中国实用儿科杂志

中国实用儿科杂志 ›› 2021, Vol. 36 ›› Issue (11): 853-857.DOI: 10.19538/j.ek2021110611

• 党为人民谋健康的 100 年·专题笔谈 • 上一篇    下一篇

小分子靶向药JAK抑制剂在儿童风湿免疫性疾病中的应用

  

  1. 1.上海市儿童医院  上海交通大学附属儿童医院肾脏风湿免疫科，上海  200040；2.广州市妇女儿童医疗中心，广东  广州  510180
  • 出版日期:2021-11-06 发布日期:2022-01-04
  • 通讯作者: 黄文彦，电子信箱：hwy65@hotmail.com；曾华松，电子信箱：huasongxuqing@163.com
  • 基金资助:

    上海交通大学“交大之星”计划医工交叉研究基金（YG2021QN118）；上海交通大学医学院儿科学院教学类科研课题（EKJX2020013DGD）；上海市儿童医院院教课题（2020YGZM01）

JAK inhibitors as small molecule targeted agents in the treatment of children with rheumatic and immune diseases

  1. *Department of Nephrology，Rheumatology and and Immunology，Shanghai Children’s Hospital，Shanghai Jiao Tong University，Shanghai  200040，China
  • Online:2021-11-06 Published:2022-01-04

摘要： Janus激酶（Januskinases，JAK）抑制剂是一种小分子靶向药，可以抑制相关炎症因子信号传导通路而具备抗炎作用，目前已有多种JAK抑制剂上市用于包括风湿免疫性疾病在内的各系统疾病。作为新型靶向合成类改善病情抗风湿药物，JAK抑制剂已上市用于甲氨喋呤应答不足或不耐受的成人类风湿性关节炎，且在成人强直性脊柱炎、溃疡性结肠炎、皮肌炎、血管炎等多种风湿性疾病中均有应用，但在儿童风湿免疫性疾病中仍缺乏大规模临床证据。JAK抑制剂治疗幼年特发性关节炎已进入Ⅲ期临床试验，在治疗幼年型皮肌炎、系统性红斑狼疮、多发性大动脉炎也有报道，且是相关自身炎症性疾病的靶向药物。因此，JAK抑制剂在儿童风湿免疫性疾病中的应用具有广泛前景。

关键词: 小分子靶向药, JAK抑制剂, 儿童风湿病, 自身炎症性疾病

Abstract: JAK inhibitors are small molecule targeted agents that inhibit signal transduction pathways associated with inflammatory factors to get anti-inflammatory effects. Currently，many kinds of JAK inhibitors have been used in the treatment of diseases in each system， including rheumatic and immune diseases. As novel targeted synthetic drugs，JAK inhibitors have been used for adults with rheumatoid arthritis，ankylosing spondylitis，ulcerative colitis，dermatomyositis，vasculitis，and other rheumatic disease. However，there is still insufficient clinical evidence in children with rheumatic and immune diseases. JAK inhibitors for juvenile idiopathic arthritis have entered phase 3 clinical trials，and some JAK inhibitors have been reported to be used in the treatment of juvenile dermatomyositis，systemic lupus erythematosus，multiple arteritis. JAK inhibitors are also targeted agents for some autoinflammatory diseases. Therefore，JAK inhibitors have a great prospect in the treatment of rheumatic and immune diseases in children.

Key words: small molecule targeted agent, JAK inhibitor, pediatric rheumatic disease, autoinflammatory disease