this patient population is relatively small, consensus around definitions of success, robust study of predictors of response and uniform assessment and reporting of toxicities are critical to advancing the field.
靶向B细胞的嵌合抗原受体(CAR)T细胞在治疗系统性红斑狼疮和其他风湿性疾病方面取得的初步成功,引发了人们对该技术在肿瘤学领域之外广泛应用的热情。患有严重风湿性疾病的儿科患者需要终身治疗,毒性负担大,护理成本高。迫切需要包括CAR T细胞在内的变革性治疗方法。尽管CAR T细胞疗法对儿科风湿性疾病显示出前景,但与成人相比,其护理有独特之处,需要仔细考虑并具备专业知识。为此,我们成立了综合多学科儿科自身免疫与细胞治疗(IMPACT)工作组,成员包括儿科风湿病学、肿瘤学与细胞治疗、免疫学和肾脏病学领域的国际专家,以应对将细胞疗法引入患有儿科自身免疫性疾病患者时所面临的挑战。鉴于可能带来的益处,我们主张对患有风湿性疾病的儿科患者进行CAR T细胞研究,这些患者因慢性病和药物毒性面临终身发病和死亡风险。由于这一患者群体相对较小,围绕成功定义达成共识、对反应预测因素进行有力研究以及对毒性进行统一评估和报告,对于推动该领域发展至关重要。
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