Lacy Gabrielle D, Abalem Maria Fernanda, Popova Lilia T, Santos Erin P, Yu Gina, Rakine Hanan Y, Rosenthal Julie M, Ehrlich Joshua R, Musch David C, Jayasundera K Thiran
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 Aug;41(4):315-324. doi: 10.1080/13816810.2020.1776337. Epub 2020 Jun 22.
Generate content for a patient-reported outcome (PRO) measure for use in future clinical trials for inherited retinal degenerations.
Patients at the University of Michigan Kellogg Eye Center with a clinical diagnosis of inherited retinal degeneration with varying phenotypes were recruited for interviews. First, in-depth interviews were performed to solicit a wide range of patient experiences pertaining to visual function. Coders qualitatively analyzed the transcripts from these interviews using Atlas.ti software (Version 8.1.3 (522)) to draft questionnaire items. Next, the questionnaire was tested and refined based on participant feedback in cognitive interviews and administrator feedback in the pilot survey administration (pilot interviews).
A total of 55 participants with a clinical diagnosis of inherited retinal degeneration were interviewed throughout the three study phases: in-depth interviews (n = 26), cognitive interviews (n = 16), and pilot interviews (n = 13). Coded items were analyzed for frequency of occurrence and related themes, then organized into common domains. Within each domain, PRO items were drafted to address the functional limitations or adaptations experienced by patients.
Items for a PRO measure have been drafted and evaluated for interpretability in the target inherited retinal degeneration patient population. Content validity for the items was established through a process of in-depth interviews, cognitive interviews, and pilot interviews.
生成一份患者报告结局（PRO）测量内容，用于未来遗传性视网膜变性的临床试验。
招募密歇根大学凯洛格眼科中心临床诊断为不同表型遗传性视网膜变性的患者进行访谈。首先，进行深入访谈以收集与视觉功能相关的广泛患者体验。编码人员使用Atlas.ti软件（版本8.1.3（522））对这些访谈的记录进行定性分析，以起草问卷项目。接下来，根据认知访谈中的参与者反馈和预试验管理（预试验访谈）中的管理员反馈对问卷进行测试和完善。
在三个研究阶段共访谈了55名临床诊断为遗传性视网膜变性的参与者：深入访谈（n = 26）、认知访谈（n = 16）和预试验访谈（n = 13）。对编码项目进行出现频率和相关主题分析，然后组织成共同领域。在每个领域内，起草PRO项目以解决患者经历的功能限制或适应情况。
已起草PRO测量的项目并在目标遗传性视网膜变性患者群体中对其可解释性进行了评估。通过深入访谈、认知访谈和预试验访谈的过程确立了项目的内容效度。
