艾曲泊帕治疗原发免疫性血小板减少症疗效及安全性的真实世界单中心数据分析  

Efficacy and safety of eltrombopag in the treatment of primary immune thrombocytopenia:real-world data from a single medical center

作　　者：董喜凤[1,2] 李亚兰 李念斌 林纬楠 王婷 王化泉[1,2] 李丽娟 瞿文[1,2] 邢莉民 刘鸿[1,2] 吴玉红 王国锦[1,2] 宋嘉 关晶[1,2] 王晓明 邵宗鸿[1,2] 付蓉 Dong Xifeng;Li Yalan;Li Nianbin;Lin Weinan;Wang Ting;Wang Huaquan;Li Lijuan;Qu Wen;Xing Limin;Liu Hong;Wu Yuhong;Wang Guojin;Song Jia;Guan Jing;Wang Xiaoming;Shao Zonghong;Fu Rong(Department of Hematology,Tianjin Medical University General Hospital,Tianjin 300052,China;Tianjin Key Laboratory of Bone Marrow Failure and Malignant Hemopoietic Clone Control,Tianjin 300052,China)

机构地区：[1]天津医科大学总医院血液科,天津300052 [2]天津市骨髓衰竭及癌性造血克隆防治重点实验室,天津300052

出　　处：《中华血液学杂志》2024年第3期271-276,共6页Chinese Journal of Hematology

基　　金：国家自然科学基金青年项目(32100656);天津市医学重点学科(专科)建设项目(TJYXZDXK-028A)。

摘　　要：目的观察艾曲泊帕治疗原发免疫性血小板减少症(ITP)的疗效及安全性,并分析相关影响因素。方法对自2018年1月至2022年3月在天津医科大学总医院血液科应用艾曲泊帕的198例ITP患者进行回顾性分析。结果198例ITP患者,男70例、女128例,中位年龄45(7~88)岁,新诊断ITP 130例(65.7%),持续性ITP 25例(12.6%),慢性ITP 43例(21.7%)。基线出血评分:4分以下患者占84.3%,4分及以上患者占15.7%。艾曲泊帕6周内有效率(初始有效率)为78.8%,其中完全反应(CR)率为49.1%,CR1率为14.6%,CR2率为15.2%;中位起效时间7(7,14)d。艾曲泊帕25、50、75 mg剂量组初始反应率分别为74.1%、85.9%和60.0%(P=0.031),50 mg剂量组初始反应率高于25 mg和75 mg剂量组。在治疗过程中,有70.7%的患者维持初始剂量,8.6%的患者调整到50 mg剂量组,6.1%的患者调整到75 mg剂量组,2例患者调整到100 mg剂量组。且调整剂量后25、50、75 mg各个剂量组的持续有效率分别为83.6%、85.3%和85.7%,差异无统计学意义;调整后,100 mg剂量组共4例,持续有效率为100.0%。艾曲泊帕治疗6周后,ITP患者出血评分总体降低,4分以上病例数降低为0,4分患者比例降低,1~2分患者比例变化不显著。艾曲泊帕治疗中,肝功能异常发生率为7.7%,经药物治疗均能好转,未见有合并血栓病例发生。ITP类型与巨核细胞数量分别是艾曲泊帕疗效的影响因素。新诊断的、持续性、慢性ITP患者对艾曲泊帕的初始有效率分别为85.3%、56%和76.2%(P=0.003);巨核细胞数量减少、正常和增多的ITP患者对艾曲泊帕的初始有效率分别为61.8%、87.1%和84.3%(P=0.009)。结论艾曲泊帕作为二线及以上治疗对成人ITP起效时间较快且具有良好的安全性。艾曲泊帕50 mg、25 mg剂量组相比较,50 mg剂量组初始有效率显著增高。新诊断类型的ITP、巨核细胞数量正常或增多的ITP患者对艾曲泊帕初始反应率更高。Objective This study aimed at investigating the efficacy and safety of eltrombopag in the treatment of adult primary immune thrombocytopenia(ITP)and evaluated the factors influencing its efficacy and side effects.Methods A total of 198 patients with adult ITP who were admitted to Tianjin Medical University General Hospital between January 2018 and March 2022 were retrospectively analyzed.The efficacy of each starting dose of eltrombopag was evaluated,and adverse events were analyzed.The factors influencing efficacy were investigated,including sex,age,adult ITP type,platelet antibodies,and combined drug treatments.Results Of the 198 patients,70 males and 128 females with a median age of 45 years(18-88 years)were included;130(65.7%)had newly diagnosed adult ITP,25(12.6%)had persistent adult ITP,and 43(21.7%)had chronic adult ITP.The bleeding event scores at baseline were assessed;84.3%had scores of<4 and 15.7%had scores of≥4.The eltrombopag response rate(initial response)at 6 weeks was 78.8%(complete response[CR]:49.0%;CR1:14.6%;CR2:15.2%).The median response time to eltrombopag was 7(7,14)days.The initial response rates to 25,50,and 75 mg eltrombopag were 74.1%,85.9%,and 60.0%,respectively(P=0.031).The initial response rate to the 50 mg dose was significantly higher than that of the 25-mg and 75-mg doses.Two patients received 100 mg as the starting dose,and their initial response was 0.Regarding dose adjustment,70.7%of the patients remained on the starting dose,8.6%underwent dose adjustment to 50 mg,and 6.1%underwent dose adjustment to 75 mg.Another two patients underwent dose adjustment to 100 mg.After dose adjustment,the persistent response rates were 83.6%,85.3%,and 85.7%for the 25-,50-,and 75-mg doses,respectively,with no significant difference.After dose adjustment,the sustained efficacy rate for the 100-mg dose(4 patients)was 100.0%.After 6 weeks of treatment with eltrombopag,the overall bleeding score of patients with ITP decreased.The number of patients with a score of≥4 decreased to 0,the number of pa

关 键 词：艾曲泊帕 原发免疫性血小板减少症 有效性 安全性 

分 类 号：R55[医药卫生—血液循环系统疾病]