Partners MS Center, Brigham and Women's Hospital, Boston, MA, United States; Department of Neurology, Harvard Medical School, Boston, MA, United States.
Partners MS Center, Brigham and Women's Hospital, Boston, MA, United States; Department of Neurology, Harvard Medical School, Boston, MA, United States.
Mult Scler Relat Disord. 2020 May;40:101944. doi: 10.1016/j.msard.2020.101944. Epub 2020 Jan 11.
Outcome measures typically used to evaluate disease modifying therapies (DMTs) provide important information regarding their effects on disease activity, but they do not capture the full impact of living with multiple sclerosis (MS). Patient reported outcome measures (PROs) are increasingly being used to capture an individual's subjective experience of disease. We compared DMTs across a wide range of PRO outcomes in individuals with MS.
Subjects enrolled in SysteMS completed the computer adaptive testing version of the Neuro-QoL within four weeks of a clinical neurological exam. Neuro-QoL measures included the following 11 health-related quality of life (HRQOL) domains: Ability to participate in Social Roles and Activities, Anxiety, Cognitive Function, Depression, Emotional and Behavioral Dyscontrol, Fatigue, Lower Extremity Function (mobility), Positive Affect and Wellbeing, Satisfaction with Social Roles and Activities, Stigma, and Upper Extremity Function (fine motor). Treatments were grouped based on the three main modes of delivery: injectable, oral and infusion. The three treatment groups were compared using linear regression adjusting for two sets of covariates (set 1: age, sex, disease duration and EDSS; set 2: age, sex, disease duration, EDSS and treatment duration). We also compared the individual treatments using linear regression.
After adjusting for the first set of clinical and demographic features of MS, there was a difference between treatment groups for Upper Extremity Function and Stigma. Subjects using injectable treatments reported better functioning in terms of Upper Extremity Function and Stigma than subjects using infusion treatments. In addition, subjects using injectable treatments reported better Upper Extremity Function than subjects treated with oral DMTs. When all individual treatments were compared, interferon-treated subjects reported significantly better functioning in terms of Stigma than natalizumab treated subjects. When further adjusting for time on treatment, the group differences were attenuated and no longer statistically significant.
We examined differences between MS treatment groups across a wide range of HRQOL outcomes. The results suggest that overall there are few differences between treatments on the physical, cognitive and emotional dimensions of well-being.
用于评估疾病修正疗法（DMT）的结果测量通常提供有关其对疾病活动影响的重要信息，但它们并未捕捉到多发性硬化症（MS）患者的全部生活影响。患者报告的结果测量（PRO）越来越多地用于捕捉个体对疾病的主观体验。我们比较了 MS 患者在广泛的 PRO 结果中使用的 DMT。
在进行临床神经检查的四周内，SysteMS 中的受试者完成了神经 QoL 的计算机自适应测试版本。神经 QoL 测量包括以下 11 个健康相关生活质量（HRQOL）领域：参与社会角色和活动的能力、焦虑、认知功能、抑郁、情绪和行为失控、疲劳、下肢功能（移动性）、积极影响和幸福感、对社会角色和活动的满意度、耻辱感和上肢功能（精细运动）。根据三种主要的给药方式将治疗方法分为三组：注射、口服和输注。使用线性回归调整两组协变量（第一组：年龄、性别、疾病持续时间和 EDSS；第二组：年龄、性别、疾病持续时间、EDSS 和治疗持续时间）比较三组治疗组。我们还使用线性回归比较了个体治疗方法。
在调整了 MS 的第一组临床和人口统计学特征后，治疗组之间在上肢功能和耻辱感方面存在差异。使用注射治疗的患者在上肢功能和耻辱感方面的报告比使用输注治疗的患者更好。此外，使用注射治疗的患者在上肢功能方面的报告比使用口服 DMT 治疗的患者更好。当比较所有个体治疗方法时，干扰素治疗的患者在耻辱感方面的报告比那他珠单抗治疗的患者明显更好。进一步调整治疗时间后，组间差异减弱且不再具有统计学意义。
我们检查了 MS 治疗组在广泛的 HRQOL 结果方面的差异。结果表明，在身体、认知和情感幸福感方面，治疗之间总体上差异不大。
