Product Lifecycle Services - NBS, Novartis Healthcare Private Limited, Hyderabad, India.
Product Lifecycle Services - NBS, Novartis Global Service Center, Dublin, Ireland.
Eur J Neurol. 2017 Sep;24(9):1099-1107. doi: 10.1111/ene.13339. Epub 2017 Jul 11.
Multiple patient-reported outcomes (PROs) are currently being used in multiple sclerosis (MS) but their application is inconsistent and guidance on the appropriateness of each tool is lacking. The objective of our study was to identify MS-specific PROs and systematically to assess the development process and the reliability and validity of various instruments. A systematic literature search was conducted on multiple data sources, including MEDLINE, Embase (using the Ovid platform) and Google Scholar, from 1996 to March 2015. Search terms included combinations of MS, PROs and quality of life. Randomized controlled trials or observational studies conducted on patients with MS and published in English were included. In addition, the PROQOLID database was explored. The MS-specific PROs were systematically assessed using the Evaluating the Measurement of Patient-Reported Outcomes tool. In total, 8094 articles were screened and 405 PROs were identified from 1102 relevant articles. PROs were classified into MS-specific (n = 82) and non-MS-specific (n = 323). The results for the eight PROs that are most commonly used in MS clinical trials are presented here. For these eight PROs, the overall summary scores ranged between 50.1 and 68.7. The Multiple Sclerosis Impact Scale-29 had the best overall mean score (68.7), followed by the Leeds Multiple Sclerosis Quality of Life (67.0). This is the first study to provide a standardized assessment of all PROs for MS. There is a lack of data on content validity for PROs used in MS research, which indicates the need for a robust instrument in MS developed according to the US Food and Drug Administration guidelines.
目前在多发性硬化症（MS）中使用了多种患者报告的结局（PRO），但它们的应用不一致，缺乏对每种工具的适宜性的指导。我们的研究目的是确定 MS 特异性 PRO，并系统地评估各种工具的开发过程以及可靠性和有效性。我们在多个数据源上进行了系统的文献检索，包括 MEDLINE、Embase（使用 Ovid 平台）和 Google Scholar，检索时间从 1996 年到 2015 年 3 月。搜索词包括 MS、PRO 和生活质量的组合。纳入的研究为发表在英文期刊上的、针对 MS 患者的随机对照试验或观察性研究。此外，还探索了 PROQOLID 数据库。使用评估患者报告结局测量工具对 MS 特异性 PRO 进行了系统评估。共筛选了 8094 篇文章，从 1102 篇相关文章中确定了 405 个 PRO。将 PRO 分为 MS 特异性（n=82）和非 MS 特异性（n=323）。本文介绍了在 MS 临床试验中最常用的八个 PRO 的结果。对于这八个 PRO，总体汇总得分在 50.1 到 68.7 之间。多发性硬化症影响量表-29 的总体平均得分最高（68.7），其次是利兹多发性硬化症生活质量量表（67.0）。这是第一项对所有 MS PRO 进行标准化评估的研究。用于 MS 研究的 PRO 的内容效度数据缺乏，这表明需要根据美国食品和药物管理局的指南开发用于 MS 的稳健仪器。
