Table_1_Investigating the Added Value of the EQ-5D-5L With Two Bolt-On Items in Patients With Hemophilia.DOCX

Objective: This study examined the impact of adding two condition-specific bolt-on items to the EQ-5D-5L and assessed their psychometric properties in patients with hemophilia.
Methods: The data were obtained from a nationwide cross-sectional online survey of patients with hemophilia in China. Self-reported and proxy-reported data were analyzed separately. Ceiling effect, informativity, and discriminatory power of the EQ-5D-5L with two bolt-on items, dignity (DG), and bleeding (BL), were examined. Spearman's rank correlation (rho) was used to assess the associations of the EQ-5D-5L and two bolt-on items with the Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) and SF-12. Multiple regression analysis was performed to evaluate the explained variance of the EQ-5D-5L and bolt-on items in predicting EQ-VAS scores.
Results: A total of 895 patients and 222 caregivers completed the questionnaire. The ceiling effect decreased from 1.9 to 0.6% and 5.9 to 0.9% when using the EQ-5D-5L and the EQ-5D-5L with two bolt-on items among participants with both self- and proxy-completed questionnaires. Both DG and BL were strongly correlated with Haem-A-QoL sum score [rho: DG = 0.64 (patient) vs. 0.66(proxy); BL = 0.49 (patient) vs. 0.31 (proxy)], SF-12 mental component [rho: DG = −0.36 (patient) vs. −0.41 (proxy); BL = −0.53 (patient) vs. −0.57(proxy)], and SF-12 physical component [rho: DG = −0.61 (patient) vs. −0.61 (proxy); BL = −0.35 (patient) vs. −0.39 (proxy)]. Known-group comparisons confirmed that the two bolt-on items had satisfactory discriminatory power. Multiple regression analysis indicated that adding two bolt-on items significantly increased the ability to predict EQ-VAS scores. The adjusted R2 increased by 8.2 and 8.8% for reports completed by the patients or patients' proxy respondents, respectively.
Conclusion: Adding the DG and BL bolt-on items can increase performance on the EQ-5D-5L in patients with hemophilia. A future valuation study will be carried out.

研究目的：本研究旨在探讨在EQ-5D-5L（EQ-5D-5L）量表中新增两个针对特定病症的附加条目后对其性能的影响，并评估该量表在血友病患者中的心理测量学特性。
研究方法：本研究数据来源于针对中国血友病患者开展的一项全国性横断面线上调查。研究分别对自评数据与代理报告数据进行独立分析。针对新增尊严（dignity, DG）与出血（bleeding, BL）两个附加条目的EQ-5D-5L量表，本研究对其天花板效应、信息性与区分能力进行了考察。采用斯皮尔曼秩相关系数（rho）评估EQ-5D-5L及两个附加条目分别与成人血友病生活质量问卷（Haem-A-QoL）、SF-12（SF-12）量表的关联情况。通过多元回归分析，评估EQ-5D-5L与附加条目在预测EQ-VAS（EQ-VAS）评分时所能解释的变异量。
研究结果：最终共有895名血友病患者与222名照护者完成了问卷调研。在同时完成自评与代理报告问卷的受试者中，单独使用EQ-5D-5L量表时的天花板效应从1.9%降至0.6%，联用两个附加条目后则从5.9%降至0.9%。尊严（DG）与出血（BL）两个条目均与Haem-A-QoL总分存在较强相关性[相关系数rho：患者自评组DG=0.64，代理报告组DG=0.66；患者自评组BL=0.49，代理报告组BL=0.31]，与SF-12精神健康维度得分亦存在显著相关[rho：患者自评组DG=-0.36，代理报告组DG=-0.41；患者自评组BL=-0.53，代理报告组BL=-0.57]，同时与SF-12躯体健康维度得分相关[rho：患者自评组DG=-0.61，代理报告组DG=-0.61；患者自评组BL=-0.35，代理报告组BL=-0.39]。已知组别比较结果证实，两个附加条目具备良好的区分能力。多元回归分析结果显示，新增两个附加条目可显著提升EQ-VAS评分的预测性能：患者自评组与代理报告组的校正R²分别提升8.2%与8.8%。
研究结论：本研究表明，在EQ-5D-5L量表中加入尊严（DG）与出血（BL）两个附加条目，可有效提升该量表在血友病患者中的应用表现。未来将开展针对该改良版量表的效用价值评估研究。