诺西那生治疗脊髓性肌萎缩症的快速卫生技术评估  

Rapid Health Technology Assessment of Nusinersen in the Treatment of Spinal Muscular Atrophy

作　　者：余玺辉 郭嘉亮 李迎春 陈永壮 张文 莫小兰 YU Xihui;GUO Jialiang;LI Yingchun;CHEN Yongzhuang;ZHANG Wen;MO Xiaolan(Department of Pharmacy,Guangzhou Women and Children′s Medical Center,Guangdong Provincial Clinical Research Center for Child Health,Guangzhou,Guagdong 510623,China;Department of endocrinology,Guangzhou Women and Children′s Medical Center,Guangdong Provincial Clinical Research Center for Child Health,Guangzhou,Guagdong 510623,China)

机构地区：[1]广州市妇女儿童医疗中心药学部,广东省儿童健康与疾病临床医学研究中心,广东广州510623 [2]广州市妇女儿童医疗中心内分泌科,广东省儿童健康与疾病临床医学研究中心,广东广州510623

出　　处：《今日药学》2023年第4期284-289,共6页Pharmacy Today

基　　金：广东省医院药学研究基金(2021A35,2023A27);广州市妇女儿童医疗中心院内基金项目(GWCMC2020-5-010)。

摘　　要：目的通过快速卫生技术评估诺西那生钠注射液的有效性、安全性和经济性,为决策者提供证据参考。方法系统检索PubMed、CNKI等数据库,对有效性、安全性和经济性结果进行综合比较与分析。结果初始检索共获得121篇题录,最终共纳入1篇HTA报告、2篇Meta分析和4篇经济学研究。有效性方面,诺西那生钠注射液可以改善脊髓性肌萎缩症患者的运动功能,降低其死亡风险。安全性方面,诺西那生钠注射液的不良反应发生率与对照组相近。经济性方面,国外研究显示,诺西那生钠注射液可延长患者的质量调整生命年但不具有较好的成本效益。结论诺西那生钠注射液可显著改善部分临床结局指标。后续应开展更多的研究,以进一步讨论其临床使用价值。OBJECTIVE To evaluate the efficacy,safety and economy of nusinersen by rapid health technology assessment tools and methods,and to provide evidence reference for decision makers.METHODS We systematically searched PubMed,CNKI and other databases to make qualitative and comprehensive comparison and analysis of the effectiveness,safety and economic results.RESULTS A total of 121 titles were obtained in the initial search,and one HTA report,two meta⁃analyses and four economic studies were finally included.In terms of efficacy,nusinersen improved motor function and reduced the risk of death in patients with spinal muscular atrophy.In terms of safety,the incidence of adverse reactions of nusinersen was similar to that of the control group.In terms of economy,foreign studies had shown that nusinersen prolonged the quality adjusted life years of patients but was not cost⁃effective.CONCLUSION Nusinersen can significantly improve some clinical outcomes without increasing the safety risk.More studies should be carried out to further discuss its clinical value.

关 键 词：诺西那生 快速卫生技术评估 脊髓性肌萎缩症 

分 类 号：R95[医药卫生—药学]