奥马珠单抗治疗儿童哮喘的快速卫生技术评估  

Omalizumab in the treatment of children with asthma:a rapid health technology assessment

作　　者：刘佳宁 曾力楠[2,3,4] 王鹂鹂 李海龙 黄亮 成果[4,7,8] 杨春松[2,3,4] 张伶俐 LIU Jianing;ZENG Linan;WANG Lili;LI Hailong;HUANG Liang;CHENG Guo;YANG Chunsong;ZHANG Lingli(Chinese Evidence-based Medicine Center,West China Hospital,Sichuan University,Chengdu 610041,P.R.China;Department of Pharmacy/Evidence-Based Pharmacy Center,West China Second University Hospital,Sichuan University,Children's Medicine Key Laboratory of Sichuan Province,Chengdu 610041,P.R.China;NMPA Key Laboratory for Technical Research on Drug Products in vitro and in vivo Correlation,Chengdu 610041,P.R.China;Key Laboratory of Birth Defects and Related Diseases of Women and Children,Sichuan University,Ministry of Education,Chengdu 610041,P.R.China;West China School of Medicine,Sichuan University,Chengdu 610041,P.R.China;Department of Pediatric Pulmonology and Lmmunology,West China Second University Hospital,Sichuan University,Chengdu 610041,P.R.China;Department of Pediatrics,West China Second University Hospital,Sichuan University,Chengdu 610041,P.R.China;Laboratory of Molecular Translational Medicine,Center for Translational Medicine,Sichuan University,Chengdu 610041,P.R.China)

机构地区：[1]四川大学华西医院中国循证医学中心,成都610041 [2]四川大学华西第二医院药学部/循证药学中心,儿童药物四川省重点实验室,成都610041 [3]国家药品监督管理局药物制剂体内外相关性技术研究重点实验室,成都610041 [4]出生缺陷与相关妇儿疾病教育部重点实验室,成都610041 [5]四川大学华西临床医学院,成都610041 [6]四川大学华西第二医院小儿呼吸免疫科,成都610041 [7]四川大学华西第二医院儿科,成都610041 [8]四川大学转化医学中心分子转化医学实验室,成都610041

出　　处：《中国循证医学杂志》2025年第3期303-310,共8页Chinese Journal of Evidence-based Medicine

基　　金：中华国际医学交流基金会资助项目(编号:Z-2017-24-2301);中国医药教育协会资助项目(编号:23H1139)。

摘　　要：目的通过快速卫生技术评估(HTA)的方法,评价奥马珠单抗用于治疗儿童哮喘的有效性、安全性、经济性。方法计算机检索INAHTA网站及PubMed、Embase、Cochrane Library、Sino Med、CNKI、VIP和WanFang Data数据库,检索时限均为建库至2024年8月。由2名研究者独立进行文献筛选、质量评价及数据提取。通过访谈咨询医生和患者的用药和治疗意见,调查奥马珠单抗临床应用情况。结果共纳入28个研究,含4个HTA,18个系统评价/Meta分析,6个药物经济学(PE)研究,患者包括中重度过敏性哮喘(17个研究)及中重度持续性过敏性哮喘(9个研究)。奥马珠单抗可降低哮喘的显著临床加重发生率、降低哮喘发作次数/出现哮喘症状的天数、改善患者生活质量,并可降低严重不良事件发生率,尤其是与哮喘恶化相关的不良事件。国外PE研究显示,ICER在30109~78009英镑/QALY之间;我国PE研究发现奥马珠单抗的ICER为211217美元/QALY,均高于预先设定的阈值。结论奥马珠单抗可改善中重度过敏性哮喘及中重度持续性过敏性哮喘儿童患者临床症状且未增加不良事件,但其价格昂贵,尚不具备成本-效果。Objective To evaluate the efficacy,safety,and economics of omalizumab for the treatment of pediatric asthma through a rapid health technology assessment(HTA).Methods The search was conducted on INAHTA website and databases such as PubMed,Embase,Cochrane Library,SinoMed,CNKI,VIP,and WanFang Data from inception to August 2024.Literature screening,quality evaluation,and data extraction were conducted independently by two investigators.An interview was conducted to consult the medication and treatment opinions of doctors and patients to investigate its clinical application.Results A total of 28 articles,including HTA articles(4),SR/Meta analyses(18),and pharmacoeconomics articles(6)were included.Omalizumab could reduce the incidence of clinical exacerbations,decrease the number of asthma attacks/days with asthma symptoms,and improve the quality of life.The results of the safety evaluation showed that omalizumab could reduce the rate of serious adverse events,especially those related to worsening asthma.Foreign pharmacoeconomic studies showed ICER ranging from£30109 to£78009.A pharmacoeconomic study in China found an ICER of$211217/QALY for omalizumab,which were both above the pre-set thresholds.Conclusion Omalizumab is a treatment for patients who suffer from moderate to severe persistent allergic asthma or moderate to severe allergic asthma.It has been found to improve disease symptoms without increasing serious adverse events.However,it is not considered cost-effective due to its high price.

关 键 词：奥马珠单抗 儿童哮喘 有效性 安全性 经济性 快速卫生技术评估 

分 类 号：R72[医药卫生—儿科]