Department of Pediatrics, Turku University Hospital, Turku.
Department of Pediatrics, Kuopio University Hospital, Kuopio.
Rheumatology (Oxford). 2020 Apr 1;59(4):732-741. doi: 10.1093/rheumatology/kez291.
To evaluate the patterns of usage, efficacy and safety of tocilizumab in polyarticular JIA.
An observational study of 56 consecutive polyarticular JIA patients was conducted using patient charts and electronic JIA databases. Efficacy was assessed by tocilizumab survival, rates of low disease activity (LDA) and of inactive disease by 10-joint Juvenile Arthritis Disease Activity Score (JADAS-10), and of clinically inactive disease according to Wallace's preliminary criteria. Efficacy and rate of adverse events (AEs) were evaluated during a 24-month period after tocilizumab commencement.
Tocilizumab was started on average as third-line biological agent (median, range first- to fourth-line) at a median disease duration of 5.2 years (interquartile range 3.0-7.7). Survival rates were 82% at 12 months and 64% at 24 months. The reasons for discontinuation were inadequate treatment effect in 50%, AE plus inadequate treatment effect in 37.5% and AE alone in 12.5%. LDA (JADAS-10 ⩽3.9) was reached in 58% at 12 months and in 84% at 24 months, inactive disease (JADAS-10 ⩽0.7) in 19% and 44%, and clinically inactive disease in 28% and 46%, respectively. The rate of AEs was 200.9/100 patient years and of serious AEs 12.9/100 patient years.
Survival of tocilizumab was high and a large proportion of the treatment-resistant patients reached LDA at 12 months of treatment. The LDA rate continued to increase throughout 24 months. The rates of AEs and serious AEs were higher than in register studies but lower than in the originator study of tocilizumab.
评估托珠单抗在多关节型幼年特发性关节炎中的使用模式、疗效和安全性。
采用患者病历和电子幼年特发性关节炎数据库进行了一项 56 例连续多关节型幼年特发性关节炎患者的观察性研究。通过托珠单抗生存、低疾病活动度（LDA）和 10 个关节幼年特发性关节炎疾病活动度评分（JADAS-10）的无疾病发生率、根据 Wallace 初步标准的无临床疾病发生率评估疗效。在托珠单抗开始使用后的 24 个月内评估疗效和不良事件（AE）发生率。
托珠单抗平均作为三线生物制剂（中位数，范围一线至四线）开始使用，中位疾病病程为 5.2 年（四分位距 3.0-7.7）。12 个月和 24 个月的生存率分别为 82%和 64%。停药的原因分别为治疗效果不佳 50%、AE 加治疗效果不佳 37.5%和 AE 单独 12.5%。12 个月时达到 LDA（JADAS-10 ⩽3.9）的比例为 58%，24 个月时为 84%，达到无疾病（JADAS-10 ⩽0.7）的比例为 19%和 44%，达到临床无疾病的比例分别为 28%和 46%。AE 发生率为 200.9/100 患者年，严重 AE 发生率为 12.9/100 患者年。
托珠单抗的生存率较高，大量治疗抵抗患者在治疗 12 个月时达到 LDA。LDA 率在 24 个月内持续增加。AE 和严重 AE 的发生率高于登记研究，但低于托珠单抗原始研究。
