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密歇根视网膜变性问卷:一种用于遗传性视网膜变性的患者报告结局工具。

The Michigan Retinal Degeneration Questionnaire: A Patient-Reported Outcome Instrument for Inherited Retinal Degenerations.

机构信息

Kellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, USA.

Kellogg Eye Center, 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, São Paulo, Brazil.

出版信息

Am J Ophthalmol. 2021 Feb;222:60-68. doi: 10.1016/j.ajo.2020.08.032. Epub 2020 Aug 26.

DOI:10.1016/j.ajo.2020.08.032
PMID:32858027
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7907279/
Abstract

PURPOSE

To create a psychometrically validated patient-reported outcome measure for inherited retinal degenerations.

DESIGN

Qualitative and quantitative patient-reported outcome (PROs) questionnaire development using item response theory validation.

METHODS

One hundred twenty-eight patients with a diagnosis of an inherited retinal degeneration at the Kellogg Eye Center (University of Michigan) were recruited and administered a 166-item questionnaire comprising 7 expert-defined domains. The questionnaire was re-administered 4-16 days later to a subset of 25 participants to assess test-retest variability. Graded response models were fit by Cai's Metropolis-Hastings Robbins-Monro algorithm using the R (version 3.6.3) package mirt. Model data were fit to assess questionnaire dimensionality, to estimate item information, and to score participants. Poorly functioning items were removed, and the model was refit to create the final questionnaire.

RESULTS

The psychometrically validated PROs measure was reduced to a 59-item questionnaire measuring 7 unidimesnional domains: central vision, color vision, contrast sensitivity, scotopic function, photopic peripheral vision, mesopic peripheral vision, and photosensitivity. A total of 39 items were removed because of poor factor loading, low item information, poor person-ability differentiation, or high item-level interdependence. This novel questionnaire produces a reliable domain score for person ability that does not show significant test-retest variability across repeated administration.

CONCLUSIONS

The final PRO questionnaire, known as the Michigan Retinal Degeneration Questionnaire, is psychometrically validated and available for use in the evaluation of patients with inherited retinal degenerations.

摘要

目的

开发一种经过心理测量学验证的遗传性视网膜变性患者报告结局测量工具。

设计

使用项目反应理论验证,进行定性和定量的患者报告结局(PRO)问卷开发。

方法

在密歇根大学凯洛格眼科中心(Kellogg Eye Center)招募了 128 名诊断为遗传性视网膜变性的患者,并对其进行了一项包含 7 个专家定义领域的 166 项问卷测试。随后,对 25 名参与者中的一小部分进行了 4-16 天的二次测试,以评估测试-重测的变异性。使用 Cai 的 Metropolis-Hastings Robbins-Monro 算法通过 R(版本 3.6.3)包 mirt 拟合等级反应模型。模型数据用于评估问卷的维度性、估计项目信息和对参与者进行评分。剔除功能不佳的项目,然后重新拟合模型以创建最终的问卷。

结果

经过心理测量学验证的 PRO 测量工具被简化为一个 59 项的问卷,测量 7 个单维度领域:中央视力、色觉、对比敏感度、暗适应功能、明适应周边视力、中间适应周边视力和光敏感。共有 39 个项目因因子负荷不良、项目信息低、人员能力区分差或项目水平的相互依存性高而被剔除。这个新颖的问卷产生了一个可靠的人员能力领域得分,在多次重复测试中没有显著的测试-重测变异性。

结论

最终的 PRO 问卷,即密歇根视网膜变性问卷,具有心理测量学验证,并可用于评估遗传性视网膜变性患者。

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