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富马酸二甲酯治疗复发缓解型多发性硬化症:患者报告的结果与观点

Dimethyl Fumarate in the Treatment of Relapsing-Remitting Multiple Sclerosis: Patient Reported Outcomes and Perspectives.

作者信息

Ozel Osman, Vaughn Caila B, Eckert Svetlana P, Jakimovski Dejan, Lizarraga Alexis A, Weinstock-Guttman Bianca

机构信息

Jacobs MS Center for Treatment and Research, Department of Neurology, Jacobs School of Medicine and Biomedical Sciences, State University of New York at Buffalo, Buffalo, NY, USA.

Buffalo Neuroimaging Analysis Center, Department of Neurology, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York, Buffalo, NY, USA.

出版信息

Patient Relat Outcome Meas. 2019 Dec 11;10:373-384. doi: 10.2147/PROM.S168095. eCollection 2019.

Abstract

Dimethyl fumarate (DMF) is a commonly prescribed oral medication for the treatment of relapsing forms of multiple sclerosis (MS) with a wide range of hypothesized downstream mechanisms of action. Randomized clinical trials have established its clinical efficacy by using standard objective clinical measures. However, MS is a chronic disease that, apart from physical ailments, can affect an individual's mood, psychosocial status, and quality of life which cannot be captured by using only objective assessment tools. Given the challenge of determining the efficacy of the treatment in a real-world clinical setting, the use of patient-reported outcomes (PROs) may help us to better address these aspects of patient care and establish a more patient-centered approach to MS care. To date, a review of PubMed identified six studies which reported on PROs in patients who are taking DMF. In total, twelve different kinds of PRO measures were utilized and 6359 patients provided at least one form of PRO in these studies. Upon review of these studies, we were able to conclude that people with MS had decreased quality of life compared to the healthy population in the US. MS patients on DMF, however, had better health-related quality of life assessment scores compared to those using a placebo. Previous studies also suggested that DMF decreased work productivity impairment scores after one year of use compared to baseline. DMF was associated with less impairment in fatigue and depression scales along with improved treatment quality assessment and adherence scores. This review will present a brief synopsis of the published literature and will provide indications for future directions with respect to PROs and DMF in people with MS.

摘要

富马酸二甲酯(DMF)是一种常用的口服药物,用于治疗复发型多发性硬化症(MS),其下游作用机制有多种假说。随机临床试验已通过标准客观临床指标证实了其临床疗效。然而,MS是一种慢性病,除了身体疾病外,还会影响个体的情绪、心理社会状态和生活质量,而仅使用客观评估工具无法涵盖这些方面。鉴于在现实临床环境中确定治疗效果面临挑战,使用患者报告结局(PROs)可能有助于我们更好地处理患者护理的这些方面,并建立一种更以患者为中心的MS护理方法。截至目前,对PubMed的回顾发现有六项研究报告了服用DMF患者的PROs。这些研究总共使用了十二种不同的PRO测量方法,6359名患者在这些研究中至少提供了一种形式的PRO。在对这些研究进行审查后,我们能够得出结论,与美国健康人群相比,MS患者的生活质量有所下降。然而,与使用安慰剂的患者相比,服用DMF的MS患者的健康相关生活质量评估得分更高。先前的研究还表明,与基线相比,DMF使用一年后工作生产力受损得分有所降低。DMF与疲劳和抑郁量表的损伤减轻相关,同时治疗质量评估和依从性得分有所提高。本综述将简要概述已发表的文献,并为MS患者中PROs和DMF的未来研究方向提供指引。

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本文引用的文献

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Review: Patient-reported outcomes in multiple sclerosis care.综述:多发性硬化症护理中的患者报告结局。
Mult Scler Relat Disord. 2019 Aug;33:61-66. doi: 10.1016/j.msard.2019.05.019. Epub 2019 May 27.
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