• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验

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.

DOI:10.1136/rmdopen-2019-001091
PMID:32396520
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7003379/
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/7003379/6d2e21dc08a9/rmdopen-2019-001091f05.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/fa91171be589/rmdopen-2019-001091f01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/90410f90ef39/rmdopen-2019-001091f02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/50b20395b264/rmdopen-2019-001091f03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/7b53095ecc44/rmdopen-2019-001091f04.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/6d2e21dc08a9/rmdopen-2019-001091f05.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/fa91171be589/rmdopen-2019-001091f01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/90410f90ef39/rmdopen-2019-001091f02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/50b20395b264/rmdopen-2019-001091f03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/7b53095ecc44/rmdopen-2019-001091f04.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b9a/7003379/6d2e21dc08a9/rmdopen-2019-001091f05.jpg

相似文献

1
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.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。
RMD Open. 2020 Jan;6(1). doi: 10.1136/rmdopen-2019-001091.
2
3
Optimizing the Start Time of Biologics in Polyarticular Juvenile Idiopathic Arthritis: A Comparative Effectiveness Study of Childhood Arthritis and Rheumatology Research Alliance Consensus Treatment Plans.优化多关节幼年特发性关节炎生物制剂的起始时间:儿童关节炎和风湿病研究联盟共识治疗计划的比较有效性研究。
Arthritis Rheumatol. 2021 Oct;73(10):1898-1909. doi: 10.1002/art.41888. Epub 2021 Sep 3.
4
Summary of AHRQ's Comparative Effectiveness Review of Disease-Modifying Antirheumatic Drugs for Children with Juvenile Idiopathic Arthritis.美国医疗保健研究与质量局(AHRQ)对用于青少年特发性关节炎患儿的改善病情抗风湿药的比较效果评价总结。
J Manag Care Pharm. 2012 Jan-Feb;18(1 Suppl B):1-16. doi: 10.18553/jmcp.2012.18.S1-B.1.
5
Outcome Monitoring and Clinical Decision Support in Polyarticular Juvenile Idiopathic Arthritis.多关节型幼年特发性关节炎的结局监测和临床决策支持。
J Rheumatol. 2020 Feb;47(2):273-281. doi: 10.3899/jrheum.190268. Epub 2019 Jul 15.
6
Improved Disease Course Associated With Early Initiation of Biologics in Polyarticular Juvenile Idiopathic Arthritis: Trajectory Analysis of a Childhood Arthritis and Rheumatology Research Alliance Consensus Treatment Plans Study.早期使用生物制剂治疗对幼年特发性关节炎多关节型患者病情改善相关:儿童关节炎和风湿病研究联盟共识治疗方案研究的轨迹分析。
Arthritis Rheumatol. 2021 Oct;73(10):1910-1920. doi: 10.1002/art.41892. Epub 2021 Aug 27.
7
Prescribing Patterns and Impact of Factors Associated with Time to Initial Biologic Therapy among Children with Non-systemic Juvenile Idiopathic Arthritis.儿童非系统性幼年特发性关节炎患者初始生物治疗时间相关因素的处方模式及影响。
Paediatr Drugs. 2021 Mar;23(2):171-182. doi: 10.1007/s40272-021-00436-4. Epub 2021 Mar 2.
8
Comparative effectiveness and persistence of TNFi and non-TNFi in juvenile idiopathic arthritis: a large paediatric rheumatology centre in the USA.比较生物制剂和非生物制剂在青少年特发性关节炎中的疗效和持久性:美国一家大型儿科风湿病中心。
Rheumatology (Oxford). 2021 Sep 1;60(9):4063-4073. doi: 10.1093/rheumatology/keaa877.
9
The role of synthetic drugs in the biologic era: therapeutic strategies for treating juvenile idiopathic arthritis.合成药物在生物制剂时代的作用:治疗幼年特发性关节炎的治疗策略
Expert Opin Pharmacother. 2016;17(5):703-14. doi: 10.1517/14656566.2016.1133592. Epub 2016 Jan 13.
10
The clinical effectiveness and cost-effectiveness of abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis: a systematic review and economic evaluation.阿巴西普、阿达木单抗、依那西普和托珠单抗治疗幼年特发性关节炎的临床有效性和成本效益:一项系统评价和经济学评估
Health Technol Assess. 2016 Apr;20(34):1-222. doi: 10.3310/hta20340.

引用本文的文献

1
Literature analysis and implication of biologic therapy for children with non-systemic juvenile idiopathic arthritis in real-world settings.现实环境中生物疗法对非系统性幼年特发性关节炎患儿的文献分析及意义
Intractable Rare Dis Res. 2025 Aug 31;14(3):162-174. doi: 10.5582/irdr.2025.01035.
2
Update on Biologic Therapy in Juvenile Idiopathic Arthritis: A Five-year Narrative Review.青少年特发性关节炎生物治疗的最新进展:一项为期五年的叙述性综述
Biologics. 2025 Jul 23;19:425-441. doi: 10.2147/BTT.S486359. eCollection 2025.
3
A Simple Nonparametric Least-Squares-Based Causal Inference for Heterogeneous Treatment Effects.

本文引用的文献

1
Treating juvenile idiopathic arthritis to target: recommendations of an international task force.靶向治疗幼年特发性关节炎:国际工作组的建议。
Ann Rheum Dis. 2018 Jun;77(6):819-828. doi: 10.1136/annrheumdis-2018-213030. Epub 2018 Apr 11.
2
The Childhood Arthritis and Rheumatology Research Alliance Consensus Treatment Plans: Toward Comparative Effectiveness in the Pediatric Rheumatic Diseases.儿童关节炎和风湿病研究联盟共识治疗计划:走向儿科风湿病的比较疗效。
Arthritis Rheumatol. 2018 May;70(5):669-678. doi: 10.1002/art.40395. Epub 2018 Mar 31.
3
Predictors and long-term outcome in Greek adults with juvenile idiopathic arthritis: a 17-year continuous follow-up study.
一种基于非参数最小二乘法的异质性治疗效果简单因果推断方法。
J Nonparametr Stat. 2025;37(1):169-203. doi: 10.1080/10485252.2024.2367674. Epub 2024 Jul 15.
4
Comparative Effectiveness and Safety of the JAK Inhibitors and Biologic Disease-Modifying Antirheumatic Drugs in Treating Children With Nonsystemic Juvenile Idiopathic Arthritis: A Bayesian Meta-Analysis of Randomized Controlled Trials.JAK抑制剂与生物性改善病情抗风湿药治疗非系统性幼年特发性关节炎患儿的疗效和安全性比较:一项随机对照试验的贝叶斯荟萃分析
ACR Open Rheumatol. 2025 Feb;7(2):e11788. doi: 10.1002/acr2.11788.
5
Bayesian causal inference for observational studies with missingness in covariates and outcomes.贝叶斯因果推断在协变量和结局缺失的观察性研究中的应用。
Biometrics. 2023 Dec;79(4):3624-3636. doi: 10.1111/biom.13918. Epub 2023 Aug 8.
6
Pharmacological treatment patterns in patients with juvenile idiopathic arthritis in the Netherlands: a real-world data analysis.荷兰青少年特发性关节炎患者的药物治疗模式:真实世界数据分析。
Rheumatology (Oxford). 2023 Feb 23;62(SI2):SI170-SI180. doi: 10.1093/rheumatology/keac299.
7
Real-world data reveals the complexity of disease modifying anti-rheumatic drug treatment patterns in juvenile idiopathic arthritis: an observational study.真实世界数据揭示了幼年特发性关节炎疾病修饰抗风湿药物治疗模式的复杂性:一项观察性研究。
Pediatr Rheumatol Online J. 2022 Apr 11;20(1):25. doi: 10.1186/s12969-022-00682-x.
8
Disparities in Pediatric Rheumatic Diseases.儿科风湿病的差异。
Rheum Dis Clin North Am. 2022 Feb;48(1):183-198. doi: 10.1016/j.rdc.2021.09.014.
9
A scoping review of studies using observational data to optimise dynamic treatment regimens.使用观察性数据优化动态治疗方案的研究的范围综述。
BMC Med Res Methodol. 2021 Feb 22;21(1):39. doi: 10.1186/s12874-021-01211-2.
希腊青少年特发性关节炎成年患者的预测因素及长期预后:一项为期17年的连续随访研究。
Rheumatology (Oxford). 2017 Nov 1;56(11):1928-1938. doi: 10.1093/rheumatology/kex265.
4
The new Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry: design, rationale, and characteristics of patients enrolled in the first 12 months.新的儿童关节炎与风湿病研究联盟(CARRA)登记处:前12个月登记患者的设计、原理及特征
Pediatr Rheumatol Online J. 2017 Apr 17;15(1):30. doi: 10.1186/s12969-017-0160-6.
5
EULAR recommendations for the management of rheumatoid arthritis with synthetic and biological disease-modifying antirheumatic drugs: 2016 update.EULAR 推荐的类风湿关节炎治疗策略:2016 年更新版
Ann Rheum Dis. 2017 Jun;76(6):960-977. doi: 10.1136/annrheumdis-2016-210715. Epub 2017 Mar 6.
6
Bayesian Nonparametric Estimation for Dynamic Treatment Regimes with Sequential Transition Times.具有顺序转移时间的动态治疗方案的贝叶斯非参数估计
J Am Stat Assoc. 2016;111(515):921-935. doi: 10.1080/01621459.2015.1086353. Epub 2016 Oct 18.
7
A Bayesian nonparametric approach to marginal structural models for point treatments and a continuous or survival outcome.一种用于点治疗以及连续或生存结局的边际结构模型的贝叶斯非参数方法。
Biostatistics. 2017 Jan;18(1):32-47. doi: 10.1093/biostatistics/kxw029. Epub 2016 Jun 26.
8
Clinical outcome measures in juvenile idiopathic arthritis.青少年特发性关节炎的临床结局指标
Pediatr Rheumatol Online J. 2016 Apr 18;14(1):23. doi: 10.1186/s12969-016-0085-5.
9
2015 American College of Rheumatology Guideline for the Treatment of Rheumatoid Arthritis.2015 年美国风湿病学会类风湿关节炎治疗指南。
Arthritis Rheumatol. 2016 Jan;68(1):1-26. doi: 10.1002/art.39480. Epub 2015 Nov 6.
10
Treating rheumatoid arthritis to target: 2014 update of the recommendations of an international task force.类风湿关节炎达标治疗:国际特别工作组2014年推荐意见更新
Ann Rheum Dis. 2016 Jan;75(1):3-15. doi: 10.1136/annrheumdis-2015-207524. Epub 2015 May 12.