PDF(Pubmed)
Abstract：
BACKGROUND: Preference-based measures of health-related quality of life (HRQoL), such as the EQ-5D or the SF-6D, are essential for health economic evaluation. However, they are rarely included in clinical trials of ankylosing spondylitis (AS). This study aims to develop mapping algorithms to predict EQ-5D-3L and EQ-5D-5L health utility scores from the Bath Ankylosing Disease Activity Index (BASDAI) and the Bath Ankylosing Spondylitis Functional Index (BASFI).
METHODS: Patients with AS were recruited from the largest tertiary hospital in Shandong province, China, between December 2019 and October 2020. Patients were selected by convenience sampling method according to the following criteria: (1) diagnosed with AS according to the New York criteria; (2) aged 18 years and above; and (3) without mental disorders; (4) able to understand the questionnaires; (5) without serious complications. There were 243 patients who completed the face-to-face questionnaire survey, and 5 cases with missing values in key variables were excluded. Ordinary least squares, censored least absolute deviations, Tobit, adjusted limited dependent variable mixture model and beta-mixture model (BM) in the direct approach and ordered logit and multinomial logit (Mlogit) model in the response approach were used to develop mapping algorithms. Mean absolute error, root mean square error, Spearman\'s correlation coefficient and concordance correlation coefficient were used to access predictive performance.
RESULTS: The 238 patients with AS had a mean age of 35.19 (SD = 9.59) years, and the majority (74.47%) were male. The observed EQ-5D-3L and EQ-5D-5L health utility values were 0.88 (SD = 0.12) and 0.74 (SD = 0.27), respectively. The EQ-5D-5L had higher conceptual overlap with the BASDAI and BASFI than the EQ-5D-3L did. The Mlogit was the best-performing model for the EQ-5D-3L, and the BM showed better performance in predicting EQ-5D-5L than other direct and indirect mapping models did.
CONCLUSIONS: This study demonstrates that the EQ-5D-5L, rather than EQ-5D-3L, should be selected as the target outcome measure of HRQoL in patients with AS in China, and the BM mapping algorithm could be used to predict EQ-5D-5L values from BASDAI and BASFI for health economic evaluation.
METHODS: Patients with AS were recruited from the largest tertiary hospital in Shandong province, China, between December 2019 and October 2020. Patients were selected by convenience sampling method according to the following criteria: (1) diagnosed with AS according to the New York criteria; (2) aged 18 years and above; and (3) without mental disorders; (4) able to understand the questionnaires; (5) without serious complications. There were 243 patients who completed the face-to-face questionnaire survey, and 5 cases with missing values in key variables were excluded. Ordinary least squares, censored least absolute deviations, Tobit, adjusted limited dependent variable mixture model and beta-mixture model (BM) in the direct approach and ordered logit and multinomial logit (Mlogit) model in the response approach were used to develop mapping algorithms. Mean absolute error, root mean square error, Spearman\'s correlation coefficient and concordance correlation coefficient were used to access predictive performance.
RESULTS: The 238 patients with AS had a mean age of 35.19 (SD = 9.59) years, and the majority (74.47%) were male. The observed EQ-5D-3L and EQ-5D-5L health utility values were 0.88 (SD = 0.12) and 0.74 (SD = 0.27), respectively. The EQ-5D-5L had higher conceptual overlap with the BASDAI and BASFI than the EQ-5D-3L did. The Mlogit was the best-performing model for the EQ-5D-3L, and the BM showed better performance in predicting EQ-5D-5L than other direct and indirect mapping models did.
CONCLUSIONS: This study demonstrates that the EQ-5D-5L, rather than EQ-5D-3L, should be selected as the target outcome measure of HRQoL in patients with AS in China, and the BM mapping algorithm could be used to predict EQ-5D-5L values from BASDAI and BASFI for health economic evaluation.
摘要：
背景：基于偏好的健康相关生活质量（HRQoL）测量，例如EQ-5D或SF-6D，对卫生经济评价至关重要。然而,它们很少被纳入强直性脊柱炎(AS)的临床试验.这项研究旨在开发映射算法，以根据巴斯强直性疾病活动指数（BASDAI）和巴斯强直性脊柱炎功能指数（BASFI）预测EQ-5D-3L和EQ-5D-5L健康效用得分。
方法：从山东省最大的三级医院招募AS患者，中国,2019年12月至2020年10月。采用方便抽样法根据以下标准选择患者：（1）符合纽约标准诊断为AS；（2）年龄18岁及以上；（3）无精神障碍；（4）能够理解问卷；（5）无严重并发症。有243名患者完成了面对面的问卷调查，排除5例关键变量缺失值的病例。普通最小二乘,审查最小绝对偏差，Tobit,直接方法中的调整有限因变量混合模型和β混合模型（BM）以及响应方法中的有序logit和多项式logit（Mlogit）模型用于开发映射算法。平均绝对误差,均方根误差，使用Spearman相关系数和一致性相关系数来获得预测性能。
结果：238例AS患者的平均年龄为35.19（SD=9.59）岁，男性占多数(74.47%)。观察到的EQ-5D-3L和EQ-5D-5L健康效用值分别为0.88（SD=0.12）和0.74（SD=0.27），分别。EQ-5D-5L与BASDAI和BASFI的概念重叠高于EQ-5D-3L。Mlogit是EQ-5D-3L性能最好的型号，BM在预测EQ-5D-5L方面表现出比其他直接和间接映射模型更好的性能。
结论：这项研究表明，EQ-5D-5L，而不是EQ-5D-3L，应选择作为中国AS患者HRQoL的目标结局指标,BM映射算法可用于预测BASDAI和BASFI的EQ-5D-5L值，以进行卫生经济学评价。
方法：从山东省最大的三级医院招募AS患者，中国,2019年12月至2020年10月。采用方便抽样法根据以下标准选择患者：（1）符合纽约标准诊断为AS；（2）年龄18岁及以上；（3）无精神障碍；（4）能够理解问卷；（5）无严重并发症。有243名患者完成了面对面的问卷调查，排除5例关键变量缺失值的病例。普通最小二乘,审查最小绝对偏差，Tobit,直接方法中的调整有限因变量混合模型和β混合模型（BM）以及响应方法中的有序logit和多项式logit（Mlogit）模型用于开发映射算法。平均绝对误差,均方根误差，使用Spearman相关系数和一致性相关系数来获得预测性能。
结果：238例AS患者的平均年龄为35.19（SD=9.59）岁，男性占多数(74.47%)。观察到的EQ-5D-3L和EQ-5D-5L健康效用值分别为0.88（SD=0.12）和0.74（SD=0.27），分别。EQ-5D-5L与BASDAI和BASFI的概念重叠高于EQ-5D-3L。Mlogit是EQ-5D-3L性能最好的型号，BM在预测EQ-5D-5L方面表现出比其他直接和间接映射模型更好的性能。
结论：这项研究表明，EQ-5D-5L，而不是EQ-5D-3L，应选择作为中国AS患者HRQoL的目标结局指标,BM映射算法可用于预测BASDAI和BASFI的EQ-5D-5L值，以进行卫生经济学评价。
