阿巴西普治疗幼年特发性关节炎的安全性和有效性：来自 PRINTO/PRCSG 登记处的结果。
Safety and effectiveness of abatacept in juvenile idiopathic arthritis: results from the PRINTO/PRCSG registry.
机构信息
Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, USA.
PRI Research, Bad Bramstedt, Germany.
出版信息
Rheumatology (Oxford). 2024 Sep 1;63(SI2):SI195-SI206. doi: 10.1093/rheumatology/keae025.
OBJECTIVE
The aim of this study was to report the interim 5-year safety and effectiveness of abatacept in patients with JIA in the PRINTO/PRCSG registry.
METHODS
The Abatacept JIA Registry (NCT01357668) is an ongoing observational study of children with JIA receiving abatacept; enrolment started in January 2013. Clinical sites enrolled patients with JIA starting or currently receiving abatacept. Eligible patients were assessed for safety (primary end point) and effectiveness over 10 years. Effectiveness was measured by clinical 10-joint Juvenile Arthritis Disease Activity Score (cJADAS10) in patients with JIA over 5 years. As-observed analysis is presented according to the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guidelines.
RESULTS
As of 31 March 2020, 587 patients were enrolled; 569 are included in this analysis (including 134 new users) with 1214.6 patient-years of safety data available. Over 5 years, the incidence rate (IR) per 100 patient-years of follow-up of serious adverse events was 5.52 (95% CI: 4.27, 7.01) and of events of special interest was 3.62 (95% CI: 2.63, 4.86), with 18 serious infections [IR 1.48 (95% CI: 0.88, 2.34)]. As early as month 3, 55.9% of patients achieved cJADAS10 low disease activity and inactive disease (20.3%, 72/354 and 35.6%, 126/354, respectively), sustained over 5 years. Disease activity measures improvement over 5 years across JIA categories.
CONCLUSION
Abatacept was well tolerated in patients with JIA, with no new safety signals identified and with well-controlled disease activity, including some patients achieving inactive disease or remission.
TRIAL REGISTRATION
Clinicaltrials.gov, NCT01357668.
目的
本研究旨在报告 PRINTO/PRCSG 注册研究中接受阿巴西普治疗的幼年特发性关节炎（JIA）患者的 5 年中期安全性和有效性。
方法
阿巴西普 JIA 注册研究（NCT01357668）是一项正在进行的接受阿巴西普治疗的 JIA 患儿的观察性研究；于 2013 年 1 月开始入组。临床研究中心招募开始接受或正在接受阿巴西普治疗的 JIA 患者。对患者进行 10 年安全性（主要终点）和有效性评估。在 5 年内，通过 JIA 的 10 个关节临床疾病活动评分（cJADAS10）评估有效性。根据《加强观察性研究的报告质量（STROBE）指南》进行观察性分析。
结果
截至 2020 年 3 月 31 日，共入组 587 例患者；569 例患者（包括 134 例新使用者）纳入本次分析，有 1214.6 患者-年的安全性数据可用。在 5 年的随访中，严重不良事件的发生率（IR）为每 100 患者-年 5.52（95%CI：4.27，7.01），特殊关注事件的发生率为 3.62（95%CI：2.63，4.86），有 18 例严重感染[IR 1.48（95%CI：0.88，2.34）]。早在第 3 个月，55.9%的患者达到 cJADAS10 低疾病活动和无疾病活动（分别为 20.3%，72/354 和 35.6%，126/354），且持续 5 年。5 年来，JIA 各分类的疾病活动指标均有改善。
结论
阿巴西普在 JIA 患者中耐受性良好，未发现新的安全性信号，且疾病活动得到良好控制，包括一些患者达到无疾病活动或缓解。
试验注册
Clinicaltrials.gov，NCT01357668。
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