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多发性硬化症患者离散选择实验和联合分析研究的系统评价。

A Systematic Review of Discrete-Choice Experiments and Conjoint Analysis Studies in People with Multiple Sclerosis.

机构信息

Leeds Institute for Health Sciences, University of Leeds, Leeds, UK.

School of Dentistry, University of Leeds, Leeds, UK.

出版信息

Patient. 2018 Aug;11(4):391-402. doi: 10.1007/s40271-017-0296-y.

Abstract

BACKGROUND

Multiple sclerosis (MS) is a chronic disabling, inflammatory, and degenerative disease of the central nervous system that, in most cases, requires long-term disease-modifying treatment (DMT). The drugs used vary in efficacy and adverse effect profiles. Several studies have used attribute-based stated-preference methods, primarily to investigate patient preferences for initiating or escalating DMT.

OBJECTIVES

To conduct a systematic review of attribute-based stated-preference studies in people with MS to identify common methods employed and to assess study quality, with reference to the specific challenges of this disease area.

METHODS

We conducted a systematic search for studies related to attribute-based stated-preference and MS in multiple databases, including Cochrane and MEDLINE. Studies were included if they were published in a peer-reviewed journal, were on the topic of MS, and used a survey methodology that measured stated preferences for attributes of a whole. Analysis was conducted using narrative synthesis and summary statistics. Study quality was judged against the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conjoint analysis checklist.

RESULTS

We identified 16 relevant articles reporting 17 separate studies, all but one focusing on DMTs. Most studies were discrete-choice experiments. Study quality was generally high, but we recommend the following: (1) that consideration of sample sizes be improved, (2) that survey design choices be justified and documented, (3) that qualitative approaches for attribute and level selection be incorporated to better involve patients, and (4) that reporting of experimental practice be improved. The effects of DMTs on reproduction and the impact of how risk and uncertainty are presented were identified as neglected research topics. The ISPOR conjoint analysis checklist was found to be unsuitable for the assessment of study quality.

CONCLUSION

Attribute-based stated preference is a useful method with which to examine the preferences of people with MS in their choice of DMT. However, further research embracing the methodological recommendations identified, particularly greater use of qualitative methods in attribute development, is needed.

摘要

背景

多发性硬化症(MS)是一种中枢神经系统的慢性致残性、炎症性和退行性疾病,在大多数情况下需要长期的疾病修正治疗(DMT)。所使用的药物在疗效和不良反应谱方面存在差异。一些研究已经使用基于属性的陈述偏好方法,主要是为了调查患者对启动或升级 DMT 的偏好。

目的

对多发性硬化症患者基于属性的陈述偏好研究进行系统综述,以确定常用方法,并参考该疾病领域的具体挑战,评估研究质量。

方法

我们在多个数据库(包括 Cochrane 和 MEDLINE)中对与基于属性的陈述偏好和多发性硬化症相关的研究进行了系统检索。如果研究发表在同行评议的期刊上,主题是多发性硬化症,并且使用了测量整体属性的偏好的调查方法,则将其纳入研究。分析采用叙述性综合和汇总统计。使用国际药物经济学和结果研究学会(ISPOR)联合分析检查表来评估研究质量。

结果

我们确定了 16 篇相关文章,报道了 17 项独立研究,除一项外,其余均聚焦于 DMT。大多数研究都是离散选择实验。研究质量总体较高,但我们建议:(1)改进样本量的考虑,(2)证明和记录调查设计选择,(3)纳入属性和水平选择的定性方法,以更好地让患者参与,(4)改进实验实践的报告。DMT 对生殖的影响以及风险和不确定性呈现方式的影响被确定为被忽视的研究课题。发现国际药物经济学和结果研究学会(ISPOR)联合分析检查表不适合评估研究质量。

结论

基于属性的陈述偏好是一种有用的方法,可以用来研究多发性硬化症患者在选择 DMT 时的偏好。然而,需要进一步研究,包括在属性开发中更多地使用定性方法,以采纳本文提出的方法学建议。

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