摘要目的:系统评价艾曲波帕治疗免疫性血小板减少症(ITP)成年患者的有效性及安全性。方法:计算机检索PubMed、Embase、Cochrane图书馆等英文数据库，以及万方数据知识服务平台、维普中文科技期刊数据库、中国知网(CNKI)及中国生物医学文献数据库(CBM)等中文数据库中，关于艾曲波帕治疗ITP成年患者的临床研究文献。随机对照试验(RCT)中，研究组患者接受口服艾曲波帕治疗，对照组接受安慰剂治疗；单臂临床试验中，患者接受口服艾曲波帕治疗。文献检索时间设定为上述数据库建库至2020年10月30日。由2位研究者按照本研究设定的文献纳入和排除标准独立筛选文献，评价纳入文献的质量并提取资料。采用Revman 5.3软件对艾曲波帕治疗ITP成年患者的有效性和安全性进行Meta分析。艾曲波帕治疗ITP成年患者有效性及安全性的主要结局评价指标包括有效性指标：总反应率(ORR)、其他ITP治疗药物减低剂量或停药发生率；安全性指标：总不良反应发生率、严重不良反应率、出血事件发生率。结果:通过文献筛选，共计10篇临床研究文献符合本研究纳入标准，其中包含RCT 7项，单臂临床试验3项。纳入Meta分析的ITP成年患者为872例，其中研究组患者为663例，对照组为209例。针对艾曲波帕治疗ITP成年患者有效性和安全性的Meta分析结果如下。①与对照组ITP成年患者相比，研究组患者的ORR较高，并且差异有统计学意义(15.1%比65.1%， RR＝4.18，95% CI：3.03~5.78， P<0.001)。艾曲波帕治疗ITP成年患者期间其他ITP治疗药物减低剂量或停药发生率为47%(95% CI：39%~56%)。②研究组与对照组患者的总不良反应(69.1%比67.4%)、严重不良反应发生率(8.9%比8.8%)分别比较，差异均无统计学意义( RR＝1.01，95% CI：0.85~1.21， P＝0.90； RR＝0.93，95% CI：0.56~1.55， P＝0.79)。研究组患者出血事件发生率低于对照组，并且差异有统计学意义(50.6%比65.3%， RR＝0.79，95% CI：0.70~0.89， P<0.01)。 结论:艾曲波帕治疗ITP成年患者总有效率较高，并且安全性较好，患者出血风险较低。对于接受艾曲波帕治疗的ITP成年患者，可减少其其他ITP药物治疗剂量。

abstractsObjective:To systematically evaluate efficacy and safety of eltrombopag in treatment of adult patients with immune thrombocytopenia (ITP).Methods:From the date of databases inception to October 30, 2020, English databases, including PubMed, Embase, Cochrane library, as well as Chinese databases, including Wanfang Date Knowledge Service Platform, Weipu Chinese Science and Technology Journal Database, China National Knowledge Infrastructure (CNKI) and Chinese Biomedical Literature Database (CBM), were searched to identify clinical researches about eltrombopag in treatment of ITP adult patients received oral eltrombopag in study group and ITP patients received placebo in control group in randomized controlled trials (RCT), and patients received oral eltrombopag in single-arm clinical studies. Two researchers independently screened literature according to inclusion and exclusion criteria of literatures set in this study, and evaluated quality of included literatures and extracted related materials. Efficacy and safety of eltrombopag in treatment of adult patients with ITP were Meta analyzed with Revman 5.3. Main outcome evaluation indexes of effectiveness and safety of eltrombopag in treatment of adult patients with ITP included effectiveness indicator: overall response rate (ORR) and other ITP drugs reduction or discontinuation, and safety indicators: incidence rate of overall adverse reactions, severe adverse reactions, bleeding events.Results:Through literatures screening, a total of 10 clinical researches met the inclusion criteria in this study, including 7 RCT and 3 single-arm clinical studies. A total of 872 adult patients with ITP were included. And 663 patients in study group and 209 patients in control group were included. Meta-analysis results of efficacy and safety of eltrombopag in treatment of adult patients with ITP were as follows. ① ORR of ITP adult patients in study group was higher than that of control group, and the difference was statistically significant (15.1% vs 65.1%, RR=4.18, 95% CI: 3.03-5.78, P<0.001). Incidence rate of other ITP drugs reduction or discontinuation was 47% (95% CI: 39%-56%) during treatment of ITP adult patients with eltrombopag. ② There were no statistically significant differences in incidence rate of overall adverse reactions (69.1% vs 67.4%) and severe adverse reactions (8.9% vs 8.8%) between study group and control group ( RR=1.01, 95% CI: 0.85-1.21, P=0.90; RR=0.93, 95% CI: 0.56-1.55, P=0.79). Incidence rate of bleeding events in study group was lower than that of control group, and the difference was statistically significant (50.6% vs 65.3%, RR=0.79, 95% CI: 0.70-0.89, P<0.01). Conclusions:Eltrombopag is effective in treatment of adult patients with ITP with higher safety and lower risk of bleeding. ITP adult patients who treated with eltrombopag may receive reduced does of other drugs to treat ITP.