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LEAVO 试验中黄斑水肿患者的视力到 EQ-5D、带视觉附加模块的 EQ-5D 以及 VFQ-UI 的映射。

Mapping From Visual Acuity to EQ-5D, EQ-5D With Vision Bolt-On, and VFQ-UI in Patients With Macular Edema in the LEAVO Trial.

机构信息

School of Health and Related Research, University of Sheffield, Sheffield, England, UK.

School of Health and Related Research, University of Sheffield, Sheffield, England, UK.

出版信息

Value Health. 2020 Jul;23(7):928-935. doi: 10.1016/j.jval.2020.03.008. Epub 2020 Jun 2.

Abstract

OBJECTIVES

Mappings to convert clinical measures to preference-based measures of health such as the EQ-5D-3L are sometimes required in cost-utility analyses. We developed mappings to convert best-corrected visual acuity (BCVA) to the EQ-5D-3L, the EQ-5D-3L with a vision bolt-on (EQ-5D V), and the Visual Functioning Questionnaire-Utility Index (VFQ-UI) in patients with macular edema caused by central retinal vein occlusion.

METHODS

We used data from Lucentis, Eylea, Avastin in vein occlusion (LEAVO), which is a phase-3 randomized controlled trial comparing ranibizumab, aflibercept, and bevacizumab in 463 patients with observations at 6 time points. We estimated adjusted limited dependent variable mixture models consisting of 1 to 4 distributions (components) using BCVA in each eye, age, and sex to predict utility within the components and BCVA as a determinant of component membership. We compared model fit using mean error, mean absolute error, root mean square error, Akaike information criteria, Bayesian information criteria, and visual inspection of mean predicted and observed utilities and cumulative distribution functions.

RESULTS

Mean utility scores were 0.82 for the EQ-5D-3L, 0.79 for the EQ-5D V, and 0.88 for the VFQ-UI. The best-fitting models for the EQ-5D and EQ-5D V had 2 components (with means of approximately 0.44 and 0.85), and the best-fitting model for VFQ-UI had 3 components (with means of approximately 0.95, 0.74, and 0.90).

CONCLUSIONS

Models with multiple components better predict utility than those with single components. This article provides a valuable addition to the literature, in which previous mappings in visual acuity have been limited to linear regressions, resulting in unfounded assumptions about the distribution of the dependent variable.

摘要

目的

在成本效用分析中,有时需要将临床指标转换为基于偏好的健康指标,例如 EQ-5D-3L。我们开发了将最佳矫正视力(BCVA)转换为 EQ-5D-3L、带有视力附加物的 EQ-5D-3L(EQ-5D V)和视觉功能问卷效用指数(VFQ-UI)的映射方法,用于治疗由视网膜中央静脉阻塞引起的黄斑水肿患者。

方法

我们使用了 Lucentis、Eylea、Avastin in vein occlusion(LEAVO)中的数据,这是一项比较 ranibizumab、aflibercept 和 bevacizumab 在 463 名患者中的 3 期随机对照试验,观察了 6 个时间点。我们使用每只眼睛的 BCVA、年龄和性别,对包含 1 到 4 个分布(成分)的调整后的有限因变量混合模型进行了估计,以预测各成分内的效用,同时 BCVA 作为成分归属的决定因素。我们使用平均误差、平均绝对误差、均方根误差、Akaike 信息准则、贝叶斯信息准则以及对平均预测和观察效用和累积分布函数的直观检查,比较了模型拟合度。

结果

EQ-5D-3L 的平均效用评分为 0.82,EQ-5D V 为 0.79,VFQ-UI 为 0.88。EQ-5D 和 EQ-5D V 的最佳拟合模型有 2 个成分(平均值约为 0.44 和 0.85),VFQ-UI 的最佳拟合模型有 3 个成分(平均值约为 0.95、0.74 和 0.90)。

结论

多成分模型比单成分模型更好地预测效用。本文为文献提供了有价值的补充,之前的视力映射仅限于线性回归,对因变量的分布做出了不合理的假设。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fd33/7427317/f8f916f0f45a/gr1.jpg

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