Importance There is a need for validated clinical end points that are reliably able to quantify potential therapeutic effects of future treatments targeting age-related macular degeneration (AMD) before the onset of serious visual impairment. Objective To assess the reliability and discriminatory power of 5 simple chart-based visual function (VF) tests as potential measures for clinical trial end points with regulatory and patient-access intention in intermediate AMD (iAMD). Design, Setting, and Participants This international noninterventional study took place at 18 tertiary ophthalmology departments across Europe. Participants were recruited between April 2018 and March 2020 and were identified during routine clinical review. Participants with no AMD and early AMD were recruited from hospital staff, friends, and family of participants with AMD and via referrals from community ophthalmologists and optometrists. The repeatability and discriminatory power of 5 simple chart-based assessments of VF (best-corrected visual acuity [BCVA], low-luminance visual acuity [LLVA], Moorfields Acuity Test [MAT], Pelli-Robson Contrast Sensitivity [CS], and International Reading Speed Test [IReST]) were assessed in a repeated-measures design. VF assessments were performed on day 0 and day 14. Participants with early AMD, iAMD, late AMD, and no AMD were recruited. Main Outcomes and Measures Intraclass correlation coefficients (ICCs) and Bland-Altman 95% limits of agreement (LoA) were computed to assess repeatability. Area under the receiver operating characteristic curves (AUCs) determined the discriminatory ability of all measures to classify individuals as having no AMD or iAMD and to differentiate iAMD from its neighboring disease states. Results A total of 301 participants (mean [SD] age, 71 [7] years; 187 female participants [62.1%]) were included in the study. Thirty-four participants (11.3%) had early AMD, 168 (55.8%) had iAMD, 43 (14.3%) had late AMD, and 56 (18.6%) had no AMD. ICCs for all VF measures ranged between 0.88 and 0.96 when all participants were considered, indicating good to excellent repeatability. All measures displayed excellent discrimination between iAMD and late AMD (AUC, 0.92-0.99). Early AMD was indistinguishable from iAMD on all measures (AUC, 0.54-0.64). CS afforded the best discrimination between no AMD and iAMD (AUC, 0.77). Under the same conditions, BCVA, LLVA, and MAT were fair discriminators (AUC, 0.69-0.71), and IReST had poor discrimination (AUC, 0.57-0.61). Conclusions and Relevance BCVA, LLVA, MAT, CS, and IReST had adequate repeatability in this multicenter, multiexaminer setting but limited power to discriminate between no AMD and iAMD. The prognostic power of these variables to predict conversion from iAMD to late AMD is being examined in the ongoing longitudinal part of the MACUSTAR study.

中文翻译：

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年龄相关性黄斑变性患者基于图表的视觉功能测试的可重复性和辨别力：MACUSTAR 研究报告。

重要性 需要经过验证的临床终点，能够可靠地量化未来针对年龄相关性黄斑变性 (AMD) 的治疗在严重视力障碍发作之前的潜在治疗效果。目的 评估 5 种简单的基于图表的视觉功能 (VF) 测试的可靠性和辨别力，作为在中间 AMD (iAMD) 中具有监管和患者访问意图的临床试验终点的潜在衡量标准。设计、设置和参与者 这项国际非干预性研究在欧洲的 18 个三级眼科进行。参与者于 2018 年 4 月至 2020 年 3 月期间招募，并在常规临床审查期间确定。没有 AMD 和早期 AMD 的参与者是从医院工作人员、朋友、和 AMD 参与者的家人，并通过社区眼科医生和验光师的推荐。5 种简单的基于图表的 VF 评估（最佳矫正视力 [BCVA]、低亮度视力 [LLVA]、Moorfields 视力测试 [MAT]、Pelli-Robson 对比敏感度 [CS] 和国际阅读速度测试 [IReST]）采用重复测量设计进行评估。在第 0 天和第 14 天进行了 VF 评估。招募了早期 AMD、iAMD、晚期 AMD 和无 AMD 的参与者。主要结果和措施 计算组内相关系数 (ICC) 和 Bland-Altman 95% 一致性限制 (LoA) 以评估可重复性。接受者操作特征曲线下面积 (AUC) 决定了所有措施将个体分类为没有 AMD 或 iAMD 并将 iAMD 与其邻近疾病状态区分开来的区分能力。结果共有 301 名参与者（平均 [SD] 年龄，71 [7] 岁；187 名女性参与者 [62.1%]）被纳入研究。34 名参与者 (11.3%) 患有早期 AMD，168 名 (55.8%) 患有 iAMD，43 名 (14.3%) 患有晚期 AMD，56 名 (18.6%) 没有 AMD。当考虑所有参与者时，所有 VF 测量的 ICC 范围在 0.88 和 0.96 之间，表明可重复性良好至极好。所有测量都显示出 iAMD 和晚期 AMD 之间的出色区分（AUC，0.92-0.99）。早期 AMD 在所有指标上都与 iAMD 没有区别（AUC，0.54-0.64）。CS 在无 AMD 和 iAMD 之间提供了最好的区分（AUC，0.77）。相同条件下，BCVA、LLVA、MAT是公平判别者（AUC，0.69-0.71），IReST判别力差（AUC，0.57-0.61）。结论和相关性 BCVA、LLVA、MAT、CS 和 IReST 在这种多中心、多检查者设置中具有足够的可重复性，但区分无 AMD 和 iAMD 的能力有限。MACUSTAR 研究正在进行的纵向部分正在检查这些变量预测从 iAMD 转变为晚期 AMD 的预后能力。