Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, USA.
Department of Ophthalmology and Otolaryngology, University of Sao Paulo Medical School, Sao Paulo, Brazil.
Ophthalmic Genet. 2020 Feb;41(1):1-6. doi: 10.1080/13816810.2020.1731836. Epub 2020 Feb 26.
Patient-reported outcome (PRO) measures have the potential to uniquely capture patient experience and serve as an outcome measure in inherited retinal degeneration (IRD) gene therapy trials. An IRD-specific patient-reported outcome measure may yield valuable information that has not been obtained from inherited retinal dystrophy gene therapy trials published to-date. Existing PRO measures have inherent limitations for use in IRD gene therapy trials. Developing an applicable patient-reported outcome measure for such trials needs to incorporate patient input from the target population, demonstrate sound psychometric properties, and be made in accordance with U.S. Food and Drug Administration (FDA) guidelines. This review will discuss the currently available PRO instruments, their limitations for IRD therapeutic trials, and suggestions for future PRO development in IRD populations. The PRO instruments highlighted were identified in PubMed search of English-language journals and previously published review articles.
患者报告的结局（PRO）测量具有独特捕捉患者体验的潜力，并可作为遗传性视网膜变性（IRD）基因治疗试验的结局测量指标。IRD 特异性患者报告结局测量指标可能会提供有价值的信息，而这些信息目前尚未从已发表的遗传性视网膜营养不良基因治疗试验中获得。现有的 PRO 测量指标在用于 IRD 基因治疗试验时存在固有局限性。为这些试验开发适用的患者报告结局测量指标需要纳入目标人群的患者意见，表现出良好的心理测量特性，并符合美国食品和药物管理局（FDA）的指导方针。本综述将讨论目前可用的 PRO 工具，它们在 IRD 治疗试验中的局限性，以及对 IRD 人群中未来 PRO 发展的建议。在 PubMed 对英文期刊和已发表的综述文章的搜索中确定了突出显示的 PRO 工具。
