Braithwaite Tasanee, Calvert Melanie, Gray Alastair, Pesudovs Konrad, Denniston Alastair K
Centre for Patient Reported Outcomes Research and NIHR Birmingham Biomedical Research Centre, University of Birmingham, Edgbaston, Birmingham, UK,
Moorfields Eye Hospital, London, UK,
Patient Relat Outcome Meas. 2019 Jan 24;10:9-24. doi: 10.2147/PROM.S162802. eCollection 2019.
This review article considers the rising demand for patient-reported outcome measures (PROMs) in modern ophthalmic research and clinical practice. We review what PROMs are, how they are developed and chosen for use, and how their quality can be critically appraised. We outline the progress made to develop PROMs in each clinical subspecialty. We highlight recent examples of the use of PROMs as secondary outcome measures in randomized controlled clinical trials and consider the impact they have had. With increasing interest in using PROMs as primary outcome measures, particularly where interventions have been found to be of equivalent efficacy by traditional outcome metrics, we highlight the importance of instrument precision in permitting smaller sample sizes to be recruited. Our review finds that while there has been considerable progress in PROM development, particularly in cataract, glaucoma, medical retina, and low vision, there is a paucity of useful tools for less common ophthalmic conditions. Development and validation of item banks, administered using computer adaptive testing, has been proposed as a solution to overcome many of the traditional limitations of PROMs, but further work will be needed to examine their acceptability to patients, clinicians, and investigators.
这篇综述文章探讨了现代眼科研究和临床实践中对患者报告结局测量指标(PROMs)需求不断上升的情况。我们回顾了PROMs是什么,它们是如何开发和选择使用的,以及如何对其质量进行严格评估。我们概述了在每个临床亚专业中开发PROMs所取得的进展。我们重点介绍了PROMs在随机对照临床试验中作为次要结局指标使用的最新实例,并考虑了它们所产生的影响。随着越来越多的人关注将PROMs用作主要结局指标,特别是在传统结局指标发现干预措施具有同等疗效的情况下,我们强调了仪器精度在允许招募更小样本量方面的重要性。我们的综述发现,虽然在PROMs开发方面取得了相当大的进展,特别是在白内障、青光眼、视网膜内科和低视力领域,但对于不太常见的眼科疾病,有用的工具却很匮乏。有人提议开发和验证使用计算机自适应测试进行管理的题库,作为克服PROMs许多传统局限性的一种解决方案,但还需要进一步开展工作来检验患者、临床医生和研究人员对它们的接受程度。