Cao Yiyin, Li Haofei, Cheng Ling Jie, King Madeleine T, Kemmler Georg, Cella David, Yu Hongjuan, Huang Weidong, Luo Nan
School of Health Management, Harbin Medical University, Harbin, 150081, China.
Saw Swee Hock School of Public Health, National University of Singapore, Singapore, Singapore.
Health Econ Rev. 2024 Oct 1;14(1):79. doi: 10.1186/s13561-024-00560-0.
To perform a comparison of the measurement properties of two cancer-specific Multi-Attribute Utility Instruments (MAUIs), EORTC QLU-C10D and FACT-8D, in Chinese patients with hematologic malignancies (HM).
We conducted a longitudinal study on patients with HM in China, using QLU-C10D and FACT-8D at baseline and follow-up (3-4 months from baseline). We assessed: (i) convergent validity using Spearman's rank correlation test (r) with EQ-5D-5L; (ii) clinical-groups validity by differentiating cancer stages, overall health assessment (OHA), Eastern Cancer Oncology Group (ECOG) performance status, and mental health status. We also examined clinical validity with effect size (ES) and relative efficiency (RE); (iii) responsiveness to changes in patient self-perception using receiver operating characteristics (ROC) curves and area under the curves (AUC); and (iv) agreement using intraclass correlation coefficients (ICC) and visualized with Bland-Altman plot.
Among the 308 patients with HM at baseline, 131 completed the follow-up survey. Agreement between the two measures was high (ICC = 0.76). Both measures were highly correlated with EQ-5D-5 L and significantly differentiated (p < 0.001) among groups categorized by cancer stage, OHA performance status, and mental health. ESs for QLU-C10D were numerically higher for cancer stage, OHA, and performance status (ES = 0.53-1.49), whereas ES was higher for FACT-8D and mental health status (ES = 1.35). Responsiveness was higher for QLU-C10D (AUC = 0.84) compared to FACT-8D (AUC = 0.78).
Both QLU-C10D and FACT-8D are valid cancer-specific MAUIs for evaluating patients with HM. However, scholars should consider their slight differences in focus when choosing between the two measures.
比较两种癌症特异性多属性效用工具（MAUIs），即欧洲癌症研究与治疗组织生活质量问卷核心模块-10维度（EORTC QLU-C10D）和癌症治疗功能评价系统-8维度（FACT-8D），在中国血液系统恶性肿瘤（HM）患者中的测量属性。
我们对中国的HM患者进行了一项纵向研究，在基线和随访时（基线后3 - 4个月）使用QLU-C10D和FACT-8D。我们评估了：（i）使用Spearman等级相关检验（r）与EQ-5D-5L评估收敛效度；（ii）通过区分癌症分期、总体健康评估（OHA）、东部肿瘤协作组（ECOG）体能状态和心理健康状态评估临床组效度。我们还使用效应量（ES）和相对效率（RE）检验临床效度；（iii）使用受试者工作特征（ROC）曲线和曲线下面积（AUC）评估对患者自我认知变化的反应性；以及（iv）使用组内相关系数（ICC）评估一致性，并通过Bland-Altman图进行可视化。
在基线时的308例HM患者中，131例完成了随访调查。两种测量方法之间的一致性较高（ICC = 0.76）。两种测量方法均与EQ-5D-5L高度相关，并且在按癌症分期、OHA表现状态和心理健康分类的组之间有显著差异（p < 0.001）。QLU-C10D在癌症分期、OHA和体能状态方面的效应量在数值上更高（ES = 0.53 - 1.49），而FACT-8D在心理健康状态方面的效应量更高（ES = 1.35）。与FACT-8D（AUC = 0.78）相比，QLU-C10D的反应性更高（AUC = 0.84）。
QLU-C10D和FACT-8D都是评估HM患者有效的癌症特异性MAUIs。然而，学者们在两种测量方法之间进行选择时应考虑它们在重点方面的细微差异。
