| 摘要: |
| 目的评价托珠单抗治疗全身型幼年特发性关节炎（SoJIA）的有效性与安全性。方法选择难治性或有巨噬细胞活化综合征特征的SoJIA7例，2016年1月1日之前收住的4例患儿均予每4周静脉滴注1次托珠单抗；2016年1月1日之后收住的3例患儿均予每2周静脉滴注1次托珠单抗，持续6次后若疾病维持非活动状态，减量为每4周静脉滴注1次托珠单抗。观察治疗前、治疗后4、8周的肿胀关节数、压痛关节数、活动受限关节数、ESR、C反应蛋白（CRP）、铁蛋白、Hb、激素使用剂量等指标，记录随访期间所有不良事件。结果治疗后8周肿胀关节数、压痛关节数、活动受限关节数均较治疗前明显减少，ESR、CRP均较治疗前明显下降，Hb较治疗前上升，差异均有统计学意义（均P＜0.05）。治疗后12周平均激素剂量减少57.94%。7例患儿共输注101次托珠单抗，发生可能与药物相关的不良事件8次，发生率为7.9％。7例累积托珠单抗使用时间为6.76年，共出现27次感染，年发生率为3.99次/年，均为轻度或中度感染，最常见为上呼吸道感染。7例患者中，5例处于疾病非活动状态，完全缓解6个月以上4例。结论托珠单抗联合抗风湿药物治疗SoJIA临床起效快，持续缓解率高，安全性良好。 |
| 关键词: 全身型幼年特发性关节炎 托珠单抗 疗效 |
| DOI：10.12056/j.issn.1006-2785.2017.39.21.2017-1594 |
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| Efficacy and safety of tocilizumab in treatment of systemic juvenile idiopathic arthritis |
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the Second Affiliated Hospital of Wenzhou Medical University
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| Abstract: |
| Objective To assess the efficacy and safety of tocilizumab (TCZ) in treatment of systemic juvenile idiopathic arthritis (SoJIA). Methods Seven patients with refractory SoJIA or with SoJIA of macrophage activation syndrome (MAS) were enrolled. TCZ was initiated intravenously every 4 weeks in 4 patients who were admitted before January 2016, and every 2 weeks in 3 patients who were admitted after January 2016. If patients maintained inactive disease after 6 injections, the TCZ were administrated every 4 weeks. The number of swollen joints, tender joints, joints with limited range of motion were documented, and the levels of ESR, C-reactive protein (CRP), ferritin, hemoglobin were measured before treatment, 4 and 8 weeks after treatment. The adverse events were recorded during follow-up period. Results At 8 weeks after treatment the number of
swollen joints, tender joints, joints with limited range of motion were significantly reduced, the levels of ESR, CRP were significantly decreased and the level of hemoglobin was significantly increased (all P＜0.05). At 12 weeks after treatment the mean dose of glucocorticoid decreased by 57.94%. There were 8 adverse events probably associated with TCZ among 101
injections with an incidence rate of 7.9% . Cumulative usage time of TCZ in 7 patients was 6.76 years, and 27 infections occurred, most of which were mild to moderate, and upper respiratory infection was the most common one. Among 7 patients,
5 cases achieved inactive condition and 4 cases achieved disease remission. Conclusion Tocilizumab combined with DMARDs is safe and effective in treatment of children with SoJIA. |
| Key words: Systemic onset juvenile idiopathic arthritis Tocilizumab Effect |
