阿伐曲泊帕治疗异基因造血干细胞移植后血小板减少症疗效与安全性的14例真实世界数据评价  

Efficacy and safety of avatrombopag for thrombocytopenia following allogeneic hematopoietic stem cell transplantation:A real-world data evaluation on 14 cases

作　　者：刘恩伊[1,2,3,4] 方鹏 信红亚[1,2,3,4] 李淑均 刘弋[1,2,3,4] 徐雅靖 陈焱[1,2,3,4] LIU Enyi;FANG Peng;XIN Hongya;LI Shujun;LIU Yi;XU Yajing;CHEN Yan(Department of Hematology,Xiangya Hospital,Central South University,Changsha 410008;National Clinical Research Center for Geriatric Disorders,Xiangya Hospital,Changsha 410008;Hunan Hematologic Neoplasms Clinical Medical Research Center,Changsha 410008;National Clinical Research Center for Hematologic Diseases,First Affiliated Hospital of Soochow University,Suzhou Jiangshu 215006,China)

机构地区：[1]中南大学湘雅医院血液科,长沙410008 [2]国家老年疾病临床医学研究中心(湘雅医院),长沙410008 [3]湖南省血液肿瘤临床医学研究中心,长沙410008 [4]国家血液系统疾病临床医学研究中心,苏州大学附属第一医院,江苏苏州215006

出　　处：《中南大学学报（医学版）》2023年第3期376-385,共10页Journal of Central South University ：Medical Science

基　　金：国家自然科学基金(81974002);湖南省自然科学基金(2021JJ41006);国家血液系统疾病临床医学研究中心转化研究课题(2021WWC02);湖北陈孝平科学技术发展基金(CXPJJH12000009-102)。

摘　　要：目的:异基因造血干细胞移植(allogeneic hematopoietic stem cell transplantation,allo-HSCT)后血小板减少是一种常见的严重并发症,会导致出血风险增加和预后不良。传统的治疗策略包括输注血小板或CD34^(+)造血干细胞、使用糖皮质激素、注射人免疫球蛋白或重组人血小板生成素,但并未获得令人满意的治疗效果。本研究探讨新型的血小板生成素受体激动剂阿伐曲泊帕治疗移植后血小板减少症的疗效,以期为该药用于临床提供更多的循证医学证据。方法:回顾性分析2020年9月至2021年9月因allo-HSCT后血小板减少症接受阿伐曲泊帕治疗的14例患者。其中,8例为延迟的血小板植入(delayed platelet engraftment,DPE),6例为继发性血小板恢复失败(secondary failure of platelet recovery,SFPR)。评估阿伐曲泊帕的疗效和治疗的安全性及患者的生存情况。结果:阿伐曲泊帕的中位治疗时间为34 d,无患者因不良反应或不耐受药物停止治疗。与治疗前比较,患者血小板计数(P=0.0001)和巨核细胞计数(P=0.0010)均明显增加,血红蛋白水平明显升高(P=0.0017)。13例患者停药后的最佳血小板计数达到完全反应标准。14例患者的中位随访时间为371 d,2年总生存率为78.6%。结论:阿伐曲泊帕能有效地提高allo-HSCT后血小板减少症患者的血小板计数,安全性良好,是移植后血小板减少症较好的治疗选择。Objective:Thrombocytopenia following allogeneic hematopoietic stem cell transplantation(allo-HSCT)is a common and serious complication that leads to an increased risk of bleeding and poor prognosis.Traditional strategies consist of platelet transfusion,glucocorticoid therapy,intravenous human immunoglobulin,recombinant human thrombopoietin injection,and CD34^(+)-selected hematopoietic stem cell transplantation,but the effects of these treatments are not satisfactory and the treatment continues to be challenged.This study aims to determine the treating efficacy of avatrombopag,a novel thrombopoietin receptor agonist,on thrombocytopenia after allo-HSCT,and to increase the evidence-based medical evidence for the clinical use of this drug.Methods:Fourteen patients with thrombocytopenia after allo-HSCT underwent avatrombopag treatment from September 2020 to September 2021 were retrospectively studied.Of these patients,8 patients had delayed platelet engraftment(DPE)and 6 cases had secondary failure of platelet recovery(SFPR).The efficacy and safety of the treatment and the survival of the patients were assessed.Results:The median treatment time of avatrombopag was 34 days,and no patients stopped treatment due to adverse reactions or drug intolerance.Compared with the treatment before,the levels of platelet count,megakaryocytes,and hemoglobin in patients were significantly increased(P=0.0001,P=0.0010,and P=0.0017,respectively).The optimal platelet count of 13 patients reached the complete response standard after drug withdrawal.The median follow-up time of 14 patients was 371 days,and the 2-year overall survival rate was 78.6%.Conclusion:Avatrombopag is effective on increasing platelet counts in patients with thrombocytopenia after allo-HSCT,with a good safety profile.It is a suitable therapeutic option for thrombocytopenia after allo-HSCT.

关 键 词：血小板减少症 异基因造血干细胞移植 血小板生成素 阿伐曲泊帕 

分 类 号：R457.7[医药卫生—治疗学] R558.2[医药卫生—临床医学]