Background

There is increasing emphasis on using patient-reported outcomes (PROs) to complement traditional clinical outcomes in medical research, including in multiple sclerosis (MS). Research, particularly in oncology and heart failure, has shown that PROs can be prognostic of hard clinical endpoints such as survival time (time from study entry until death). However, unlike in oncology or cardiology, it is unknown whether PROs are associated with survival time in neurological diseases. The Multiple Sclerosis Impact Scale–29 (MSIS-29) is a PRO sensitive to short-term change in MS, with questions covering both physical and psychological quality of life. This study aimed to investigate whether MSIS-29 scores can be prognostic for survival time in MS, using a large observational cohort of people with MS.

Methods and findings

From 15 July 2004 onwards, MSIS-29 questionnaires were completed by people with MS registered with the MS Society Tissue Bank (n = 2,126, repeated 1 year later with n = 872 of the original respondents). By 2014, 264 participants (12.4%) had died. Higher baseline MSIS-29 physical (MSIS-29-PHYS) score was associated with reduced survival time (subgroup with highest scores versus subgroup with lowest scores: hazard ratio [HR] 5.7, 95% CI 3.1–10.5, p < 0.001). Higher baseline MSIS-29 psychological score was also associated with reduced survival time (subgroup with highest scores versus subgroup with lowest scores: HR 2.8, 95% CI 1.8–4.4, p < 0.001). In those with high baseline MSIS-29 scores, mortality risk was even greater if the MSIS-29 score worsened over 1 year (HR 2.3, 95% CI 1.2–4.4, p = 0.02). MSIS-29-PHYS scores were associated with survival time independent of age, sex, and patient-reported Expanded Disability Status Scale score in a Cox regression analysis (per 1-SD increase in MSIS-29-PHYS score: HR 1.8, 95% CI 1.1–2.9, p = 0.03). A limitation of the study is that this cohort had high baseline age and disability levels; the prognostic value of MSIS-29 for survival time at earlier disease stages requires further investigation.

Conclusions

This study reports that PROs can be prognostic for hard clinical outcomes in neurological disease, and supports PROs as a meaningful clinical outcome for use in research and clinical settings.

中文翻译：

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患者报告的多发性硬化症结局和生存：​​一项使用多发性硬化症影响量表29的10年回顾性队列研究

背景

在医学研究中，包括多发性硬化症（MS）中，越来越多地强调使用患者报告的结果（PRO）来补充传统的临床结果。研究，特别是在肿瘤学和心力衰竭方面的研究表明，PRO可以预示诸如生存时间（从研究进入到死亡的时间）之类的严格的临床终点。但是，与肿瘤学或心脏病学不同，尚不清楚PROs是否与神经系统疾病的生存时间相关。多发性硬化症影响量表–29（MSIS-29）是对MS短期变化敏感的PRO，其问题涵盖了生理和心理生活质量。这项研究的目的是使用大量的MS观察人群来研究MSIS-29评分是否可以预测MS的生存时间。

方法和发现

从2004年7月15日开始，MSIS-29问卷由在MS协会组织银行注册的MS人士完成（n = 2,126，一年后重复，原始受访者中的n = 872）。到2014年，有264位参与者（12.4％）死亡。较高的基线MSIS-29物理（MSIS-29-PHYS）评分与生存时间减少相关（得分最高的组与得分最低的子组：危险比[HR] 5.7，95％CI 3.1-10.5，p < 0.001）。较高的基线MSIS-29心理得分也与生存时间减少相关（得分最高的小组与得分最低的小组：HR 2.8，95％CI 1.8-4.4，p <0.001）。在基线MSIS-29评分较高的人群中，如果MSIS-29评分在1年内恶化，则死亡风险甚至更高（HR 2.3，95％CI 1.2-4.4，p = 0.02）。在Cox回归分析中，MSIS-29-PHYS得分与生存时间相关，与年龄，性别和患者报告的扩展残疾状态量表得分无关（MSIS-29-PHYS得分每1-SD增加：HR 1.8，95％ CI 1.1–2.9，p = 0.03）。该研究的局限性在于该队列具有较高的基线年龄和残疾水平。MSIS-29对疾病早期阶段生存时间的预后价值需要进一步研究。

结论

这项研究报告说，PROs可以预示神经系统疾病的硬性临床预后，并支持PROs作为有意义的临床预后，用于研究和临床环境。