Purpose

Generate content for a patient-reported outcome (PRO) measure for use in future clinical trials for inherited retinal degenerations.

Methods

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).

Results

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.

Conclusions

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.

中文翻译：

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用于视网膜变性治疗试验的患者报告结果测量的内容生成。

目的

生成患者报告结果 (PRO) 测量的内容，以用于未来遗传性视网膜变性的临床试验。

方法

密歇根大学凯洛格眼科中心临床诊断为具有不同表型的遗传性视网膜变性的患者被招募进行采访。首先，进行了深入访谈，以征求与视觉功能有关的各种患者体验。编码人员使用 Atlas.ti 软件（版本 8.1.3 (522)）对这些访谈的笔录进行定性分析，以起草问卷项目。接下来，根据认知访谈中的参与者反馈和试点调查管理（试点访谈）中的管理员反馈对问卷进行了测试和完善。

结果

共有 55 名临床诊断为遗传性视网膜变性的参与者在三个研究阶段接受了采访：深度访谈 (n = 26)、认知访谈 (n = 16) 和试点访谈 (n = 13)。对编码项目的出现频率和相关主题进行分析，然后将其组织到公共域中。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。

结论

PRO 测量的项目已经起草并评估在目标遗传性视网膜变性患者群体中的可解释性。项目的内容效度是通过深入访谈、认知访谈和试点访谈的过程建立的。