中国实用儿科杂志 ›› 2026, Vol. 41 ›› Issue (8): 650-653.DOI: 10.19538/j.ek2026080607

• 专题笔谈 • 上一篇    下一篇

Janus激酶抑制剂在儿童风湿免疫性疾病中的应用

  

  1. 中国医学科学院北京协和医学院 北京协和医院,北京  100730
  • 出版日期:2026-08-06 发布日期:2026-09-17
  • 通讯作者: 马明圣,电子信箱:mamingsheng@pumch.cn
  • 基金资助:
    协和人才培育计划 C 类项目(UBJ10837)

Janus kinase inhibitors in pediatric rheumatic and immune diseases

  1. Peking Union Medical College Hospital,Peking Union Medical College,Chinese Academy of Medical Sciences,Beijing  100730,China
  • Online:2026-08-06 Published:2026-09-17

摘要: Janus 激酶(JAK)抑制剂通过调控 JAK- 信号转导及转录激活因子(STAT)通路影响多种细胞因子信号,为儿童风湿免疫性疾病提供了区别于传统免疫抑制剂和单靶点生物制剂的新选择。目前托法替布和巴瑞替尼在非全身型幼年特发性关节炎中已有较明确随机对照试验证据,乌帕替尼在儿科中应用证据正在积累。在幼年皮肌炎中,JAK 抑制剂可作为难治性或特定类型患者的个体化靶向治疗选择,尤其是皮肤病变明显、Ⅰ型干扰素特征突出、合并间质性肺病等患儿;而在儿童系统性红斑狼疮中的应用仍以难治、复发、激素依赖或特殊高炎症表现的患儿为主;此外可用于Ⅰ型干扰素病及风湿免疫病继发巨噬细胞活化综合征。目前临床应用 JAK 抑制剂时,基于疾病表型、炎症通路、既往治疗反应和个体风险进行分层决策,未来在儿童患者的应用将重视血药浓度监测及个体化剂量精准调整。

关键词: font-family:", font-size:17px, background-color:#FFFFFF, ">JAK 抑制剂, 儿童, 风湿免疫性疾病, 血药浓度监测

Abstract: Janus kinase inhibitors provide a new therapeutic option distinct from conventional immunosuppressants and single target biologics for pediatric rheumatic and immune diseases by modulating multiple cytokine signals through the JAK-STAT pathway. Currently,there have been clear evidence of randomized controlled trials for tofacitinib and baricitinib in non-systemic juvenile idiopathic arthritis,while evidence for upadacitinib in polyarticular juvenile idiopathic arthritis is accumulating. In juvenile dermatomyositis,JAK inhibitors may be considered as individualized targeted therapeutic options for patients with refractory disease or specific phenotypes,particularly for children with prominent cutaneous involvement,marked type I interferon activation,or concomitant interstitial lung disease. In childhood onset systemic lupus erythematosus,their use remains mainly confined to refractory,relapsing,steroid-dependent or specific hyperinflammatory phenotypes. Besides,JAK inhibitors can also be used in type I interferonopathies and macrophage activation syndrome secondary to rheumatic and immune diseases.Clinical application of JAK inhibitors should be guided by stratified decision-making based on disease phenotype,inflammatory pathways,previous treatment response and individual risk profiles. In the future,their use in child patients should focus on monitoring of blood drug concentration and precise adjustment of individualized dose.

Key words: JAK inhibitors, child, rheumatic and immune disorders, blood drug concentration monitoring