马昔腾坦治疗肺动脉高压有效性、安全性和经济性的快速卫生技术评估  

Effectiveness, Safety and Economy of Macitentan in the Treatment of Pulmonary Arterial Hypertension: A Rapid Health Technology Assessment

作　　者：魏安华[1] 曾露[1] 桂玲[1] 贡雪芃[1] Wei Anhua;Zeng Lu;Gui Ling;Gong Xuepeng(Department of Pharmacy,Tongji Hospital,Tongji Medical College,Huazhong Uni-versity of Science and Technology,Wuhan 430030,China)

机构地区：[1]华中科技大学同济医学院附属同济医院药学部,武汉430033

出　　处：《中国药师》2021年第12期2197-2203,共7页China Pharmacist

基　　金：国家自然科学基金青年项目(编号:81803841)。

摘　　要：目的:快速评价马昔腾坦治疗肺动脉高压(PAH)的有效性、安全性和经济性,为临床药物选择和决策提供循证依据。方法:系统检索PubMed、Embase、the Cochrane Library、CNKI、WanFang Data等国内外文献数据库,同时检索卫生技术评估(HTA)相关网站和数据库,检索时限均为建库起至2021年4月8日。由2位评价者根据纳入与排除标准独立筛选文献、提取资料和评价质量后,对结果进行描述性汇总分析。结果:共纳入文献19篇,其中HTA报告2篇,系统评价/Meta分析15篇,药物经济学研究2篇。有效性分析结果显示,相比于安慰剂,马昔腾坦单药或联合治疗可降低PAH患者的临床恶化率和再入院治疗率,提高运动耐量、血流动力学和心脏指数等指标(P<0.05),但对病死率的影响差异无统计学意义(P>0.05)。安全性分析结果显示,马昔腾坦不增加中断治疗率和严重不良事件(SAEs)发生率,但会增加贫血的发生风险(P<0.05)。经济性分析结果显示,由于不同国家不同支付方的意愿支付阈值不同,马昔腾坦的经济性结果存在不一致性,目前缺乏中国人群的经济学评价数据。结论:马昔腾坦治疗PAH具有良好的有效性和安全性,而经济性有待进一步研究评价。Objective: To evaluate the effectiveness, safety and economy of macitentan in the treatment of pulmonary arterial hypertension(PAH) by using rapid health technology assessment. Methods: PubMed, Embase, Web of Science, Cochrane Library, health technology assessment(HTA) database, CNKI, Wanfang databases and HTA relative websites and databases were systematically searched from inception to April 8 th 2021. Two reviewers independently identified the studies, extracted the data and assessed the quality of included studies. Results: A total of 19 literature were included, involving 2 HTA reports, 15 systematic reviews/meta-analysis and 2 pharmacoeconomic studies. The analysis results of effectiveness showed that compared with placebo, monotherapy and combination therapy with macitentan could significantly reduce the risk of clinical worsening and hospitalization, and increase 6 min walking distance(6-MWD), pulmonary vascular resistance(PVR) and cardiac indexes(P<0.05), while there was no significant effect on mortality(P>0.05). The analysis results of safety displayed that monotherapy and combination therapy with macitentan could increase the risk of anemia(P<0.05), while there was no significant difference in the incidence of drug withdrawal and serious adverse events compared with placebo or other treatments. Considering the willingness payment threshold was different in different countries and different payers, the analysis results of pharmacoeconomic studies were inconsistent, and currently there was a lack of cost-effectiveness analysis of macitentan in China. Conclusion: Macitentan is an efficacious and safe choice for PAH, while the economic analysis is necessary to perform in future.

关 键 词：马昔腾坦 肺动脉高压 有效性 安全性 经济性 快速卫生技术评估 

分 类 号：F407.7[经济管理—产业经济]