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使用多种建模方法从修订后的囊性纤维化问卷中映射EQ-5D指数。

Mapping the EQ-5D index from the cystic fibrosis questionnaire-revised using multiple modelling approaches.

作者信息

Acaster Sarah, Pinder Binny, Mukuria Clara, Copans Amanda

机构信息

Oxford Outcomes Ltd, an Icon plc Company, Seacourt Tower, West Way, Oxford, OX2 0JJ, UK.

ScHARR, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.

出版信息

Health Qual Life Outcomes. 2015 Mar 12;13:33. doi: 10.1186/s12955-015-0224-6.

Abstract

BACKGROUND

This study was designed to develop a mapping algorithm to estimate EQ-5D utility values from Cystic Fibrosis Questionnaire-Revised (CFQ-R) data.

METHODS

A cross-sectional survey of adults with cystic fibrosis (CF) was conducted in the UK. The survey consisted of the CFQ-R, the EQ-5D and a background questionnaire. Eight regression models, exploring item and domain level predictors, were evaluated using three different modelling approaches: ordinary least squares (OLS), Tobit, and a two-part model (TPM). Predictive performance in each model was assessed by intraclass correlations, information criteria (Bayesian information criteria and Alkaike information criteria), and root mean square error (RMSE).

RESULTS

The survey was completed by 401 participants. For all modelling approaches the best performing item level model included all items, and the best performing domain level model included the CFQ-R Physical-, Role- and Emotional-functioning, Vitality, Eating Disturbances, Weight, and Digestive Symptoms domains and a selection of squared terms. Overall, the item level TPM, including age and gender covariates performed best within sample validation, but OLS and TPM domain models with squared terms performed best out-of-sample and are recommended for mapping purposes.

CONCLUSIONS

Domain and item level models using all three modelling approaches reached an acceptable degree of predictive performance with domain models performing well in out-of-sample validation. These mapping functions can be applied to CFQ-R datasets to estimate EQ-5D utility values for economic evaluations of interventions for patients with cystic fibrosis. Further research evaluating model performance in an independent sample is encouraged.

摘要

背景

本研究旨在开发一种映射算法,以根据囊性纤维化问卷修订版(CFQ-R)数据估算EQ-5D效用值。

方法

在英国对成年囊性纤维化(CF)患者进行了一项横断面调查。该调查包括CFQ-R、EQ-5D和一份背景问卷。使用三种不同的建模方法评估了八个探索项目和领域水平预测因素的回归模型:普通最小二乘法(OLS)、 Tobit模型和两部分模型(TPM)。通过组内相关性、信息准则(贝叶斯信息准则和赤池信息准则)和均方根误差(RMSE)评估每个模型的预测性能。

结果

401名参与者完成了调查。对于所有建模方法,表现最佳的项目水平模型包括所有项目,表现最佳的领域水平模型包括CFQ-R的身体功能、角色功能、情绪功能、活力、饮食障碍、体重和消化症状领域以及一些平方项。总体而言,在样本内验证中,包括年龄和性别协变量的项目水平TPM表现最佳,但带有平方项的OLS和TPM领域模型在样本外表现最佳,建议用于映射目的。

结论

使用所有三种建模方法的领域和项目水平模型都达到了可接受的预测性能水平,领域模型在样本外验证中表现良好。这些映射函数可应用于CFQ-R数据集,以估算EQ-5D效用值,用于囊性纤维化患者干预措施的经济评估。鼓励在独立样本中进一步评估模型性能的研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c03/4364087/30af4d6989b5/12955_2015_224_Fig1_HTML.jpg

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