Effectiveness of tumor necrosis factor inhibitors in rheumatoid arthritis in an observational cohort study - Comparison of patients according to their eligibility for major randomized clinical trials
Effectiveness of tumor necrosis factor inhibitors in rheumatoid arthritis in an observational cohort study - Comparison of patients according to their eligibility for major randomized clinical trials
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DOI：
10.1002/art.22193
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发表时间：
2006-11-01
影响因子：
--
通讯作者：
Listing, Joachim
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文献类型：
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作者：
Zink, Angela;Strangfeld, Anja;Listing, Joachim
客观的。随机临床试验 (RCT) 评估按严格纳入标准定义的选定患者组的治疗效果。这些试验在预测“现实世界”患者的治疗效果方面的价值是有限的。这项观察性队列研究旨在根据主要试验的资格评估肿瘤坏死因子 (TNF) 抑制剂对类风湿性关节炎 (RA) 患者的有效性，以补充随机对照试验中获得的知识。方法。利用德国生物制剂注册类风湿性关节炎生物治疗观察（RABBIT [德语]）的数据，我们调查了有多少接受 TNF 抑制剂（英夫利昔单抗、依那西普或阿达木单抗）治疗的 RA 患者有资格参加导致药物批准的主要临床试验。此外，使用美国风湿病学会 20% (ACR20) 和 50% (ACR50) 改善反应标准比较了合格和不合格患者的治疗效果。 结果。 RABBIT 登记中只有 21-33% 的患者有资格参加主要试验。在这些患者中，ACR20 和 ACR50 改善反应（表明治疗效果）与已发表试验中的反应率相当。那些被认为不符合试验资格的患者的 ACR 反应率较低；然而，绝对改善与符合条件的患者相似。不符合条件的患者基线疾病活动度较低、合并症较多、功能状态较低。结论。随机对照试验队列仅反映了在常规护理中接受生物制剂治疗的一小部分患者。在临床环境中，生物制剂治疗的适应症与试验的纳入标准并不相同。尽管这些患有长期严重类风湿性关节炎且不符合主要试验纳入标准的患者取得的相对改善较小，但大多数此类患者仍将从生物治疗中受益。
Objective. Randomized clinical trials (RCTs) evaluate the efficacy of treatments in selected groups of patients defined by strict inclusion criteria. The value of these trials in predicting therapeutic effectiveness in "real world" patients is limited. This observational cohort study was designed to complement the knowledge obtained in RCTs by evaluating the effectiveness of tumor necrosis factor (TNF) inhibitors in patients with rheumatoid arthritis (RA) according to their eligibility for the major trials.Methods. Using the data from the German biologics register Rheumatoid Arthritis Observation of Biologic Therapy (RABBIT [in German]), we investigated how many of the RA patients who were treated with a TNF inhibitor (infliximab, etanercept, or adalimumab) would have been eligible for the major clinical trials that led to approval of the drugs. In addition, therapeutic effectiveness was compared in the eligible and ineligible patients using the American College of Rheumatology 20% (ACR20) and 50% (ACR50) improvement response criteria.Results. Only 21-33% of the patients in the RABBIT register would have been eligible for the major trials. In these patients, the ACR20 and ACR50 improvement responses, indicating therapeutic effectiveness, were comparable with the response rates in the published trials. ACR response rates were lower in those patients considered ineligible for the trials; however, absolute improvement was similar to that in eligible patients. Ineligible patients had lower baseline disease activity, more comorbidity, and lower functional status.Conclusion. RCT cohorts reflect only a minor proportion of the patients treated with biologic agents in routine care. In the clinic setting, the indications for treatment with biologic agents are not identical to the inclusion criteria for trials. Despite the smaller relative improvement achieved in these patients with longstanding, severe RA who would not fulfill the inclusion criteria of a major trial, the majority of such patients would nevertheless benefit from biologic therapy.