Altabee Rana, Carr Siobhan B, Abbott Janice, Cameron Rory, Office Daniel, Simmonds Nicholas J, Whitty Jennifer A, Turner David, Barton Garry
Health Economics Group, Norwich Medical School, University of East Anglia, Norwich NR4 7TJ, Norfolk, UK; College of Applied Medical Sciences, King Saud Bin Abdulaziz University for Health Sciences, Jeddah 22384, Saudi Arabia; King Abdullah International Medical Research Center, Jeddah 22384, Saudi Arabia.
Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London SW3 6NP, UK; National Heart and Lung Institute, Imperial College London, London SW7 2BX, UK.
Respir Med Res. 2024 Nov;86:101137. doi: 10.1016/j.resmer.2024.101137. Epub 2024 Aug 20.
The EQ-5D is the recommended measure to capture health-related quality of life (HRQoL), recognised for use in health technology appraisal bodies. In order to assess whether it is appropriate to use the EQ-5D for making decisions about the cost-utility of treatments in cystic fibrosis (CF), this study assesses the performance of the EQ-5D-5L in adults and adolescents with CF.
This was a cross-sectional observational survey study of patients with CF attending a single large CF centre. Participants were asked to complete a survey that included two HRQoL measures; the EQ-5D-5L and CF Quality of Life (CFQoL) questionnaires.
Among 213 participants, the median EQ-5D-5L index score was 0.76 (IQR 0.66 - 0.84) and the visual analogue (EQ-VAS) was 70 (60 - 80). Both the EQ-5D index and EQ-VAS discriminated between disease severity based on lung function (p = 0.01 and p < 0.01, respectively) and pulmonary exacerbation (p = 0.02 and p < 0.01, respectively); however, EQ-VAS differentiated between more lung function severity groups compared to EQ-5D index. The EQ-5D-5L demonstrated convergent validity as its dimensions, index score, and EQ-VAS had significant correlations with most CFQoL domains. Though, EQ-VAS significantly predicted more domains of CFQoL (4 domains) compared to EQ-5D index (only 1 domain).
The generic EQ-5D-5L performed adequately in discriminating between CF disease severity, and its index score and EQ-VAS had moderate correlations with CFQoL. However, using a complementary condition-specific measure alongside the EQ-5D-5L can provide better insight of HRQoL in CF and benefit the process of cost-utility analysis.
EQ-5D是用于评估健康相关生活质量（HRQoL）的推荐工具，被健康技术评估机构认可使用。为了评估在囊性纤维化（CF）中使用EQ-5D来做出治疗成本效用决策是否合适，本研究评估了成人和青少年CF患者中EQ-5D-5L的性能。
这是一项对在一个大型CF中心就诊的CF患者进行的横断面观察性调查研究。参与者被要求完成一项调查，其中包括两项HRQoL测量；EQ-5D-5L和CF生活质量（CFQoL）问卷。
在213名参与者中，EQ-5D-5L指数得分中位数为0.76（四分位距0.66 - 0.84），视觉模拟评分（EQ-VAS）为70（60 - 80）。EQ-5D指数和EQ-VAS均能根据肺功能（分别为p = 0.01和p < 0.01）以及肺部加重情况（分别为p = 0.02和p < 0.01）区分疾病严重程度；然而，与EQ-5D指数相比，EQ-VAS能在更多肺功能严重程度组之间进行区分。EQ-5D-5L显示出收敛效度，因为其维度、指数得分和EQ-VAS与大多数CFQoL领域具有显著相关性。不过，与EQ-5D指数（仅1个领域）相比，EQ-VAS能显著预测更多CFQoL领域（4个领域）。
通用的EQ-5D-5L在区分CF疾病严重程度方面表现良好，其指数得分和EQ-VAS与CFQoL具有中等相关性。然而，在EQ-5D-5L之外使用一种补充的针对特定疾病的测量方法可以更好地洞察CF中的HRQoL，并有益于成本效用分析过程。
