Systematic review and meta-analysis of health state utility for global patients with hemophilia

FENG Junchao; LI Shunping; FANG Yunhai; SHI Zhao

doi:10.6039/j.issn.1001-0408.2023.24.10

您当前的位置：

首页 >

文章列表页 >

Systematic review and meta-analysis of health state utility for global patients with hemophilia

更新时间：2023-12-22

- Systematic review and meta-analysis of health state utility for global patients with hemophilia
- ZHONGGUO YAOFANG Vol. 34, Issue 24, Pages: 3012-3019(2023)
- 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：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2023.24.10
  CLC： R956
- Published：30 December 2023，
  
  Received：24 July 2023，
  
  Revised：23 November 2023，
扫描看全文
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

references

中华医学会血液学分会血栓与止血学组，中国血友病协作组. 血友病治疗中国指南：2020年版[J]. 中华血液学杂志，2020，41（4）：265-271.

Thrombosis and Hemostasis Group，Chinese Society of Hematology，Chinese Medical Association/Hemophilia Treatment Center Collaborative Network of China. Chinese guidelines on the treatment of hemophilia：version 2020[J]. Chin J Hematol，2020，41（4）：265-271.

薛峰，戴菁，陈丽霞，等. 中国血友病诊治报告2023[J]. 诊断学理论与实践，2023，22（2）：89-115.

XUE F，DAI J，CHEN L X，et al. Report on diagnosis and treatment of hemophilia in China 2023[J]. J Diagn Concepts Pract，2023，22（2）：89-115.

李常印，殷玉如. 罕见病医疗保障机制研究：以血友病为例[J]. 中国医疗保险，2023（7）：104-110.

LI C Y，YIN Y R. Study on medical security mechanism of rare diseases：a case study of hemophilia[J]. China Health Insur，2023（7）：104-110.

刘国恩. 中国药物经济学评价指南2020[M]. 北京：中国市场出版社，2020：43-47.

LIU G E. China guidelines for pharmacoeconomic evaluations 2020[M]. Beijing：China Market Press，2020：43-47.

TORRANCE G W. Utility approach to measuring health-related quality of life[J]. J Chronic Dis，1987，40（6）：593-603.

张瑶，马爱霞. 重组人凝血因子Ⅷ的小剂量预防方案对比按需治疗方案用于中国A型血友病患者的成本-效用分析[J]. 中国药物经济学，2021，16（7）：62-66.

ZHANG Y，MA A X. Cost-utility analysis of low-dose prophylaxis vs on-demand treatment of recombinant human coagulation factor Ⅷ for Chinese patients with type A hemophilia[J]. China J Pharm Econ，2021，16（7）：62-66.

曾宪涛，刘慧，陈曦，等. Meta分析系列之四：观察性研究的质量评价工具[J]. 中国循证心血管医学杂志，2012，4（4）：297-299.

ZENG X T，LIU H，CHEN X，et al. Meta-analysis series Ⅳ：quality evaluation tools for observational research[J]. Chin J Evid Based Cardiovasc Med，2012，4（4）：297-299.

张天嵩，董圣杰，周支瑞. 高级Meta分析方法：基于Stata实现[M]. 上海：复旦大学出版社，2015：112-128.

ZHANG T S，DONG S J，ZHOU Z R. Advanced meta-analysis in Stata[M]. Shanghai：Fudan University Press，2015：112-128.

DJULBEGOVIC B，GOLDSMITH G，VAUGHN D，et al. Comparison of the quality of life between HIV-positive haemophilia patients and HIV-negative haemophilia patients[J]. Haemophilia，1996，2（3）：166-172.

MINERS A H，SABIN C A，TOLLEY K H，et al. Asses-sing health-related quality-of-life in individuals with haemophilia[J]. Haemophilia，1999，5（6）：378-385.

BARR R D，SALEH M，FURLONG W，et al. Health status and health-related quality of life associated with hemophilia[J]. Am J Hematol，2002，71（3）：152-160.

NARAINE V S，RISEBROUGH N A，OH P，et al. Health-related quality-of-life treatments for severe haemophilia：utility measurements using the Standard Gamble technique[J]. Haemophilia，2002，8（2）：112-120.

GRINGERI A，MANTOVANI L G，SCALONE L，et al. Cost of care and quality of life for patients with hemophilia complicated by inhibitors：the COCIS Study Group[J]. Blood，2003，102（7）：2358-2363.

WASSERMAN J，ADAY L A，BEGLEY C E，et al. Measuring health state preferences for hemophilia：development of a disease-specific utility instrument[J]. Haemophilia，2005，11（1）：49-57.

HOOTS W K，EBBESEN L S，KONKLE B A，et al. Se-condary prophylaxis with recombinant activated factor Ⅶ improves health-related quality of life of haemophilia patients with inhibitors[J]. Haemophilia，2008，14（3）：466-475.

FORSYTH A L，WITKOP M，LAMBING A，et al. Associations of quality of life，pain，and self-reported arthritis with age，employment，bleed rate，and utilization of hemophilia treatment center and health care provider services：results in adults with hemophilia in the HERO study[J]. Patient Prefer Adherence，2015，9：1549-1560.

CAVAZZA M，KODRA Y，ARMENI P，et al. Social/economic costs and quality of life in patients with haemophilia in Europe[J]. Eur J Health Econ，2016，17（Suppl 1）：53-65.

FISCHER K，KLEIJN P D，NEGRIER C，et al. The association of haemophilic arthropathy with health-related quality of life：a post hoc analysis[J]. Haemophilia，2016，22（6）：833-840.

OSOOLI M，STEEN CARLSSON K，BAGHAEI F，et al. The association between health utility and joint status among people with severe haemophilia A：findings from the KAPPA register[J]. Haemophilia，2017，23（3）：e180-e187.

SOUCIE J M，GROSSE S D，SIDDIQI A E，et al. The effects of joint disease，inhibitors and other complications on health-related quality of life among males with severe haemophilia A in the United States[J]. Haemophilia，2017，23（4）：e287-e293.

WITKOP M，NEFF A，BUCKNER T W，et al. Self-reported prevalence，description and management of pain in adults with haemophilia：methods，demographics and results from the pain，functional impairment，and quality of life（P-FiQ）study[J]. Haemophilia，2017，23（4）：556-565.

BATT K，BOGGIO L，NEFF A，et al. Patient-reported outcomes and joint status across subgroups of US adults with hemophilia with varying characteristics：results from the Pain，Functional Impairment，and Quality of Life（P-FiQ）study[J]. Eur J Haematol，2018，100（Suppl 1）：14-24.

BOOTH J，OLADAPO A，WALSH S，et al. Real-world comparative analysis of bleeding complications and health-related quality of life in patients with haemophilia A and haemophilia B[J]. Haemophilia，2018，24（5）：e322-e327.

KEMPTON C L，RECHT M，NEFF A，et al. Impact of pain and functional impairment in US adults with haemophilia：patient-reported outcomes and musculoskeletal evaluation in the pain，functional impairment and quality of life（P-FiQ）study[J]. Haemophilia，2018，24（2）：261-270.

KOEBERLEIN-NEU J，RUNKEL B，HILBERG T. Cost-utility of a six-month programmed sports therapy（PST）in patients with haemophilia[J]. Haemophilia，2018，24（3）：385-394.

O’HARA J，WALSH S，CAMP C，et al. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life[J]. Health Qual Life Outcomes，2018，16（1）：84.

CARROLL L，BENSON G，LAMBERT J，et al. Real-world utilities and health-related quality-of-life data in hemophilia patients in France and the United Kingdom[J]. Patient Prefer Adherence，2019，13：941-957.

DAVARI M，GHARIBNASERI Z，RAVANBOD R，et al. Health status and quality of life in patients with severe hemophilia A：a cross-sectional survey[J]. Hematol Rep，2019，11（2）：7894.

HOXER C S，ZAK M，BENMEDJAHED K，et al. Utility valuation of health states for haemophilia and related complications in Europe and in the United States[J]. Haemophilia，2019，25（1）：92-100.

MAHLANGU J，OLDENBURG J，CALLAGHAN M U，et al. Health-related quality of life and health status in persons with haemophilia A with inhibitors：a prospective，multicentre，non-interventional study（NIS）[J]. Haemophilia，2019，25（3）：382-391.

BAEK H J，PARK Y S，YOO K Y，et al. Health-related quality of life of moderate and severe haemophilia patients：results of the haemophilia-specific quality of life index in Korea[J]. PLoS One，2020，15（9）：e0238686.

LOTFI F，TALEBIANPOUR H，KESHAVARZ K，et al. Cost-utility analysis of factor Ⅷ diet therapies prepared using blood plasma vs. recombinant technique for patients with hemophilia A[J]. Daru，2020，28（1）：287-293.

TAYLOR S，ROOM J，BARKER K. Physical activity le-vels in men with haemophilia：a single centre UK survey[J]. Haemophilia，2020，26（4）：718-725.

BURKE T，ASGHAR S，O’HARA J，et al. Clinical，humanistic，and economic burden of severe hemophilia B in the United States：results from the CHESS US and CHESS US+ population surveys[J]. Orphanet J Rare Dis，2021，16（1）：143.

JOHNSTON K，STOFFMAN J M，MICKLE A T，et al. Preferences and health-related quality-of-life related to disease and treatment features for patients with hemophilia A in a Canadian general population sample[J]. Patient Prefer Adherence，2021，15：1407-1417.

O’HARA J，MARTIN A P，NUGENT D，et al. Evidence of a disability paradox in patient-reported outcomes in haemophilia[J]. Haemophilia，2021，27（2）：245-252.

OLDENBURG J，TRAN H，PEYVANDI F，et al. Health-related quality of life and health status in adolescent and adult people with haemophilia A without factor Ⅷ inhibitors：a non-interventional study[J]. Haemophilia，2021，27（3）：398-407.

XU R H，DONG D，LUO N，et al. Evaluating the psychometric properties of the EQ-5D-5L and SF-6D among patients with haemophilia[J]. Eur J Health Econ，2021，22（4）：547-557.

NIU J Y，NING L W，ZHANG Q，et al. Health-related quality of life of patients with haemophilia：a cross-sectional survey in the Northeast of China[J]. BMJ Open，2022，12（2）：e056668.

RODRIGUEZ-SANTANA I，DASMAHAPATRA P，BURKE T，et al. Health-related quality of life，direct medical and societal costs among children with moderate or severe haemophilia in Europe：multivariable models of the CHESS-PAEDs study[J]. Orphanet J Rare Dis，2022，17（1）：150.

RODRIGUEZ-SANTANA I，DASMAHAPATRA P，BURKE T，et al. Differential humanistic and economic burden of mild，moderate and severe haemophilia in European adults：a regression analysis of the CHESS Ⅱ study[J]. Orphanet J Rare Dis，2022，17（1）：148.

SHAIKH A，PEDRA G，CAWSON M，et al. Examining the impact of haemophilia treatment on health-related quality of life[J]. Haemophilia，2022，28（5）：796-805.

STEEN CARLSSON K，WINDING B，ASTERMARK J，et al. Pain，depression and anxiety in people with haemophilia from three Nordic countries：cross-sectional survey data from the MIND study[J]. Haemophilia，2022，28（4）：557-567.

XU R H，DONG D，LUO N，et al. Mapping the haem-A-QoL to the EQ-5D-5L in patients with hemophilia[J]. Qual Life Res，2022，31（5）：1533-1544.

ZHANG W H，XIE S T，XUE F，et al. Health-related qua-lity of life among adults with haemophilia in China：a comparison with age-matched general population[J]. Haemophilia，2022，28（5）：776-783.

BURKE T，RODRIGUEZ-SANTANA I，CHOWDARY P，et al. Humanistic burden of problem joints for children and adults with haemophilia[J]. Haemophilia，2023，29（2）：608-618.

BENJAMIN K，VERNON M K，PATRICK D L，et al. Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials：an ISPOR COA emerging good practices task force report[J]. Value Health，2017，20（7）：838-855.

连小赟，李魁星，华宝来，等. 北京地区单中心成人重型血友病A患者的三级预防治疗[J]. 中国医学科学院学报，2015，37（4）：398-401.

LIAN X Y，LI K X，HUA B L，et al. Low-dose tertiary prophylactic therapy for severe haemophilia A adults in A single medical center in Beijing area[J]. Acta Acad Med Sin，2015，37（4）：398-401.

林艾灵，谭宝滢，宣建伟，等. 成人血友病A患者预防治疗的多维度价值综述[J]. 中国卫生经济，2023，42（4）：61-64.

LIN A L，TAN B Y，XUAN J W，et al. A review on the multidimensional value of prophylactic treatment in adult hemophilia A patients[J]. Chin Health Econ，2023，42（4）：61-64.

MANCO-JOHNSON M J，SOUCIE J M，GILL J C. Prophylaxis usage，bleeding rates，and joint outcomes of hemophilia，1999 to 2010：a surveillance project[J]. Blood， 2017，129（17）：2368-2374.

吴洁琪，曹林林，孙利华. EQ-5D和SF-6D测量同期健康效用值的比较研究[J]. 卫生经济研究，2020，37（2）：42-45.

WU J Q，CAO L L，SUN L H. Comparative study of EQ-5D and SF-6D in measuring health utility value over the same period[J]. Health Econ Res，2020，37（2）：42-45.

李顺平，吴晶，陈钢. 多属性健康效用量表研制与应用述评[J]. 中国卫生经济，2020，39（10）：5-8.

LI S P，WU J，CHEN G. Review of development and application of multi-attribute utility instruments[J]. Chin Health Econ，2020，39（10）：5-8.

WANG H，CAO C Q，GUO C H，et al. An evaluation of EQ-5D-3L health utility scores using five country-specific tariffs in a rural population aged 45-69 years in Hua county，Henan Province，China[J]. Health Qual Life Outcomes，2020，18（1）：228.

李顺平，窦蕾，房云海. 中国血友病药物经济学评价研究的现状与展望[J]. 罕见病研究，2022，1（4）：468-473.

LI S P，DOU L，FANG Y H. Current situation and pro-spect of pharmacoeconomic evaluation of hemophilia in China[J]. J Rare Dis，2022，1（4）：468-473.

Views

下载量

CSCD

Alert me when the article has been cited

提交

Tools

Publicity Resources

Systematic evaluation of the efficacy and safety of intravenous human immunoglobulin combined with cyclophosphamide in the treatment of systemic lupus erythematosus

Effects of ivabradine on vascular endothelial function in patients with coronary artery disease： a meta-analysis

Meta-analysis about ADRB1 Arg389Gly polymorphism on the efficacy of bisoprolol

Meta-analysis of efficacy and safety of aspirin versus other anticoagulants in the prevention of thromboembolism after orthopedic surgery

Meta-analysis of the efficacy and safety of Saccharomyces boulardii versus Bifidobacterium triple live bacteria in the treatment of pediatric diarrhea

Related Author

ZHANG Junke

HAO Jie

ZHANG Yi

ZHANG Rui

LU Xiaojing

LIU Kefeng

CHEN Congling

YANG Xian

Related Institution

Dept. of Pharmacy， the First Affiliated Hospital of Zhengzhou University

Henan Provincial Center for Drug Clinical Comprehensive Evaluation

Dept. of Cardiology， Nanjing Drum Tower Hospital

School of Basic Medicine and Clinical Pharmacy， China Pharmaceutical University

Dept. of Pharmacy， Nanjing Drum Tower Hospital

Address：8/F,129 Daping Main Street,Yuzhong District,Chongqing 400042,P.R.China
Technical support is provided by Beijing Founder Electronics co., LTD 渝ICP备15012710号公网安备50010302001817
It is recommended to read the content of this site in Chrome&IE9+. Please switch to extreme mode in browser 360.
Cookies We use cookies to help provide and enhance our service and tailor content. By continuing, you agree to the use of cookies.

⁰