Pilz Micha J, Seyringer Simone, Nerich Virginie, King Madeleine T, Norman Richard, Gamper Eva M
Health Outcomes Research Unit, University Hospital of Psychiatry II, Medial University of Innsbruck, 6020, Innsbruck, Austria.
Department of Nuclear Medicine, Medical University of Innsbruck, 6020, Innsbruck, Austria.
Pharmacoeconomics. 2025 Apr 27. doi: 10.1007/s40273-025-01501-3.
The Quality of Life Utility - Core 10 Dimensions (QLU-C10D) is a disease-specific preference-based measure (PBM) designed to obtain health state utility values from patients with cancer. Previously, satisfactory psychometric properties were established from retrospective trial analyses using clinical anchors. This study aimed to validate the QLU-C10D against two generic PBMs in a prospective sample of Austrian and French patients with cancer using patient-reported anchors.
Patients completed the European Organisation of Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 (QLQ-C30), EQ-5D-5L and Short Form 36 (SF-36) at study baseline (any time during anti-cancer treatment) plus a follow-up assessment 3-6 months later. Sociodemographic and clinical characteristics were assessed. QLU-C10D and SF-6Dv2 utilities were calculated from QLQ-C30 and SF-36 data, respectively. German, French and UK value sets were applied for all three PBMs. Floor and ceiling effects were assessed. Known-group validity (independent t-test) and responsiveness (paired t-tests) were assessed respectively by ability to detect health status differences and changes over time according to patient-rated overall quality of life/health perception assessed by the QLQ-C30 Global Health Status scale, the EQ-5D-5L VAS and the SF-36 General Health scale.
A total of 465 patients were included in the analysis. QLU-C10D index scores (intra-class correlation) and domains (Pearson) were correlated with EQ-5D-5L and Short-Form Six Dimensions (SF-6Dv2) conceptual counterparts. Correlation coefficients for the index scores of QLU-C10D and the generic PBMs ranged from 0.63 to 0.81. The QLU-C10D detected statistically significant differences between groups at baseline in 100% of tests performed (n = 27). For changes over time, QLU-C10D detected expected effects in 68% of cases (n = 29). In comparison with the generic PBMs, QLU-C10D detected differences and changes with a higher statistical efficiency in 76% of cases (77 of 102).
The QLU-C10D is a fit-for-purpose ready-to-use PBM to estimate health state utilities of patients with cancer. This study adds to evidence that QLU-C10D has appropriate psychometric properties and appears to have higher statistical efficiency than generic PBMs in cancer.
生活质量效用 - 核心10维度（QLU - C10D）是一种针对特定疾病的基于偏好的测量方法（PBM），旨在从癌症患者中获取健康状态效用值。此前，通过使用临床锚点的回顾性试验分析确定了令人满意的心理测量特性。本研究旨在使用患者报告的锚点，在前瞻性的奥地利和法国癌症患者样本中，针对两种通用PBM对QLU - C10D进行验证。
患者在研究基线（抗癌治疗期间的任何时间）完成欧洲癌症研究与治疗组织（EORTC）生活质量问卷 - 核心30（QLQ - C30）、EQ - 5D - 5L和简明健康调查问卷36（SF - 36），并在3 - 6个月后进行随访评估。评估社会人口统计学和临床特征。分别从QLQ - C30和SF - 36数据计算QLU - C10D和SF - 6Dv2效用值。对所有三种PBM应用德国、法国和英国的值集。评估地板效应和天花板效应。根据QLQ - C30全球健康状况量表、EQ - 5D - 5L视觉模拟量表（VAS）和SF - 36总体健康量表评估的患者自评总体生活质量/健康感知，分别通过检测健康状态差异和随时间变化的能力来评估已知组效度（独立t检验）和反应性（配对t检验）。
共有465名患者纳入分析。QLU - C10D指数得分（组内相关性）和领域（Pearson相关性）与EQ - 5D - 5L和简明形式六维度（SF - 6Dv2）的概念对应物相关。QLU - C10D指数得分与通用PBM的相关系数范围为0.63至0.81。在进行的所有测试（n = 27）中，QLU - C10D在基线时检测到组间有统计学显著差异的比例为100%。对于随时间的变化，QLU - C10D在68%的病例（n = 29）中检测到预期效果。与通用PBM相比，QLU - C10D在76%的病例（102例中的77例）中检测差异和变化时具有更高的统计效率。
QLU - C10D是一种适用于估计癌症患者健康状态效用的即用型PBM。本研究进一步证明QLU - C10D具有适当的心理测量特性，并且在癌症方面似乎比通用PBM具有更高的统计效率。
