中国实用儿科杂志 ›› 2026, Vol. 41 ›› Issue (8): 685-689.DOI: 10.19538/j.ek2026080615

• 专题笔谈 • 上一篇    下一篇

嵌合抗原受体T在儿童风湿免疫性疾病中的应用

  

  1. 浙江大学医学院附属儿童医院 风湿免疫过敏科,浙江  杭州  310000
  • 出版日期:2026-08-06 发布日期:2026-09-17
  • 通讯作者: 电子信箱:meipinglu@126.zju.edu.cn
  • 基金资助:
    国家自然科学基金(82570052);浙江省“尖兵”“领雁”研发攻关计划(2023C03032)

Application of chimeric antigen receptor T cells in pediatric rheumatic and immune diseases   

  1. Rheumatology Immunology and Allergy,Children’s Hospital Zhejiang University School of Medicine,Hangzhou 310000,China 
  • Online:2026-08-06 Published:2026-09-17

摘要: 儿童风湿性疾病是一组以免疫功能紊乱为基础、可累及多系统和多器官的疾病,主要包括幼年特发性关节炎(JIA)、儿童系统性红斑狼疮(SLE)、幼年皮肌炎(JDM)、系统性硬化病(SSc)、干燥综合征(SS)及血管炎等。B 细胞异常活化、自身抗体产生及体液免疫紊乱在多种儿童风湿性疾病的发生发展中发挥重要作用,也为靶向B 细胞治疗提供了理论依据。近年来,嵌合抗原受体T(CAR-T)细胞疗法在自身免疫性疾病中的应用受到关注,尤其是靶向CD19、B 细胞成熟抗原等B 细胞相关分子的 CAR-T 细胞疗法,可通过清除异常活化或致病性B 细胞、减少自身抗体产生并促进免疫稳态重建,为重症、难治或复发性儿童风湿性疾病提供了新的治疗思路。CAR-T 在儿童风湿免疫性疾病中的应用仍处于早期临床探索阶段,现有证据主要来自个案报道、小样本病例系列、医院豁免项目及早期临床研究,尚缺乏随机对照研究和长期随访数据,其疗效持久性、复发风险及远期安全性仍需进一步验证。

关键词: 风湿免疫性疾病, 儿童, 嵌合抗原受体T 细胞疗法, 靶向治疗

Abstract: Pediatric rheumatic diseases comprise a group of immune dysregulation–mediated disorders that may involve multiple systems and organs,including juvenile idiopathic arthritis(JIA),childhood-onset systemic lupus erythematosus(cSLE),juvenile dermatomyositis(JDM),systemic sclerosis(SSc),Sjögren’s syndrome(SS),and vasculities.Aberrant B-cell activation,autoantibody production,and dysregulated humoral immunity play pivotal roles in the occurrence and progression of various pediatric rheumatic diseases,thereby providing theoretical evidence for B-cell–targeted therapies.In recent years,chimeric antigen receptor T-cell(CAR-T)therapy has attracted increasing attention in the treatment of autoimmune diseases.In particular,CAR-T therapies targeting B-cell–associated molecules,such as CD19 and B-cell maturation antigen(BCMA),offer a novel therapeutic strategy for severe,refractory,or relapsing pediatric rheumatic diseases in children through eliminating aberrantly activated or pathogenic B cells,reducing autoantibody production,and facilitating the restoration of immune homeostasis. Nevertheless,the application of CAR-T therapy in pediatric rheumatic and immune-mediated diseases remains at an early stage of clinical investigation.Current evidence is largely derived from case reports,small case series,hospital-exemption programs,and early-phase clinical studies. Randomized controlled trials and long-term follow-up data are still lacking;therefore,the durability of therapeutic responses,risk of relapse,and long-term safety require further validation.

Key words: rheumatic immune diseases, child, chimeric antigen receptor T-cell therapy, targeted therapy