School of Health and Related Research, The University of Sheffield, Sheffield, UK.
Acaster Lloyd Consulting Ltd, London, UK.
J Patient Rep Outcomes. 2024 Feb 28;8(1):24. doi: 10.1186/s41687-024-00697-w.
This study aimed to compare the psychometric performance of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions (CFQ-R-8D), a new, condition-specific, preference-based measure, with that of generic preference-based measures EQ-5D-3L and Short Form 6 dimensions (SF-6D).
Data from three trials of participants with CF aged ≥ 14 years who completed the CFQ-R and EQ-5D-3L or SF-6D were used. Analyses were undertaken to evaluate convergent validity based on correlations with CFQ-R domain scores. Known-group validity was assessed based on percent predicted forced expiratory volume in one second and pulmonary exacerbations. Responsiveness was based on correlation of change and sensitivity to change based on change in symptom severity. Effect sizes and standardized response means were estimated.
CFQ-R-8D utilities and dimensions were strongly correlated with most of the overlapping CFQ-R domain scores (ρ > 0.5); EQ-5D-3L and SF-6D utilities and dimensions had moderate (ρ > 0.3) to strong correlations in dimensions capturing similar concepts. All measures showed evidence of known-group validity (P < 0.05). Change correlations were strong for CFQ-R-8D utilities and dimensions and CFQ-R, but they were moderate for SF-6D and mostly weak ((ρ > 0.1) for EQ-5D-3L. The SF-6D had the largest mean change over time and effect sizes, followed by CFQ-R-8D and then EQ-5D-3L. Neither CFQ-R-8D or SF-6D utility scores had ceiling effects (< 9% responses in full health) compared with those of EQ-5D-3L (61-62%). In participants classified as being in full health by EQ-5D-3L, CFQ-R-8D captured CF-specific health problems, particularly cough, abdominal pain, and breathing difficulty.
The CFQ-R-8D reflected known-group differences and changes over time with stronger evidence of good psychometric performance than EQ-5D-3L and similar evidence as SF-6D. Additionally, the CFQ-R-8D captured more condition-specific symptoms than EQ-5D-3L or SF-6D, which are important determinants of health-related quality of life for people with CF.
本研究旨在比较囊性纤维化问卷修订版 8 维度(CFQ-R-8D)与通用偏好量表 EQ-5D-3L 和健康调查简表 6 维度(SF-6D)的心理测量性能。CFQ-R-8D 是一种新的、特定于疾病的、基于偏好的衡量标准。
本研究使用了来自三项年龄≥14 岁的囊性纤维化参与者试验的数据,这些参与者完成了 CFQ-R 和 EQ-5D-3L 或 SF-6D。分析基于与 CFQ-R 域评分的相关性评估收敛效度。基于预测的第一秒用力呼气量和肺部恶化的百分比来评估已知组有效性。反应性基于变化的相关性以及基于症状严重程度变化的变化敏感性进行评估。估计了效应大小和标准化反应均值。
CFQ-R-8D 效用和维度与大多数重叠的 CFQ-R 域评分高度相关(ρ>0.5);EQ-5D-3L 和 SF-6D 效用和维度在维度上具有中度(ρ>0.3)到高度相关,这些维度反映了类似的概念。所有措施均显示出已知组有效性的证据(P<0.05)。CFQ-R-8D 效用和维度以及 CFQ-R 的变化相关性较强,但 SF-6D 的相关性中等,而 EQ-5D-3L 的相关性大多较弱(ρ>0.1)。SF-6D 的平均变化最大,且效应大小最大,其次是 CFQ-R-8D,然后是 EQ-5D-3L。与 EQ-5D-3L(61-62%)相比,CFQ-R-8D 或 SF-6D 效用评分没有天花板效应(完全健康时的应答比例<9%)。在 EQ-5D-3L 被归类为完全健康的参与者中,CFQ-R-8D 捕捉到了 CF 特有的健康问题,特别是咳嗽、腹痛和呼吸困难。
CFQ-R-8D 反映了已知组之间的差异和随时间的变化,其心理测量性能的证据强于 EQ-5D-3L,与 SF-6D 的证据相似。此外,CFQ-R-8D 比 EQ-5D-3L 或 SF-6D 更能捕捉到更具体的疾病症状,这些症状是 CF 患者健康相关生活质量的重要决定因素。