Cree Bruce A C, Arnold Douglas L, Cascione Mark, Fox Edward J, Williams Ian M, Meng Xiangyi, Schofield Lesley, Tenenbaum Nadia
UCSF Weill Institute for Neurosciences, Department of Neurology, 675 Nelson Rising Lane, San Francisco, CA 94158, USA.
NeuroRx Research and Montreal Neurological Institute, Montreal, Quebec, Canada; Department of Neurology and Neurosurgery, McGill University, Montreal, Quebec, Canada.
Ther Adv Neurol Disord. 2018 May 20;11:1756286418774338. doi: 10.1177/1756286418774338. eCollection 2018.
In relapsing-remitting multiple sclerosis (RRMS), suboptimal adherence to injectable disease-modifying therapies (iDMTs; interferon β-1a/b, glatiramer acetate) is common, reducing their effectiveness. Patient retention on oral fingolimod and iDMTs was evaluated in PREFER, a randomized, parallel-group, active-controlled, open-label, 48-week study.
Patients were included if they had RRMS, were aged 18-65 years and had Expanded Disability Status Scale score up to 6, enrolled at 117 US study sites, were treatment naïve or had received only one iDMT class. Patients were randomized 1:1 (fingolimod 0.5 mg/day; preselected iDMT) by interactive voice-and-web-response system without blinding, followed up quarterly, and allowed one study-approved treatment switch after 12 weeks, or earlier for efficacy or safety reasons. The primary outcome was patient retention on randomized treatment over 48 weeks. Secondary endpoints included patient-reported outcomes, brain volume loss (BVL), and cognitive function.
Analysis of 433/436 patients receiving fingolimod and 428/439 receiving iDMTs showed that patient retention rate was significantly higher with fingolimod than with iDMTs [352 (81.3%) 125 (29.2%); 95% confidence interval 46.4-57.8%; < 0.0001]. The most common treatment switch was from iDMT to fingolimod for injection-related reasons. Patient satisfaction was greater and BVL less with fingolimod than with iDMTs, with no difference in cognitive function. Adverse events were consistent with established tolerability profiles for each treatment.
In RRMS, fingolimod was associated with better treatment retention, patient satisfaction and BVL outcomes than iDMTs. Patients may persist with iDMTs, but many may switch treatment if permitted. Treatment satisfaction fosters adherence, a prerequisite for optimal outcomes.
在复发缓解型多发性硬化症（RRMS）中，对注射用疾病修正治疗药物（iDMTs；干扰素β-1a/b、醋酸格拉替雷）依从性欠佳的情况很常见，这会降低其疗效。在一项为期48周的随机、平行组、活性药物对照、开放标签的PREFER研究中，对口服芬戈莫德和iDMTs治疗的患者留存率进行了评估。
纳入标准为患有RRMS、年龄在18至65岁之间、扩展残疾状态量表评分最高为6分、在美国117个研究地点入组、既往未接受过治疗或仅接受过一类iDMTs治疗的患者。患者通过交互式语音和网络应答系统按1:1随机分组（芬戈莫德0.5毫克/天；预先选定的iDMT），不设盲法，每季度随访一次，在12周后允许进行一次经研究批准的治疗转换，或因疗效或安全原因提前转换。主要结局是48周内随机分组治疗的患者留存率。次要终点包括患者报告的结局、脑容量损失（BVL）和认知功能。
对433/436例接受芬戈莫德治疗的患者和428/439例接受iDMTs治疗的患者进行分析，结果显示芬戈莫德治疗的患者留存率显著高于iDMTs治疗的患者[352例（81.3%）对125例（29.2%）；95%置信区间46.4-57.8%；P<0.0001]。最常见的治疗转换是因注射相关原因从iDMTs转换为芬戈莫德。与iDMTs相比，芬戈莫德治疗的患者满意度更高，BVL更低，认知功能无差异。不良事件与每种治疗已确定的耐受性特征一致。
在RRMS中，与iDMTs相比，芬戈莫德与更好的治疗留存率、患者满意度和BVL结局相关。患者可能会坚持使用iDMTs，但如果允许，许多患者可能会转换治疗。治疗满意度有助于提高依从性，而依从性是实现最佳结局的前提条件。
