Abstract:

Objective　To observe the efficacy and safety of tocilizumab against refractory systemic onset juvenile idiopathic arthritis（SoJIA）. Methods　A prospective follow-up study of 16 patients with refractory active SoJIA patients with or with tocilizumab after treatment of clinical disease activity indicators and safety. The ANOVA was used for statistical analysis. Results　After 2 weeks，12 weeks and 52 weeks of treatment，the levels of white blood cells，erythrocyte sedimentation rate and hypersensitive C-reactive protein were significantly decreased，and there were statistically significant differences（F＝26.25、145.70、517.96，P＜0.05）. JADAS27 was significantly decreased after 2 weeks，12 weeks and 52 weeks of treatment with tocilizumab（the score being 23.09±3.46，8.19±2.63，4.25±2.86 and 2.63±1.54），the difference being statistically significant（P＜0.05）. Some children had adverse reactions，2 cases of skin abnormalities，1 case of white pityriasis，1 case of skin infection；3 cases of liver enzyme abnormalities；2 cases of leukopenia. Conclusion　Tocilizumab can improve the condition of children with SoJIA in rapid and obvious way，and medium and long-term security and tolerance was good. The duration of tocilizumab therapy should be carefully assessed.

Key words: tocilizumab, children, refractory, systemic onset juvenile idiopathic arthritis

摘要：

目的　观察托珠单抗对难治性全身型幼年特发性关节炎的中长期疗效及安全性。方法　前瞻性研究2015年1月至2016年4月西安交通大学附属儿童医院免疫科住院的16例活动期难治性全身型幼年特发性关节炎患儿改用或加用托珠单抗治疗52周后临床病情活动指标及安全性。对所有观察指标采用SPSS17.0统计软件分析。结果　托珠单抗治疗2周、12周、52周后与治疗前比较，实验室指标提示白细胞（WBC）、红细胞沉降率（ESR）、超敏C反应蛋白（hsCRP）均有显著下降，差异具有统计学意义（F＝26.25、145.70、517.96，P＜0.05）。经托珠单抗治疗前及治疗2周、12周、52周后JADAS 27分别为（23.09±3.46）分、（8.19±2.63）分、（4.25±2.86）分、（2.63±1.54）分均明显下降，差异有统计学意义（P＜0.05）。部分患儿出现不良反应，2例出现皮肤异常，1例白色糠疹，1例皮肤感染；3例出现肝酶异常；2例出现WBC降低。结论　托珠单抗能快速且明显改善难治性全身型幼年特发性关节炎患儿病情，中长期安全性及耐受性良好。托珠单抗治疗疗程周期应谨慎评估。

关键词: 托珠单抗, 儿童, 难治性, 全身型幼年特发性关节炎