Longbrake Erin E, Cross Anne H, Salter Amber
Department of Neurology, Yale University, USA; Department of Neurology, Washington University in St. Louis, USA.
Department of Neurology, Washington University in St. Louis, USA.
Mult Scler J Exp Transl Clin. 2016 Jan-Dec;2. doi: 10.1177/2055217316677868. Epub 2016 Nov 1.
The advent of oral disease-modifying therapies fundamentally changed the treatment of multiple sclerosis. Nevertheless, impressions of their relative efficacy and tolerability are primarily founded on expert opinion.
The purpose of this study was to determine whether oral disease-modifying therapies were better tolerated and/or more effective for controlling multiple sclerosis compared to injectable therapies in clinical practice.
Single-center, retrospective cohort study. 480 patients initiated oral (fingolimod, teriflunomide, or dimethyl fumarate) or injectable therapy between March 2013-March 2015 and follow-up data was collected for 5-31 months. Outcomes included on-drug multiple sclerosis activity and drug discontinuation. Cox proportional hazards models were used to control for baseline differences and sensitivity analyses using propensity-weighted matching were performed.
A higher proportion of teriflunomide-treated patients experienced multiple sclerosis activity compared to those treated with injectable therapies ( = 0.0053) in the adjusted model. Breakthrough multiple sclerosis was equally prevalent among fingolimod and dimethyl fumarate-treated compared to injectable therapy-treated patients. Of patients initiating a disease-modifying therapy, 32-46% discontinued or switched treatments during the study. After controlling for baseline differences, discontinuation rates were comparable across treatment groups.
In this cohort, oral and injectable disease-modifying therapies were equally well tolerated, but teriflunomide appeared less effective for controlling multiple sclerosis activity than injectable therapies. Further study is needed.
口服疾病修正疗法的出现从根本上改变了多发性硬化症的治疗方式。然而，对其相对疗效和耐受性的认识主要基于专家意见。
本研究的目的是确定在临床实践中，与注射疗法相比，口服疾病修正疗法对控制多发性硬化症是否耐受性更好和/或更有效。
单中心回顾性队列研究。2013年3月至2015年3月期间，480例患者开始接受口服（芬戈莫德、特立氟胺或富马酸二甲酯）或注射治疗，并收集了5至31个月的随访数据。结局包括治疗期间的多发性硬化症活动情况和药物停用情况。采用Cox比例风险模型控制基线差异，并进行倾向加权匹配的敏感性分析。
在调整模型中，与接受注射疗法治疗的患者相比，接受特立氟胺治疗的患者出现多发性硬化症活动的比例更高（P = 0.0053）。与接受注射疗法治疗的患者相比，接受芬戈莫德和富马酸二甲酯治疗的患者中突破性多发性硬化症的发生率相当。在开始疾病修正疗法的患者中，32%至46%在研究期间停药或换药。在控制基线差异后，各治疗组的停药率相当。
在该队列中，口服和注射疾病修正疗法的耐受性相当，但特立氟胺在控制多发性硬化症活动方面似乎不如注射疗法有效。需要进一步研究。
