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类风湿关节炎患者使用疾病修饰药物治疗的缓解、反应、保留和持续:一项瑞典、丹麦和挪威队列研究的综合分析。

Remission, response, retention and persistence to treatment with disease-modifying agents in patients with rheumatoid arthritis: a study of harmonised Swedish, Danish and Norwegian cohorts.

机构信息

Clinical Epidemiology Division, Department of Medicine, Solna, Karolinska Institutet, Stockholm, Sweden

Department of Clinical Medicine, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.

出版信息

RMD Open. 2023 Sep;9(3). doi: 10.1136/rmdopen-2023-003027.

Abstract

OBJECTIVE

Precision medicine in rheumatoid arthritis (RA) requires a good understanding of treatment outcomes and often collaborative efforts that call for data harmonisation. We aimed to describe how harmonisation across study cohorts can be achieved and investigate how the observed proportions reaching remission vary across remission criteria, study types, disease-modifying antirheumatic drugs (DMARDs) and countries, and how they relate to other treatment outcomes.

METHODS

We used data from eight existing large-scale, clinical RA registers and a pragmatic trial from Sweden, Denmark and Norway. In these, we defined three types of treatment cohorts; methotrexate monotherapy (as first DMARD), tumour necrosis factor inhibitors (TNFi) (as first biological DMARD) and rituximab. We developed a harmonised study protocol defining time points during 36 months of follow-up, collected clinical visit data on treatment response, retention, persistence and six alternative definitions of remission, and investigated how these outcomes differed within and between cohorts, by treatment.

RESULTS

Cohort sizes ranged from ~50 to 22 000 patients with RA. The proportions reaching each outcome varied across outcome metric, but with small to modest variations within and between cohorts, countries and treatment. Retention and persistence rates were high (>50% at 1 year), yet <33% of patients starting methotrexate or TNFi, and only 10% starting rituximab, remained on drug without other DMARDs added and achieved American Congress of Rheumatology/European Alliance of Associations for Rheumatology or Simplified Disease Activity Index remission at 1 year.

CONCLUSION

Harmonisation of data from different RA data sources can be achieved without compromising internal validity or generalisability. The low proportions reaching remission, point to an unmet need for treatment optimisation in RA.

摘要

目的

类风湿关节炎(RA)的精准医学需要很好地了解治疗结果,并且通常需要协作努力,这需要数据协调。我们旨在描述如何实现研究队列之间的协调,并研究在不同缓解标准、研究类型、疾病修饰抗风湿药物(DMARDs)和国家中,观察到的达到缓解的比例如何变化,以及它们与其他治疗结果的关系。

方法

我们使用了来自瑞典、丹麦和挪威的 8 个现有大型临床 RA 登记处和一个实用临床试验的数据。在这些研究中,我们定义了三种治疗队列;甲氨蝶呤单药治疗(作为一线 DMARD)、肿瘤坏死因子抑制剂(TNFi)(作为一线生物 DMARD)和利妥昔单抗。我们制定了一个协调的研究方案,定义了 36 个月随访期间的时间点,收集了关于治疗反应、保留、持续和六种替代缓解定义的临床就诊数据,并研究了这些结果在队列内和队列之间、治疗内和治疗之间的差异。

结果

队列规模从大约 50 名到 22000 名 RA 患者不等。达到每种结果的比例因结果衡量标准而异,但在队列内和队列之间、国家和治疗之间差异较小。保留率和持续率较高(1 年时>50%),但开始甲氨蝶呤或 TNFi 的患者中<33%,开始利妥昔单抗的患者中只有 10%,在没有添加其他 DMARD 的情况下继续使用药物并达到美国风湿病学会/欧洲抗风湿病联盟或简化疾病活动指数缓解在 1 年内。

结论

来自不同 RA 数据源的数据协调可以在不影响内部有效性或普遍性的情况下实现。达到缓解的比例较低,表明 RA 的治疗优化存在未满足的需求。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/76f9/10496677/693a03cf2f10/rmdopen-2023-003027f01.jpg

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