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二甲基富马酸酯和特立氟胺在真实环境中的多发性硬化症:一项法国回顾性队列研究。

Dimethyl fumarate and teriflunomide for multiple sclerosis in a real-life setting: a French retrospective cohort study.

机构信息

Service de Neurologie, CHU Clermont-Ferrand, Université Clermont Auvergne, Clermont-Ferrand, France.

Neuro-Dol, Inserm U1107, Université Clermont Auvergne, Clermont-Ferrand, France.

出版信息

Eur J Neurol. 2019 Mar;26(3):460-467. doi: 10.1111/ene.13839. Epub 2018 Nov 9.

Abstract

BACKGROUND AND PURPOSE

Dimethyl fumarate (DMF) and teriflunomide are approved oral disease-modifying treatments for relapsing-remitting multiple sclerosis (MS). Phase 3 trials established these agents to be effective and generally well tolerated, although comparative efficacy and discontinuation rates are still unknown. The aim of this study was to assess real-world efficacy and discontinuation of DMF and teriflunomide in patients with relapsing-remitting MS.

METHODS

This retrospective observational cohort study was carried out in a French administrative region between March 2014 and July 2017. Patients who were followed by private or hospital neurologists were included. Efficacy and tolerance of the two treatments were assessed and compared by multivariate analysis, considering the duration of MS, annualized relapse rate and Expanded Disability Status Scale score at treatment initiation, treatment duration, type of prescriber and tobacco use.

RESULTS

We identified 189 DMF- and 157 teriflunomide-treated patients who had been treated for 22 ± 10 months. After correction for confounders, DMF more efficiently reduced the annualized relapse rate after 2 years than teriflunomide (0.06 vs. 0.21; P = 0.03). DMF-treated patients had more clinical and biological adverse events, resulting in a higher rate of treatment discontinuation (28% vs. 12%, P = 0.03).

CONCLUSION

In this retrospective cohort study, DMF demonstrated significantly better efficacy over 2 years than teriflunomide, but tolerance to teriflunomide was better.

摘要

背景与目的

富马酸二甲酯(DMF)和特立氟胺是批准用于治疗复发缓解型多发性硬化症(MS)的口服疾病修正治疗药物。3 期临床试验证实了这些药物的有效性和良好的耐受性,尽管其相对疗效和停药率仍不明确。本研究旨在评估 DMF 和特立氟胺在复发缓解型 MS 患者中的真实世界疗效和停药情况。

方法

这是一项在法国某行政区进行的回顾性观察性队列研究,时间范围为 2014 年 3 月至 2017 年 7 月。研究纳入了由私人或医院神经科医生随访的患者。通过多变量分析评估和比较两种治疗方法的疗效和耐受性,考虑了 MS 持续时间、治疗开始时的年复发率和扩展残疾状况量表评分、治疗持续时间、处方类型和吸烟情况。

结果

我们共确定了 189 例 DMF 治疗患者和 157 例特立氟胺治疗患者,他们的治疗持续时间为 22±10 个月。经过混杂因素校正后,DMF 在 2 年内更有效地降低了年复发率(0.06 比 0.21;P=0.03)。DMF 治疗患者出现更多的临床和生物学不良事件,导致更高的停药率(28%比 12%;P=0.03)。

结论

在这项回顾性队列研究中,DMF 在 2 年内的疗效明显优于特立氟胺,但特立氟胺的耐受性更好。

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