阵发性睡眠性血红蛋白尿症经济学评价系统综述  

A systematic review of economic evaluation of paroxysmal nocturnal hemoglobinuria

作　　者：宋泽华 刘世贤 李顺平 窦蕾[1,2,3] 王瑞雪 朱晓红[1,2,3] 陈豪 SONG Zehua;LIU Shixian;LI Shunping;DOU Lei;WANG Ruixue;ZHU Xiaohong;CHEN Hao(Centre for Health Management and Policy Research,School of Public Health,Cheeloo College of Medicine,Shandong University,Shandong Jinan 250012,China;NHC Key Lab of Health Economics and Policy Research(Shandong University),Shandong Jinan 250012,China;Center for Health Preference Research,Shandong University,Shandong Jinan 250012,China)

机构地区：[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,山东济南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),山东济南250012 [3]山东大学健康偏好研究中心,山东济南250012

出　　处：《中国医院药学杂志》2025年第2期188-196,共9页Chinese Journal of Hospital Pharmacy

基　　金：国家自然科学基金(编号:72174110)。

摘　　要：目的:了解国内外阵发性睡眠性血红蛋白尿症(paroxysmal nocturnal hemoglobinuria,PNH)经济学评价的研究进展,为后续研究和支付决策提供参考。方法:计算机检索中国知网、万方、维普、中国生物医学文献数据库、PubMed、Web of Science、Cochrane Library、Embase、EBSCO、Scopus等数据库及国际卫生技术评估网站,搜集PNH的药物经济学研究,检索时限为建库至2023年8月31日。结果:初检共获得342篇文献,最终纳入10篇,包括5篇药物经济学评价和5篇卫生技术评估报告,均是基于模型的成本-效用分析。文献质量整体较好,包含支持治疗、依库珠单抗、Ravulizumab和Pegcetacoplan 4种治疗方案。对于经典型PNH患者,依库珠单抗与支持治疗相比不具有经济性。对于成人PNH患者,Ravulizumab较依库珠单抗为绝对优势方案。对于接受C5补体抑制剂稳定剂量治疗≥3个月但贫血状态仍未改善的PNH患者,Pegcetacoplan与依库珠单抗相比,是绝对优势方案,与Ravulizumab相比的经济性尚无一致结论。结论:PNH补体抑制剂价格昂贵,较难具备经济学优势,未来仍需基于中国环境开展卫生技术评估,为卫生决策提供高质量证据,进而改善我国PNH临床诊疗水平,降低患者家庭和卫生体系的经济负担。OBJECTIVE To understand the research progress of economic evaluation of paroxysmal nocturnal hemoglobinuria(PNH) at home and abroad,so as to provide a reference for subsequent research and payment decision-making.METHODS Pharmacoeconomic research of PNH was searched in the databases of CNKI,Wanfang Data,VIP,CBM,PubMed,Web of Science,Cochrane Library,Embase,EBSCO,Scopus and the International Network of Agencies for Health Technology Assessment(INAHTA).The search time limit was from the establishment of the database to August 31,2023.RESULTS A total of 342 literatures were obtained from the initial review and 10 were finally included,including 5 pharmacoeconomic evaluations and 5HTA reports,all of which were model-based cost-utility analyses.The overall quality of the literatures was good.Four treatment options were reported,including supportive care,Eculizumab,Ravulizumab and Pegcetacoplan.For patients with classic PNH,Eculizumab was not economical compared with supportive care.For adult PNH patients,Ravulizumab dominantly superior to Eculizumab.For PNH patients treated with a stable dose of C5 complement inhibitor for more than 3 months but still without improvement in anemia status,Pegcetacoplan dominantly superior to Eculizumab,although its economic advantages compared with Ravulizumab are controversial.CONCLUSION PNH complement inhibitors are expensive and difficult to have economic advantages.In the future,health technology assessments based on the Chinese setting are still needed to provide high-quality evidence for health decision-making,thereby improving the clinical diagnosis and treatment of PNH in China and reducing the economic burden on patients' families and health systems.

关 键 词：阵发性睡眠性血红蛋白尿症 经济学评价 依库珠单抗 卫生技术评估 

分 类 号：R979.9[医药卫生—药品]