Interpreting important health-related quality of life change using the Haem-A-QoL
Interpreting important health-related quality of life change using the Haem-A-QoL
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DOI：
10.1111/hae.12642
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发表时间：
2015-09-01
期刊：
影响因子：
3.9
通讯作者：
von Mackensen, S.
中科院分区：
文献类型：
--
作者：
Wyrwich, K. W.;Krishnan, S.;von Mackensen, S.
成人血友病生活质量问卷（Haem-A-QoL）测量血友病成人的健康相关生活质量（HRQoL）;然而，尚未适当研究用于识别经历HRQoL获益的个体的变化评分阈值。本分析的目的是使用基于锚点和分布的方法，为反映关键损伤的两个Haem-A-QoL领域（“身体健康”和“运动与休闲”）和Haem-A-QoL“总分”推导适当的HRQoL应答者定义（RD）。在本分析中，使用了来自A-LONG和B-LONG（分别在血友病A中进行rFVIIIFc和在血友病B中进行rFIXFc的两项III期临床研究）中成人的数据。基于锚点的方法确定了与基线和6个月之间EQ-5D项目改善相对应的Haem-A-QoL变化;基于分布的方法检查了基线时半标准差和测量标准误的幅度。通过三角测量，得出最合适的RD。在具有基线Haem-A-QoL评分的133例A-LONG和73例B-LONG受试者中，分别有67例和51例受试者在基线和6个月随访时完成了Haem-A-QoL问卷。基于锚点和分布的估计值与观察到的Haem-A-QoL变化评分的三角测量确定了“身体健康”和“运动与休闲”领域降低10分，“总分”降低7分，作为最能指示HRQoL获益的RD阈值。两个关键Haem-A-QoL领域和“总分”的经验推导的RD是识别HRQoL显著改善的受试者的合理和实用阈值，并提供了可用于进一步分析和解释血友病临床试验数据的HRQoL RD。
The Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) measures health-related quality of life (HRQoL) in adults with haemophilia; however, change score thresholds for identifying individuals experiencing a HRQoL benefit have not been appropriately investigated. The objective of this analysis was to derive appropriate HRQoL responder definitions (RDs) for two Haem-A-QoL domains that reflect key impairments, 'Physical Health' and 'Sports & Leisure,' and the Haem-A-QoL 'Total Score' using anchor-and distribution-based methods. In this analysis, data from adults in A-LONG and B-LONG, two Phase 3 clinical studies of rFVIIIFc in haemophilia A and rFIXFc in haemophilia B, respectively, were used. The anchor-based approach identified Haem-A-QoL changes corresponding to EQ-5D item improvements between baseline and 6 months; the distribution-based methods examined the magnitude at baseline of one-half standard deviation and the standard error of measurement. Through triangulation, the most appropriate RDs were derived. Of the 133 A-LONG and 73 B-LONG subjects with baseline Haem-A-QoL scores, 67 and 51 subjects, respectively, completed the Haem-A-QoL questionnaire at both baseline and 6 months follow-up. Triangulation of anchor-and distribution-based estimates with the observed Haem-A-QoL change scores identified a 10-point reduction in the 'Physical Health' and 'Sports & Leisure' domains, and a 7-point reduction in 'Total Score' as the RD thresholds most indicative of HRQoL benefit. These empirically derived RDs for two key Haem-A-QoL domains and 'Total Score' are reasonable and practical thresholds for identifying subjects with notable improvements in HRQoL, and provides HRQoL RDs that can be used for further analysis and interpretation of data from haemophilia clinical trials.