艾曲泊帕治疗成人原发免疫性血小板减少症的快速卫生技术评估

Rapid Health Technology Assessment on the Safety,Efficacy and Economy of Eltrombopag in the Treatment of Adult Immune Thrombocytopenia

目的探讨艾曲泊帕治疗成人原发免疫性血小板减少症(ITP)的安全性、有效性和经济性的快速卫生技术评估。方法计算机检索PubMed,The Cochrane Library,Embase,以及中国知网、万方数据库等,收集艾曲泊帕和安慰剂、重组人血小板生成素(rh-TPO)、利妥昔单抗(单用或联合)、罗米司亭治疗ITP的相关文献,检索时间为自建库起至2021年1月25日,采用卫生技术评估方法中的系统评价评估工具对系统评价/Meta分析进行质量评价,采用综合卫生经济评价报告标准量表对经济学研究进行质量评价。结果共纳入系统评价/Meta分析8篇,经济学评价3篇,共6 933例患者;有效性方面,与rh-TPO、利妥昔单抗(单用或联合)及安慰剂比较,艾曲泊帕的总体反应均显著升高(P<0.05),与罗米司亭比较无显著差异(P>0.05);安全性方面,与rh-TPO、利妥昔单抗(单用或联合)及安慰剂比较,艾曲泊帕的出血事件、不良事件、抢救治疗的发生率均显著降低(P<0.05),与罗米司亭比较无显著差异;经济性方面,艾曲泊帕比罗米司亭更具成本-效果优势。结论艾曲泊帕治疗ITP的有效性、安全性、经济性均较好,可进一步开展经济学研究。

Objective To investigate the rapid health technology assessment on the safety,efficacy and economy of Eltrombopag in the treatment of adult immune thrombocytopenia(ITP).Methods The studies related Eltrombopag,placebo,recombinant human thrombopoietin(rh-TPO),Rituximab(single or combined)and Romiplostim in the treatment of ITP were collected from the databases of PubMed,The Cochrane Library,Embase,CNKI and Wanfang from the inception to January 25th,2021.The quality of the systematic review/Meta-analysis was evaluated by using the system evaluation tool list in the health technology assessment method,and the quality of the eco-nomic research was evaluated by using the Consolidated Health Economic Evaluation Reporting Standards(CHEERS)scale.Results A total of eight studies of systematic review/Meta-analysis and three studies of the economic review were included,involving 6933 pa-tients.In terms of efficacy,the overall response of Eltrombopag was significantly higher than that of rh-TPO,Rituximab(single or com-bined)and placebo(P<0.05),but there was no significant difference between the Eltrombopag and Romiplostim(P>0.05).In terms of safety,the incidence of bleeding events,adverse events and rescue treatment induced by Eltrombopag were significantly lower than that induced by rh-TPO,Rituximab(single or combined)and placebo(P<0.05),but there was no significant difference between the Eltrombopag and Romiplostim(P>0.05).In terms of economy,the cost-effectiveness of Eltrombopag was better than that of Romi-plostim.Conclusion Eltrombopag is effective,safe and economical in the treatment of ITP,and it is necessary to carry out further eco-nomic research in China.