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时机很重要:生物制剂和传统合成疾病修饰抗风湿药物早期联合治疗新诊断多关节病程幼年特发性关节炎的真实世界疗效。

Timing matters: real-world effectiveness of early combination of biologic and conventional synthetic disease-modifying antirheumatic drugs for treating newly diagnosed polyarticular course juvenile idiopathic arthritis.

机构信息

Division of Biostatistics and Epidemiology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA

Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.

出版信息

RMD Open. 2020 Jan;6(1). doi: 10.1136/rmdopen-2019-001091.

Abstract

OBJECTIVES

To compare real-world effectiveness of two adaptive treatment strategies of disease-modifying antirheumatic drugs (DMARDs) in treating children with newly diagnosed polyarticular course juvenile idiopathic arthritis (pcJIA): early aggressive use of biologic DMARDs (bDMARDs) in combination with conventional synthetic DMARDs (csDMARDs) versus conservative delayed use of bDMARDs following the initial csDMARD prescription.

METHODS

A single-centre newly diagnosed DMARD-naive pcJIA patient database (n=465) was derived from the electronic medical records between 1 January 2009 and 31 December 2018. The primary study endpoints were clinical Juvenile Arthritis Disease Activity Score (cJADAS) at 6 and 12 months following the first DMARD prescription. The secondary study endpoint was Pediatric Quality of Life Inventory (PedsQL) generic total score at 12 months. Averaged causal treatment effects were assessed using a Bayesian non-parametric casual inference method.

RESULTS

Both cJADAS and PedsQL improve over time, regardless of the treatment strategies. Compared with the conservative approach, early aggressive approach is more effective in reducing cJADAS (mean -2.17, 95% CI -3.77 to -0.56) by 6 months. Adding bDMARD after 6 months to the initial treatment provides very little added benefit. The averaged treatment effect was 6.35 (95% CI -5.89 to 18.58) improvement in PedsQL at 12 months.

CONCLUSIONS

Timing matters-early aggressive use with bDMARDs is more effective than conservative delayed treatment in lowering disease activity after 6 and 12 months of treatment.

摘要

目的

比较两种疾病修正抗风湿药物(DMARDs)的治疗方案在治疗新诊断多关节起病型幼年特发性关节炎(pcJIA)患儿中的真实世界疗效:早期联合使用生物性 DMARDs(bDMARDs)和传统合成 DMARDs(csDMARDs)的积极治疗方案与初始 csDMARD 处方后延迟使用 bDMARDs 的保守治疗方案。

方法

从 2009 年 1 月 1 日至 2018 年 12 月 31 日的电子病历中提取了一个单中心新诊断 DMARD 初治 pcJIA 患者数据库(n=465)。主要研究终点为首次 DMARD 处方后 6 个月和 12 个月的临床幼年特发性关节炎疾病活动评分(cJADAS)。次要研究终点为 12 个月时儿童生活质量量表(PedsQL)通用总分。使用贝叶斯非参数因果推理方法评估平均因果治疗效果。

结果

无论治疗策略如何,cJADAS 和 PedsQL 均随时间推移而改善。与保守方法相比,早期积极方法在 6 个月时更有效地降低 cJADAS(平均差值-2.17,95%CI-3.77 至-0.56)。在初始治疗后 6 个月添加 bDMARD 并不能带来更多益处。12 个月时 PedsQL 的平均治疗效果为 6.35(95%CI-5.89 至 18.58)。

结论

时机很重要-在治疗 6 和 12 个月后,早期积极使用 bDMARDs 比保守延迟治疗更能降低疾病活动度。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/fa91171be589/rmdopen-2019-001091f01.jpg

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