使用混合模型作为映射工具方法的进展: 在哮喘患者中将 AQLQ-S 与 EQ-5D-5L 和 HUI3进行映射
2018/07/09
摘要
背景：研究表明，将临床与个性化方法进行映射时使用混合模型效果良好。
目的：以不同的方式开发这些方法, 并在案例研究中比较性能。
方法：在获得的856例哮喘患者数据中将哮喘生活质量调查表与五级 EuroQol 五维问卷 (EQ-5D-5L) 和卫生设施指数 3 (HUI3) 进行映射。评估经调整的有限依赖变量混合模型和基于β的混合模型。探讨在全面健康与下一个值和下一个可行值的质点之间的区别。
结果：在所有案例中，对完全健康和下一个可行值之间的区别正式构建具体模型是一种改进。由于分布不均匀，对HUI3进行映射比对EQ-5D-5L进行映射需要混合模型中的更多组件。用基于β的优化混合模型映射 HUI3时包括相邻的全面健康概率质点的效用值。因为观察比例低，当估计EQ-5D-5L时情况并非如此。
结论：在组件数量相同的数据集中，基于β的混合模型略优于经调整的有限依赖变量混合模型。然而, 它们需要更多的参数和更长的估计时间。两种混合模型类型均与 EQ-5D-5L 和HUI数据紧密吻合。标准映射方法通常会导致对健康增益估计偏倚。混合模型没有这种偏倚。两者混合模型都可用于成本效益研究。今后在其他疾病领域的映射研究应考虑类似的方法。
Development of Methods for the Mapping of Utilities Using Mixture Models: Mapping the AQLQ-S to the EQ-5D-5L and the HUI3 in Patients with Asthma.
Gray LA, Hernández Alava M, Wailoo AJ.
Abstract
BACKGROUND: Studies have shown that methods based on mixture models work well when mapping clinical to preference-based methods.
OBJECTIVES: To develop these methods in different ways and to compare performance in a case study.
METHODS: Data from 856 patients with asthma allowed mapping between the Asthma Quality of Life Questionnaire and both the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) and the health utilities index mark 3 (HUI3). Adjusted limited dependent variable mixture models and beta-based mixture models were estimated. Optional inclusion of the gap between full health and the next value as well as a mass point at the next feasible value were explored.
RESULTS: In all cases, model specifications formally modeling the gap between full health and the next feasible value were an improvement on those that did not. Mapping to the HUI3 required more components in the mixture models than did mapping to the EQ-5D-5L because of its uneven distribution. The optimal beta-based mixture models mapping to the HUI3 included a probability mass at the utility value adjacent to full health. This is not the case when estimating the EQ-5D-5L, because of the low proportion of observations at this point.
CONCLUSIONS: Beta-based mixture models marginally outperformed adjusted limited dependent variable mixture models with the same number of components in this data set. Nevertheless, they require a larger number of parameters and longer estimation time. Both mixture model types closely fit both EQ-5D-5L and HUI data. Standard mapping approaches typically lead to biased estimates of health gain. The mixture model approaches exhibit no such bias. Both can be used with confidence in applied cost-effectiveness studies. Future mapping studies in other disease areas should consider similar methods.
背景：研究表明，将临床与个性化方法进行映射时使用混合模型效果良好。
目的：以不同的方式开发这些方法, 并在案例研究中比较性能。
方法：在获得的856例哮喘患者数据中将哮喘生活质量调查表与五级 EuroQol 五维问卷 (EQ-5D-5L) 和卫生设施指数 3 (HUI3) 进行映射。评估经调整的有限依赖变量混合模型和基于β的混合模型。探讨在全面健康与下一个值和下一个可行值的质点之间的区别。
结果：在所有案例中，对完全健康和下一个可行值之间的区别正式构建具体模型是一种改进。由于分布不均匀，对HUI3进行映射比对EQ-5D-5L进行映射需要混合模型中的更多组件。用基于β的优化混合模型映射 HUI3时包括相邻的全面健康概率质点的效用值。因为观察比例低，当估计EQ-5D-5L时情况并非如此。
结论：在组件数量相同的数据集中，基于β的混合模型略优于经调整的有限依赖变量混合模型。然而, 它们需要更多的参数和更长的估计时间。两种混合模型类型均与 EQ-5D-5L 和HUI数据紧密吻合。标准映射方法通常会导致对健康增益估计偏倚。混合模型没有这种偏倚。两者混合模型都可用于成本效益研究。今后在其他疾病领域的映射研究应考虑类似的方法。
(中日友好医院呼吸与危重症医学科 禹汶伯 摘译 林江涛 审校)
(Value Health. 2018 Jun;21(6):748-757. )
(Value Health. 2018 Jun;21(6):748-757. )
Development of Methods for the Mapping of Utilities Using Mixture Models: Mapping the AQLQ-S to the EQ-5D-5L and the HUI3 in Patients with Asthma.
Gray LA, Hernández Alava M, Wailoo AJ.
Abstract
BACKGROUND: Studies have shown that methods based on mixture models work well when mapping clinical to preference-based methods.
OBJECTIVES: To develop these methods in different ways and to compare performance in a case study.
METHODS: Data from 856 patients with asthma allowed mapping between the Asthma Quality of Life Questionnaire and both the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) and the health utilities index mark 3 (HUI3). Adjusted limited dependent variable mixture models and beta-based mixture models were estimated. Optional inclusion of the gap between full health and the next value as well as a mass point at the next feasible value were explored.
RESULTS: In all cases, model specifications formally modeling the gap between full health and the next feasible value were an improvement on those that did not. Mapping to the HUI3 required more components in the mixture models than did mapping to the EQ-5D-5L because of its uneven distribution. The optimal beta-based mixture models mapping to the HUI3 included a probability mass at the utility value adjacent to full health. This is not the case when estimating the EQ-5D-5L, because of the low proportion of observations at this point.
CONCLUSIONS: Beta-based mixture models marginally outperformed adjusted limited dependent variable mixture models with the same number of components in this data set. Nevertheless, they require a larger number of parameters and longer estimation time. Both mixture model types closely fit both EQ-5D-5L and HUI data. Standard mapping approaches typically lead to biased estimates of health gain. The mixture model approaches exhibit no such bias. Both can be used with confidence in applied cost-effectiveness studies. Future mapping studies in other disease areas should consider similar methods.
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了解患者对哮喘控制的观念：一项定性研究
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哮喘药物治疗依从性的障碍:风俗及环境的重要性
