Department of Neurology, University of Pennsylvania Perelman School of Medicine; Philadelphia, PA, USA; Department of Neurology Translational Center for Excellence for Neuroepidemiology and Neurological Outcomes Research, University of Pennsylvania Perelman School of Medicine; Philadelphia, PA, USA; Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania Perelman School of Medicine; Philadelphia, PA, USA; Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania Perelman School of Medicine; Philadelphia, PA, USA.
Department of Neurology, University of Pennsylvania Perelman School of Medicine; Philadelphia, PA, USA; Department of Neurology Translational Center for Excellence for Neuroepidemiology and Neurological Outcomes Research, University of Pennsylvania Perelman School of Medicine; Philadelphia, PA, USA.
Mult Scler Relat Disord. 2021 Jul;52:102944. doi: 10.1016/j.msard.2021.102944. Epub 2021 Apr 20.
Oral disease-modifying therapies, namely dimethyl fumarate, fingolimod and teriflunomide, have become standard treatments for multiple sclerosis. Clinical trials demonstrated a reduction in annual relapse rate, but real-world data is lacking, particularly in older adults. The objective of our study is to evaluate the real-world effectiveness of oral disease-modifying therapies among individuals with multiple sclerosis.
We used Optum Clinformatics Data Mart, a large dataset representative of commercially insured individuals in the United States, to conduct a retrospective cohort study of adult users of three oral disease-modifying therapies from September 2010 through September 2015. The therapies of interest included dimethyl fumarate, teriflunomide, and fingolimod. Hospitalization for multiple sclerosis, an approximation of the clinical trial endpoint for relapse, was the study outcome. Cox proportional hazards models were built to evaluate the association of demographic and clinical factors with multiple sclerosis hospitalization. A subgroup analysis was performed on individuals ages 55 years or older.
We identified 1,823, 318, and 1,156 users of dimethyl fumarate, teriflunomide, and fingolimod that met our inclusion criteria, respectively. Rates of hospitalizations for multiple sclerosis were low among these 3,297 persons (1,041 ages 55+): 36/1,000 patient-years for dimethyl fumarate, 43/1,000 for teriflunomide, and 45/1,000 for fingolimod. Multiple sclerosis hospitalization was associated with therapy switching (adjusted hazard ratio 2.21, 95% confidence interval 1.57-2.84), minority (1.44, 1.10-1.89), and history of relapse in the year preceding oral therapy initiation (5.25, 3.89-7.09).
Oral disease-modifying therapies are comparably effective for the outcome of multiple sclerosis hospitalization, even in older adults.
口服疾病修饰疗法，即二甲基富马酸、芬戈莫德和特立氟胺，已成为多发性硬化症的标准治疗方法。临床试验表明，这些疗法可降低年复发率，但缺乏真实世界的数据，特别是在老年人中。我们的研究目的是评估口服疾病修饰疗法在多发性硬化症患者中的真实世界疗效。
我们使用 Optum Clinformatics Data Mart，这是一个代表美国商业保险人群的大型数据集，对 2010 年 9 月至 2015 年 9 月期间使用三种口服疾病修饰疗法的成年患者进行回顾性队列研究。研究中感兴趣的疗法包括二甲基富马酸、特立氟胺和芬戈莫德。多发性硬化症住院治疗是复发的临床试验终点的近似值，作为研究结果。使用 Cox 比例风险模型评估人口统计学和临床因素与多发性硬化症住院之间的关联。对 55 岁及以上的个体进行了亚组分析。
我们分别确定了 1823 名、318 名和 1156 名使用二甲基富马酸、特立氟胺和芬戈莫德的患者符合纳入标准。在这 3297 名患者中，多发性硬化症住院率较低（1041 名为 55 岁及以上）：二甲基富马酸组为 36/1000 患者年，特立氟胺组为 43/1000 患者年，芬戈莫德组为 45/1000 患者年。多发性硬化症住院与治疗转换（调整后的危险比 2.21，95%置信区间 1.57-2.84）、少数民族（1.44，1.10-1.89）和口服治疗开始前一年的复发史（5.25，3.89-7.09）相关。
即使在老年人中，口服疾病修饰疗法对多发性硬化症住院的疗效也相当。
