全球血友病患者健康效用值的系统综述与Meta分析

冯俊超; 李顺平; 房云海; 史钊

doi:10.6039/j.issn.1001-0408.2023.24.10

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全球血友病患者健康效用值的系统综述与Meta分析

药物经济学 | 更新时间：2023-12-22

- 全球血友病患者健康效用值的系统综述与Meta分析
- Systematic review and meta-analysis of health state utility for global patients with hemophilia
- 中国药房 2023年34卷第24期页码：3012-3019
- 作者机构：
  
  1.山东大学齐鲁医学院公共卫生学院卫生管理与政策研究中心，济南;250012
  2.国家卫生健康委员会卫生经济与政策研究重点实验室（山东大学），济南　250012
  3.山东大学健康偏好研究中心，济南　250012
  4.山东省血液中心山东省血友病诊疗中心，济南　250014
- 作者简介：
  
  博士研究生。研究方向：罕见病诊疗保障与健康效用值测量。E-mail：202221015@mail.sdu.edu.cn
  教授，博士生导师。研究方向：健康偏好与健康结果测量、罕见病诊疗与保障、卫生技术评估。E-mail：lishunping@sdu.edu.cn
- 基金信息：
  
  国家自然科学基金面上项目(72174110)
- DOI：10.6039/j.issn.1001-0408.2023.24.10
  中图分类号： R956
- 纸质出版日期：2023-12-30，
  
  收稿日期：2023-07-24，
  
  修回日期：2023-11-23，
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冯俊超,李顺平,房云海等.全球血友病患者健康效用值的系统综述与Meta分析 ^Δ[J].中国药房,2023,34(24):3012-3019.

FENG Junchao,LI Shunping,FANG Yunhai,et al.Systematic review and meta-analysis of health state utility for global patients with hemophilia[J].ZHONGGUO YAOFANG,2023,34(24):3012-3019.
冯俊超,李顺平,房云海等.全球血友病患者健康效用值的系统综述与Meta分析 ^Δ[J].中国药房,2023,34(24):3012-3019. DOI： 10.6039/j.issn.1001-0408.2023.24.10.

FENG Junchao,LI Shunping,FANG Yunhai,et al.Systematic review and meta-analysis of health state utility for global patients with hemophilia[J].ZHONGGUO YAOFANG,2023,34(24):3012-3019. DOI： 10.6039/j.issn.1001-0408.2023.24.10.

摘要

目的

系统梳理与评价全球血友病患者的健康效用值，为今后开展相关药物经济学评价和卫生技术评估提供基础数据支持。

方法

计算机检索中国知网、万方数据、维普网、中国生物医学文献数据库、PubMed、Embase、the Cochrane Library、Scopus和Web of Science等数据库，收集血友病患者健康效用值测量的相关文献，检索时限均为建库至2023年2月。筛选文献、提取资料、评价文献质量后，采用Stata 15.1软件对健康效用值进行Meta分析。

结果

最终纳入38篇文献，纳入研究的健康效用值最高和最低值分别为0.90和0.46，使用欧洲五维健康量表（EQ-5D）系列量表测量的研究最多（73.7%）。Meta分析结果显示，全球血友病患者的健康效用值为0.69，95%置信区间为0.65～0.74，轻、中、重型患者健康效用值分别为0.79、0.70、0.64；伴有抑制物患者的健康效用值（0.64）低于不伴有抑制物的患者（0.69）；中国患者群体健康效用值为0.55，高于伊朗患者（0.46），但低于其他发达国家。

结论

不同国家/地区研究结果具有一定的异质性，发达国家血友病患者健康效用值高于发展中国家；随着血友病严重程度的加重，健康效用值下降趋势明显。

Abstract

OBJECTIVE

To systematically sort out and evaluate the health state utility of hemophiliac patients， and to provide reliable parameters for conducting pharmacoeconomic evaluation and health technology assessment.

METHODS

Retrieved from CNKI， Wanfang data， VIP， CBM， PubMed， Embase， the Cochrane Library， Scopus and Web of Science databases， relevant literature about the measurement of health state utility in hemophiliac patients was collected from the inception to February 2023. After screening literature， extracting data and evaluating the quality of literature， meta-analysis was performed for health state utility with Stata 15.1 software.

RESULTS

Thirty-eight papers were finally included， with the highest and lowest health utility values of 0.90 and 0.46， respectively. Those studies mostly adopted the EuroQol Five Dimensions Questionnaire （EQ-5D）（73.7%）. Results of meta-analysis showed that health state utility of global hemophiliac patients was 0.69，95% confidence interval was 0.65-0.74； those of patients with mild， moderate and severe hemophilia were 0.79， 0.70， and 0.64， respectively； health state utility for patients with inhibitors （0.64） was lower than that of patients without inhibitors （0.69）； health state utility for the Chinese patient population was 0.55， which was higher than that of Iranian patients （0.46）， but lower than those of other developed countries.

CONCLUSIONS

There is some heterogeneity in the results of the studies across countries/regions， with higher health state utility in developed countries than in developing countries. As the severity of hemophilia increases， the trend of decreasing health state utility is obvious.

关键词

罕见病血友病健康效用值系统评价Meta分析

Keywords

hemophiliahealth state utilitysystematic evaluationmeta-analysis

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