Seyringer Simone, Pilz Micha J, Bottomley Andrew, King Madeleine T, Norman Richard, Gamper Eva M
Department for Psychiatry, Psychotherapy and Psychosomatic Medicine,University Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
Department of Social Psychology, Personnel Development and Adult Education, Johannes Kepler University Linz, Linz, Austria.
Eur J Health Econ. 2024 Nov 20. doi: 10.1007/s10198-024-01729-4.
Many health economic evaluations rely on the validity of the utility measurement for health-related quality of life (HRQoL). While generic utility measures perform well in HRQoL assessments of many diseases and patient populations, appropriateness for cancer-specific disease burdens needs attention and condition-specific measures could be a viable option. This study assessed the clinical validity of the cancer-specific EORTC QLU-C10D, a utility scoring algorithm for the EORTC QLQ-C30, in patients with glioblastoma. We expect the EORTC QLU-C10D to be sensitive and responsive in glioblastoma patients. Furthermore, we compared its statistical efficiency with the generic utility measure EQ-5D-3L.
We used data from a multi-center randomized controlled trial (NCT00689221) with patients from 146 study sites in 25 countries. Both, the QLQ-C30 and the EQ-5D-3L, had been administered at seven assessment points together. Utilities of both measures were calculated for four country value set (Australia, Canada, UK, USA). Ceiling effects, agreement (Bland-Altman plots (BA), intra-class correlation (ICC)), were calculated to analyze construct validity. Sensitivity to known-groups (performance status; global health) and responsiveness to changes (progressive vs. non-progressive; stable vs. improved or deteriorated HRQoL) were investigated for clinical validity. Relative Efficiency (RE) was calculated to compare statistical efficiency of both utility measures.
435 patients were included at baseline and six subsequent time points (median timeframe 497 days). QLU-C10D country value set showed negligible ceiling effects (< 6.7%) and high agreement with EQ-5D-3L (ICC > 0.750). BA indicated that differences between both utility measures increased with deteriorating health states. While the QLU-C10D was more sensitive to global health groups (RE > 1.2), the EQ-5D-3L was more sensitive to performance status groups (RE < 0.7) than the other utility measure. Statistical efficiency to detect differences between change groups and within HRQoL deterioration group (RE > 1.4) favored QLU-C10D in 18 of 24 (75%) and 20 of 24 (83%) comparisons with the EQ-5D-3L respectively. Responsiveness to overall HRQoL change (RE > 3.4) also favored the QLU-C10D.
Our results indicate that the QLU-C10D is a valid utility measure to assess HRQoL in patients with glioblastoma. This facilitates the investigation of HRQoL profiles and utilities in this patient population by administering a single questionnaire, the EORTC QLQ-C30. Efficiency analyses point to higher statistical power of the QLU-C10D compared to the EQ-5D-3L.
许多卫生经济评估依赖于健康相关生活质量（HRQoL）效用测量的有效性。虽然通用效用测量在许多疾病和患者群体的HRQoL评估中表现良好，但针对癌症特定疾病负担的适用性需要关注，特定疾病的测量方法可能是一个可行的选择。本研究评估了癌症特异性的欧洲癌症研究与治疗组织（EORTC）QLU-C10D（一种针对EORTC QLQ-C30的效用评分算法）在胶质母细胞瘤患者中的临床有效性。我们期望EORTC QLU-C10D在胶质母细胞瘤患者中具有敏感性和反应性。此外，我们将其统计效率与通用效用测量EQ-5D-3L进行了比较。
我们使用了一项多中心随机对照试验（NCT00689221）的数据，该试验的患者来自25个国家的146个研究地点。QLQ-C30和EQ-5D-3L均在七个评估点同时进行了测量。针对四个国家的值集（澳大利亚、加拿大、英国、美国）计算了两种测量方法的效用。计算了天花板效应、一致性（布兰德-奥特曼图（BA）、组内相关系数（ICC））以分析结构效度。研究了对已知组（性能状态；总体健康）的敏感性以及对变化的反应性（进展性与非进展性；稳定与改善或恶化的HRQoL）以评估临床有效性。计算了相对效率（RE）以比较两种效用测量方法的统计效率。
435名患者在基线及随后的六个时间点（中位时间范围为497天）被纳入研究。QLU-C10D国家值集显示出可忽略不计的天花板效应（<6.7%），并且与EQ-5D-3L具有高度一致性（ICC>0.750）。BA表明，两种效用测量方法之间的差异随着健康状态的恶化而增加。虽然QLU-C10D对总体健康组更敏感（RE>1.2），但与另一种效用测量方法相比，EQ-5D-3L对性能状态组更敏感（RE<0.7）。在与EQ-5D-3L的24次比较中，检测变化组之间以及HRQoL恶化组内差异的统计效率（RE>1.4）分别在18次（75%）和20次（83%）比较中有利于QLU-C10D。对总体HRQoL变化的反应性（RE>3.4）也有利于QLU-C10D。
我们的结果表明，QLU-C10D是评估胶质母细胞瘤患者HRQoL的有效效用测量方法。通过使用单一问卷EORTC QLQ-C30，这有助于对该患者群体的HRQoL概况和效用进行研究。效率分析表明，与EQ-5D-3L相比，QLU-C10D具有更高的统计效力。
