艾曲泊帕治疗AA、MDS相关血小板减少的效果及安全性分析  

Analysis on the Efficacy and Safety of Eltrombopag in the Treatment of Thrombocytopenia Related to AA or MDS

作　　者：张淳嘉[1] 吴爱玲[1] 郑小玲[1] 夏维林[1] 苏秀连[1] ZHANG Chunjia;WU Ailing;ZHENG Xiaoling;XIA Weilin;SU Xiulian(Jieyang People’s Hospital,Jieyang 522000,China;不详)

机构地区：[1]揭阳市人民医院,广东揭阳522000

出　　处：《中外医学研究》2020年第25期35-37,共3页CHINESE AND FOREIGN MEDICAL RESEARCH

基　　金：揭阳市医学科学技术研究项目([2018]80号)。

摘　　要：目的:观察艾曲泊帕在临床中治疗再生障碍性贫血(aplastic anemia,AA)或骨髓增生异常综合征(myelodysplastic syndromes,MDS)相关的血小板减少的疗效和安全性。方法:采用前瞻性、非随机、对照方法,研究2018年7月1日-2019年11月1日在本院就诊的AA或MDS患者45例,按患者意愿分为治疗组(n=17)和对照组(n=28),治疗组患者予标准方案或原有方案基础联合艾曲泊帕治疗,对照组患者予标准方案或继续当前治疗方案,治疗16周评估疗效,比较两组患者的总反应率(overall response rate,ORR)和完全缓解(complete remission,CR)率等,并评估治疗组患者的艾曲泊帕相关的不良事件(adverse event,AE)。结果:治疗组ORR为76.47%,明显高于对照组的42.86%,差异有统计学意义(P=0.030),CR率为52.94%,显著高于对照组的14.29%,差异有统计学意义(P=0.006);艾曲泊帕治疗相关AE程度轻,未发生3级或以上AE。结论:经临床实践,艾曲泊帕治疗AA或MDS相关血小板减少有效且具有良好的安全性。Objective:To investigate the efficacy and safety of Eltrombopag in the treatment of thrombocytopenia related to aplastic anemia(AA) or myelodysplastic syndrome (MDS).Method:From July 1,2018 to November 1,2019,a prospective,non-randomised,controlled study was adopted in the patients with AA or MDS,and the patients were divided into treatment group (n=17) and control group (n=28) as their demands.The treatment group was treated with standard or original treatment method combined with Eltrombopag,while the control group with a standard treatment method or continue current treatment method,and the efficacy was evaluated after 16 weeks,to compare the overall response rate (ORR) and complete remission (CR) rate of the two groups of patients,and the adverse event (AE) related to Eltrombopag was evaluated at the same time.The statistical methods included Kolmogorov-Smirnov Z test and Fisher exact test.Result:The efficacy was evaluated in 16 weeks after the patient enrolled,the ORR of the treatment group (76.47%) was significantly higher than that of the control group (42.86%),the difference was statistically significant (P=0.030).The CR rate of the treatment group (52.94%) was significantly higher than that of the control group (14.29%),the difference was statistically significant (P=0.006).The AE related to Eltrombopag had light degrees,and AE at level 3 or above were not occurred.Conclusion:The Eltrombopag was a effective and safe therapeutic option for thrombocytopenia related to AA or MDS.

关 键 词：艾曲泊帕 再生障碍性贫血 骨髓增生异常综合征 血小板减少 疗效 安全性 

分 类 号：R556.5[医药卫生—血液循环系统疾病] R558.2[医药卫生—内科学]