艾曲波帕在儿童严重血小板减少血液系统疾病中的临床疗效观察  

Clinical efficacy of Eltrombopag in children with severe thrombocytopenia

作　　者：肖曜均 赵艳霞[1] 仲任[1] 姜健[1] 王玲珍[1] 徐慧娟[1] 孙立荣[1] XIAO Yaojun;ZHAO Yanxia;ZHONG Ren;JIANG Jian;WANG Lingzhen;XU Huijuan;SUN Lirong(Pediatric Hematology Oncology Department,The Affiliated Hospital of Qingdao University,Qingdao 266003,China)

机构地区：[1]山东省青岛市青岛大学附属医院小儿血液肿瘤科,山东青岛266003

出　　处：《中国小儿血液与肿瘤杂志》2023年第1期36-39,共4页Journal of China Pediatric Blood and Cancer

摘　　要：目的探讨艾曲波帕在伴有严重血小板减少的血液系统疾病(免疫性血小板减少症(ITP)、重型再生障碍性贫血(SAA)、造血干细胞移植后血小板减少(PT)中的疗效与安全性。方法回顾性分析2020年9月—2022年3月在青岛大学附属医院应用艾曲波帕治疗的23例患儿临床资料,其中ITP 10例,SAA 8例,PT 5例,评估治疗有效率及不良反应。结果23例患儿治疗前血小板计数在(1-25)×10^(9)/L,治疗前病程在0.25-60个月。ITP治疗有效率为9/10,SAA治疗有效率为5/8,PT治疗有效率为4/5,总有效率为18/23(78%)。23例患儿中仅有1例出现转氨酶升高,给予对症治疗后降至正常。结论艾曲波帕治疗儿童严重血小板减少的血液系统疾病疗效确切,不良反应少。Objective To investigate the efficacy and safety of eltrombopag in the treatment of children with severe thrombocytopenia including immune thrombocytopenic purpura(ITP),severe aplastic anemia(SAA),thrombocytopenia after hematopoietic stem cell transplantation(PT).Methods Clinical data of 23 children treated with eltrombopag in the Affiliated Hospital of Qingdao University from September 2020 to March 2022 were analyzed retrospectively,including 10 cases of ITP,8 cases of SAA and 5 cases of PT.The treatment efficacy and adverse reactions were evaluated.Results The platelet count of 23 children before treatment was(1-25)×10^(9)/L,the course of disease before treatment was(0.25-60)months.The response rate of ITP was 9/10,that of SAA was 5/8,that of PT was 4/5,and the total response rate was 18/23(78%).Only 1 of the 23 children had elevated transaminase,which decreased to normal after symptomatic treatment.Conclusions Eltrombopag is effective in the treatment of children with severe thrombocytopenia,with less adverse reactions.

关 键 词：艾曲波帕 血小板减少 免疫性血小板减少性症 再生障碍性贫血 造血干细胞移植 

分 类 号：R725.5[医药卫生—儿科]