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开发映射算法,以根据强直性脊柱炎患者的巴斯强直性脊柱炎疾病活动指数和巴斯强直性脊柱炎功能指数预测EQ-5D健康效用值。

Developing mapping algorithms to predict EQ-5D health utility values from Bath Ankylosing Spondylitis Disease Activity Index and Bath Ankylosing Spondylitis Functional Index among patients with Ankylosing Spondylitis.

作者信息

Yu Lingjia, Yang Huizhi, Lu Liyong, Fang Yingying, Zhang Xianyu, Li Shunping, Li Chaofan

机构信息

Nursing Department, Rheumatology department, Qilu hospital of Shandong University, Jinan, 250012, China.

Shunyi District Center for Disease Control and Prevention, Beijing, 101300, China.

出版信息

Health Qual Life Outcomes. 2024 Aug 7;22(1):61. doi: 10.1186/s12955-024-02276-5.

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.

CONCLUSION

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)测量方法,如EQ-5D或SF-6D,对健康经济评估至关重要。然而,它们很少被纳入强直性脊柱炎(AS)的临床试验中。本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

2019年12月至2020年10月期间,在中国山东省最大的三级医院招募AS患者。采用方便抽样法根据以下标准选择患者:(1)根据纽约标准诊断为AS;(2)年龄在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。共有243例患者完成了面对面问卷调查,排除5例关键变量存在缺失值的病例。采用直接法中的普通最小二乘法、截尾最小绝对偏差法、Tobit法、调整有限因变量混合模型和β混合模型(BM)以及响应法中的有序logit模型和多项logit(Mlogit)模型来开发映射算法。采用平均绝对误差、均方根误差、Spearman相关系数和一致性相关系数来评估预测性能。

结果

238例AS患者的平均年龄为35.19(标准差=9.59)岁,大多数(74.47%)为男性。观察到的EQ-5D-3L和EQ-5D-5L健康效用值分别为0.88(标准差=0.12)和0.74(标准差=0.27)。与EQ-5D-3L相比,EQ-5D-5L与BASDAI和BASFI的概念重叠度更高。Mlogit是EQ-5D-3L表现最佳的模型,BM在预测EQ-5D-5L方面比其他直接和间接映射模型表现更好。

结论

本研究表明,在中国AS患者中,应选择EQ-5D-5L而非EQ-5D-3L作为HRQoL的目标结局指标,并且BM映射算法可用于根据BASDAI和BASFI预测EQ-5D-5L值以进行健康经济评估。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/efdf/11304938/d1f16f04cfed/12955_2024_2276_Fig1_HTML.jpg

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