Wiethoff Isabell, Schoonvelde Stephan A C, de Boer Rudolf A, Evers Silvia M A A, Germans Tjeerd, Hirsch Alexander, Knackstedt Christian, Te Rijdt Wouter P, van Slegtenhorst Marjon A, Schinkel Arend F L, Zwetsloot Peter-Paul, Michels Michelle, Hiligsmann Mickael
Department of Health Services Research, Care and Public Health Research Institute (CAPHRI), Maastricht University, Maastricht, The Netherlands.
Department of Cardiology, Erasmus MC, Cardiovascular Institute, Thorax Center, Rotterdam, The Netherlands
Open Heart. 2025 May 27;12(1):e003143. doi: 10.1136/openhrt-2024-003143.
To assess the psychometric properties (content validity, reliability and construct validity) of generic and disease-specific health-related quality of life (HRQoL) instruments in patients with hypertrophic cardiomyopathy (HCM) and genotype-positive, phenotype-negative (G+/P-) individuals.
As part of the multicentre, observational AFFECT-HCM study, HRQoL was measured using the generic EuroQoL-5 Dimension-5 Level (EQ-5D-5L) questionnaire, the Visual Analogue Scale (EQ VAS) and the disease-specific Kansas City Cardiomyopathy Questionnaire (KCCQ). The study included G+/P- individuals and HCM patients. EQ-5D-5L profiles were translated into EQ-5D values (utilities) using the Dutch value set. All instruments were evaluated regarding their general characteristics and health dimensions (content validity). Reliability was assessed using internal consistency (Cronbach's alpha), response rate, floor/ceiling effects (percentage scoring highest/lowest), correlation and level of agreement between instruments (using Bland-Altman plots). Construct validity was assessed using the known-groups method to identify expected differences between relevant groups.
A total of 393 HCM patients and 78 G+/P- individuals were included in the psychometric assessment. Mean EQ-5D value in G+/P- individuals was 0.90 (81 EQ VAS, 93 KCCQ) and in HCM patients 0.84 (75 EQ VAS, 78 KCCQ). Ceiling effects were highest for EQ-5D values (51% in G+P; 32% in HCM), followed by the KCCQ (38% in G+P-; 12% in HCM) and the EQ VAS (8% in G+P-; 5% in HCM). KCCQ and EQ-5D values had the highest correlation (Spearman's ρ=0.77) and showed good overall agreement according to the Bland-Altman plots. In HCM, EQ-5D values showed a slightly biased pattern with EQ-5D values scoring higher than the KCCQ. The KCCQ discriminated more nuances between relevant groups.
Due to its simplicity and good overall agreement with the KCCQ-which showed slightly better discrimination-we propose from our data that the EQ-5D-5L is a suitable instrument for the HRQoL assessment in clinical practice in patients with HCM.
评估肥厚型心肌病（HCM）患者以及基因型阳性、表型阴性（G+/P-）个体中通用和疾病特异性健康相关生活质量（HRQoL）工具的心理测量特性（内容效度、信度和结构效度）。
作为多中心观察性AFFECT-HCM研究的一部分，使用通用的欧洲五维健康量表-5级（EQ-5D-5L）问卷、视觉模拟量表（EQ VAS）和疾病特异性堪萨斯城心肌病问卷（KCCQ）测量HRQoL。该研究纳入了G+/P-个体和HCM患者。使用荷兰值集将EQ-5D-5L概况转换为EQ-5D值（效用值）。对所有工具的一般特征和健康维度进行评估（内容效度）。使用内部一致性（Cronbach's α）、应答率、地板/天花板效应（得分最高/最低的百分比）、工具之间的相关性和一致性水平（使用Bland-Altman图）评估信度。使用已知组方法评估结构效度，以识别相关组之间的预期差异。
共有393例HCM患者和78例G+/P-个体纳入心理测量评估。G+/P-个体的平均EQ-5D值为0.90（EQ VAS为81，KCCQ为93），HCM患者为0.84（EQ VAS为75，KCCQ为78）。EQ-5D值的天花板效应最高（G+P-个体中为51%；HCM患者中为32%），其次是KCCQ（G+P-个体中为38%；HCM患者中为12%）和EQ VAS（G+P-个体中为8%；HCM患者中为5%）。KCCQ与EQ-5D值的相关性最高（Spearman's ρ = 0.77），根据Bland-Altman图显示总体一致性良好。在HCM中，EQ-5D值显示出略有偏差的模式，EQ-5D值得分高于KCCQ。KCCQ在相关组之间区分出更多细微差别。
由于其简单性以及与KCCQ总体一致性良好（KCCQ的区分度略好），我们从数据中提出EQ-5D-5L是HCM患者临床实践中HRQoL评估的合适工具。
