Simoneau Gabrielle, Jiang Xiaotong, Rollot Fabien, Tian Lu, Copetti Massimiliano, Guéry Matthieu, Ruiz Marta, Vukusic Sandra, de Moor Carl, Pellegrini Fabio
Biogen Canada, Mississauga, ON, Canada.
Biogen Inc., Cambridge, MA, USA.
Mult Scler J Exp Transl Clin. 2022 Aug 4;8(3):20552173221116591. doi: 10.1177/20552173221116591. eCollection 2022 Jul-Sep.
Comparing real-world effectiveness and tolerability of therapies for relapsing-remitting multiple sclerosis is increasingly important, though average treatment effects fail to capture possible treatment effect heterogeneity. With the clinical course of the disease being highly heterogeneous across patients, precision medicine methods enable treatment response heterogeneity investigations.
To compare real-world effectiveness and discontinuation profiles between dimethyl fumarate and fingolimod while investigating treatment effect heterogeneity with precision medicine methods.
Adults initiating dimethyl fumarate or fingolimod as a second-line therapy were selected from a French registry. The primary outcome was annualized relapse rate at 12 months. Seven secondary outcomes relative to discontinuation and disease progression were considered. A precision medicine framework was used to characterize treatment effect heterogeneity.
Annualized relapse rates at 12 months were similar for dimethyl fumarate and fingolimod. The odd of treatment persistence was 47% lower for patients treated with dimethyl fumarate relative to those treated with fingolimod (odds ratio: 0.53, 95% confidence interval: 0.39, 0.70). None of the five precision medicine scoring approaches identified treatment heterogeneity.
These findings substantiated the similar effectiveness and different discontinuation profiles for dimethyl fumarate and fingolimod as a second-line therapy for relapsing-remitting multiple sclerosis, with no significant effect heterogeneity observed.
比较复发缓解型多发性硬化症治疗方法的真实世界有效性和耐受性变得越来越重要，尽管平均治疗效果无法捕捉到可能存在的治疗效果异质性。由于该疾病的临床病程在患者中高度异质，精准医学方法有助于对治疗反应异质性进行研究。
比较富马酸二甲酯和芬戈莫德的真实世界有效性和停药情况，同时用精准医学方法研究治疗效果异质性。
从法国一个登记处选取开始使用富马酸二甲酯或芬戈莫德作为二线治疗的成年人。主要结局是12个月时的年化复发率。还考虑了7个与停药和疾病进展相关的次要结局。使用一个精准医学框架来描述治疗效果异质性。
富马酸二甲酯和芬戈莫德在12个月时的年化复发率相似。接受富马酸二甲酯治疗的患者持续治疗的几率比接受芬戈莫德治疗的患者低47%（优势比：0.53，95%置信区间：0.39，0.70）。五种精准医学评分方法均未发现治疗异质性。
这些发现证实了富马酸二甲酯和芬戈莫德作为复发缓解型多发性硬化症二线治疗方法具有相似的有效性和不同的停药情况，未观察到显著的效果异质性。
