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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
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| 篇名： | 全球血友病患者健康效用值的系统综述与Meta分析 |
| TITLE： | Systematic review and meta-analysis of health state utility for global patients with hemophilia |
| 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、theCochraneLibrary、Scopus和WebofScience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Stata15.1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. |
| 期刊： | 2023年第34卷第24期 |
| 作者： | 冯俊超；李顺平；房云海；史钊 |
| AUTHORS： | FENG Junchao，LI Shunping，FANG Yunhai，SHI Zhao |
| 关键字： | 罕见病；血友病；健康效用值；系统评价；Meta分析 |
| KEYWORDS： | rare diseases； hemophilia； health state utility； |
| 阅读数： | 44 次 |
| 本月下载数： | 4 次 |
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