Erwan Muros-Le Rouzic, Yanic Heer, Sean Yiu, Viola Tozzi, Stefan Braune, Philip van Hövell, Arnfin Bergmann, Corrado Bernasconi, Fabian Model, Licinio Craveiro
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Efficacy outcomes were time to first relapse and time to 24-week confirmed disability progression over 5.5 years of follow-up. Intention-to-treat analysis using all outcome data irrespective of treatment switch was applied.</p><p><strong>Results: </strong>The analyses included 611 OPERA patients and 7141 NTD patients. We built 12 paired-matched cohorts (six for each outcome, two for each DMT) to compare efficacy of OCR in OPERA with each DMT treatment pathway in NTD. Post-matching, baseline covariates and PS were well balanced (standardized mean difference <.2 for all cohorts). Over 5.5 years, patients treated with OCR showed a statistically significant reduction in the risk of relapse (hazard ratios [HRs] .30 to .54) and disability progression (HRs .51 to .67) compared with all index therapies and their treatment switching pathways in NTD. Treatment switch and/or discontinuation occurred frequently in NTD cohorts.</p><p><strong>Conclusion: </strong>OCR demonstrates superiority in controlling relapses and disability progression in RMS compared with real-world treatment pathways over a 5.5-year period. These analyses suggest that high-efficacy DMTs and high treatment persistence are critical to achieve greatest clinical benefit in RMS.</p><p><strong>Registration: </strong>OPERA I (NCT01247324), OPERA II (NCT01412333).</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":"16 ","pages":"11795735241260563"},"PeriodicalIF":2.6000,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11406495/pdf/","citationCount":"0","resultStr":"{\"title\":\"Five-year efficacy outcomes of ocrelizumab in relapsing multiple sclerosis: A propensity-matched comparison of the OPERA studies with other disease-modifying therapies in real-world lines of treatments.\",\"authors\":\"Erwan Muros-Le Rouzic, Yanic Heer, Sean Yiu, Viola Tozzi, Stefan Braune, Philip van Hövell, Arnfin Bergmann, Corrado Bernasconi, Fabian Model, Licinio Craveiro\",\"doi\":\"10.1177/11795735241260563\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Clinical trials comparing the efficacy of ocrelizumab (OCR) with other disease-modifying therapies (DMTs) other than interferon (IFN) β-1a in relapsing multiple sclerosis (RMS) are lacking.</p><p><strong>Objectives: </strong>To compare the treatment effect of OCR vs six DMTs' (IFN β-1a, glatiramer acetate, fingolimod, dimethyl fumarate, teriflunomide, natalizumab) treatment pathways used in clinical practice by combining clinical trial and real-world data.</p><p><strong>Methods: </strong>Patient-level data from OPERA trials and open-label extension phase, and from the German NeuroTransData (NTD) MS registry, were used to build 1:1 propensity score-matched (PSM) cohorts controlling for seven baseline covariates, including brain imaging activity. 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引用次数: 0
摘要
背景：在复发性多发性硬化症（RMS）中，目前尚缺乏比较奥克立珠单抗（OCR）与干扰素（IFN）β-1a以外的其他疾病修饰疗法（DMTs）疗效的临床试验：结合临床试验和实际数据，比较OCR与六种DMTs（IFN β-1a、醋酸格拉替雷、芬戈莫德、富马酸二甲酯、特利氟胺、纳他珠单抗）在临床实践中的治疗效果：方法：利用OPERA试验和开放标签扩展阶段的患者水平数据，以及德国NeuroTransData（NTD）多发性硬化症登记处的数据，建立1:1倾向得分匹配（PSM）队列，控制包括脑成像活动在内的7个基线协变量。疗效结果为首次复发时间和随访5.5年的24周确诊残疾进展时间。无论治疗转换与否，均使用所有结果数据进行意向治疗分析：分析对象包括 611 名 OPERA 患者和 7141 名 NTD 患者。我们建立了 12 个配对队列（每种结果 6 个队列，每种 DMT 2 个队列），以比较 OPERA 中 OCR 与 NTD 中每种 DMT 治疗途径的疗效。配对后，基线协变量和PS达到了很好的平衡（标准化平均差结论）：在 5.5 年的时间里，与现实世界的治疗路径相比，OCR 在控制 RMS 复发和残疾进展方面更具优势。这些分析表明，高疗效 DMTs 和高治疗持续性是 RMS 获得最大临床获益的关键：Opera I (NCT01247324)、Opera II (NCT01412333)。
Five-year efficacy outcomes of ocrelizumab in relapsing multiple sclerosis: A propensity-matched comparison of the OPERA studies with other disease-modifying therapies in real-world lines of treatments.
Background: Clinical trials comparing the efficacy of ocrelizumab (OCR) with other disease-modifying therapies (DMTs) other than interferon (IFN) β-1a in relapsing multiple sclerosis (RMS) are lacking.
Objectives: To compare the treatment effect of OCR vs six DMTs' (IFN β-1a, glatiramer acetate, fingolimod, dimethyl fumarate, teriflunomide, natalizumab) treatment pathways used in clinical practice by combining clinical trial and real-world data.
Methods: Patient-level data from OPERA trials and open-label extension phase, and from the German NeuroTransData (NTD) MS registry, were used to build 1:1 propensity score-matched (PSM) cohorts controlling for seven baseline covariates, including brain imaging activity. Efficacy outcomes were time to first relapse and time to 24-week confirmed disability progression over 5.5 years of follow-up. Intention-to-treat analysis using all outcome data irrespective of treatment switch was applied.
Results: The analyses included 611 OPERA patients and 7141 NTD patients. We built 12 paired-matched cohorts (six for each outcome, two for each DMT) to compare efficacy of OCR in OPERA with each DMT treatment pathway in NTD. Post-matching, baseline covariates and PS were well balanced (standardized mean difference <.2 for all cohorts). Over 5.5 years, patients treated with OCR showed a statistically significant reduction in the risk of relapse (hazard ratios [HRs] .30 to .54) and disability progression (HRs .51 to .67) compared with all index therapies and their treatment switching pathways in NTD. Treatment switch and/or discontinuation occurred frequently in NTD cohorts.
Conclusion: OCR demonstrates superiority in controlling relapses and disability progression in RMS compared with real-world treatment pathways over a 5.5-year period. These analyses suggest that high-efficacy DMTs and high treatment persistence are critical to achieve greatest clinical benefit in RMS.
Registration: OPERA I (NCT01247324), OPERA II (NCT01412333).
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