Department of Rheumatology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No. 56 South Lishi Road, Xicheng District, Beijing, 100045, China.
Department of Rheumatology and Immunology, Children's Hospital of Chongqing Medical University, Chongqing, China.
Clin Rheumatol. 2024 Nov;43(11):3457-3467. doi: 10.1007/s10067-024-07126-9. Epub 2024 Sep 16.
Given the limited tocilizumab (TCZ) treatment data for systemic juvenile idiopathic arthritis (sJIA) in China, we evaluated the long-term efficacy and safety of TCZ in Chinese patients with sJIA.
In this multicentre, interventional Phase IV study, patients with sJIA and inadequate clinical response to non-steroidal anti-inflammatory drugs/corticosteroids received TCZ infusions every 2 weeks based on body weight (< 30 kg, 12 mg/kg; ≥ 30 kg, 8 mg/kg), over a 52-week open-label period and an 8-week safety follow-up period. The primary endpoint was the proportion of patients with a JIA American College of Rheumatology (ACR) 30 response and absence of fever at Week 12.
Sixty-two patients were enrolled and treated (12-mg/kg group, 34; 8-mg/kg group, 28). At Week 12, 87.1% (95% confidence interval 78.8%-95.4%) of patients had JIA ACR 30 response and absence of fever; Week 52 results were similar. The proportion of JIA ACR 30/50/70/90 responders rapidly increased at Week 12, up to Week 52. High-sensitivity C-reactive protein (hsCRP) levels decreased within 4 weeks; 44/58 patients (75.9%) with elevated baseline hsCRP recovered at Week 52. Childhood Health Assessment Questionnaire pain scores, disability index scores, and mean corticosteroid dose decreased over time. Height standard deviation score changes at Week 52 indicated catch-up growth. Most adverse events (AEs) were mild (serious AE incidence, 17.7%). No deaths or macrophage activation syndrome occurred.
This is the first multicentre trial to report the efficacy and safety of TCZ in Chinese patients with sJIA at 52 weeks. No new safety concerns were found.
鉴于中国系统性幼年特发性关节炎（sJIA）患者接受托珠单抗（TCZ）治疗的数据有限，我们评估了 TCZ 在中国 sJIA 患者中的长期疗效和安全性。
在这项多中心、干预性 IV 期研究中，对非甾体抗炎药/皮质类固醇治疗反应不足的 sJIA 患者，根据体重（<30kg，12mg/kg；≥30kg，8mg/kg），每 2 周接受 TCZ 输注，治疗 52 周的开放性标签期和 8 周的安全性随访期。主要终点是第 12 周时达到 JIA 美国风湿病学会（ACR）30 缓解且无发热的患者比例。
共纳入并治疗了 62 例患者（12-mg/kg 组 34 例，8-mg/kg 组 28 例）。第 12 周时，87.1%（95%置信区间 78.8%-95.4%）的患者达到 JIA ACR 30 缓解且无发热；第 52 周的结果相似。第 12 周时 JIA ACR 30/50/70/90 应答者的比例迅速增加，直至第 52 周。高敏 C 反应蛋白（hsCRP）水平在 4 周内下降；第 52 周时，58 例基线 hsCRP 升高的患者中有 44 例（75.9%）恢复正常。儿童健康评估问卷疼痛评分、残疾指数评分和平均皮质类固醇剂量随时间逐渐降低。第 52 周时身高标准差评分的变化表明追赶生长。大多数不良事件（AE）为轻度（严重 AE 发生率 17.7%）。无死亡或巨噬细胞活化综合征发生。
这是第一项报告 TCZ 在 52 周时中国 sJIA 患者疗效和安全性的多中心试验。未发现新的安全性问题。
