rhTPO联合艾曲泊帕治疗allo-HSCT后血小板持续减少的效果及安全性  

Efficacy and safety of rhTPO combined with eltrombopag in treating persistent thrombocytopenia after allo-HSCT

作　　者：李刚 潘攀 陈欣 杨栋林 庞爱明 姜尔烈 冯四洲 韩明哲 Li Gang;Pan Pan;Chen Xin;Yang Donglin;Pang Aiming;Jiang Erlie;Feng Sizhou;Han Mingzhe(Transplant Center,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences,State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Tianjin 300020,China)

机构地区：[1]中国医学科学院血液病医院(中国医学科学院血液学研究所)移植中心、实验血液学国家重点实验室、国家血液系统疾病临床医学研究中心、细胞生态海河实验室,天津300020

出　　处：《白血病．淋巴瘤》2024年第8期456-461,共6页Journal of Leukemia & Lymphoma

基　　金：国家自然科学基金面上项目(82170217);中国医学科学院医学与健康科技创新工程项目(2022-I2M-C&T-B-091);中国医学科学院创新工程项目(2021-I2M-1-073);天津市血液与再生医学学会临床科研项目(2022 TSHRM08003)。

摘　　要：目的探讨重组人血小板生成素(rhTPO)联合小剂量艾曲泊帕治疗异基因造血干细胞移植(allo-HSCT)后持续血小板减少的效果及安全性。方法回顾性病例系列研究。回顾性分析2018年1月至2021年6月中国医学科学院血液病医院20例诊断为allo-HSCT后血小板持续减少患者临床资料。所有患者移植后未达血小析植入标准[血小板计数(Plt)连续7 d不低于20×109/L且脱离血小板输注], 接受rhTPO 15 000 U, 1次/d, 皮下注射;艾曲泊帕50 mg, 1次/d, 口服。治疗有效为治疗后Plt≥20× 109/L且连续7 d脱离血小板输注, 治疗无效为治疗后Plt<20×109/L或未能脱离血小板输注。分析rhTPO联合小剂量艾曲泊帕治疗效果;评价不良反应;比较治疗有效组和治疗无效组的临床特征;采用Kaplan-Meier法分析治疗有效组和治疗无效组的总生存(OS)、无病生存(DFS)。结果 20例患者中, 急性髓系白血病(AML)9例, 急性淋巴细胞白血病(ALL)5例, 骨髓增生异常综合征(MDS)4例, 重型再生障碍性贫血(SAA)2例;原发性血小板减少症(PFPR)10例, 继发性血小板减少症(SFPR)10例。患者移植后开始治疗的中位时间[M(Q1, Q3)]为79 d(50 d, 89 d), 治疗持续中位时间为19.5 d(15 d, 30 d)。20例中13例(65.0%)治疗有效(其中PFPR 8例, SFPR 5例), 7例(35.0%)治疗无效。治疗有效患者的治疗起效中位时间为10 d(7 d, 19 d)。联合治疗期间, 5例氨基转移酶升高至正常值上限2.5倍以上或胆红素高于2倍正常值上限, 无不良反应相关动脉栓塞、骨髓纤维化、原发病复发等。联合治疗前治疗有效组巨核细胞计数高于治疗无效组, 差异有统计学意义[14个(10个, 20个)比2.5个(2个, 4个);Z=-2.33, P=0.017];两组性别、基础疾病类型、人类白细胞抗原匹配程度、供受者血型、预处理方案、使用抗胸腺细胞球蛋白、输注CD34+细胞数量、血小板减少症类型、合并急性移植物抗宿主病、真菌或细菌感染、病毒Objective To investigate the therapeutic effect and safety of recombinant human thrombopoietin(rhTPO)combined with low-dose eltrombopag in the treatment of persistent thrombocytopenia after allogeneic hematopoietic stem cell transplantation(allo-HSCT).Methods A retrospective case series study was conducted.The retrospective analysis was conducted on the clinical data of 20 patients diagnosed with post-allo-HscT thrombocytopenia at Blood Diseases Hospital of Chinese Academy of Medical Sciences from January 2018 to June 2021.All patients didn't meet the platelet implantation criteria[without the platelet count(Plt)≥20x10°/L for a consecutive period of 7 days and discontinuation of platelet transfusion]after transplantation,and they received subcutaneous injections of rhTPO(15000 U)once daily and oral administration of eltrombopag(50 mg)once.Treatment efficacy was defined as maintaining Plt>20x10°/L for a consecutive period of 7 days after treatment and discontinuation of platelet transfusion;treatment inefficacy was defined as Plt<20x10°/L after treatment or continuation of platelet transfusion.The therapeutic effect of rhTPO combined with low-dose eltrombopag was analyzed;the adverse reactions were evaluated;the clinical characteristics were compared between the effective treatment group and ineffective treatment group;the overall survival(OS)and disease-free survival(DFS)were analyzed using Kaplan-Meier method between the effective treatment group and ineffective treatment group.ResultsAmong the 20 patients,9 were diagnosed with acute myeloid leukemia(AML),5 with acute lymphoblastic leukemia(ALL),4 with myelodysplastic syndromes(MDS),and 2 with severe aplastic anemia(SAA);10 cases were primary failure of platelet recovery(PFPR),and 10 cases were secondary failure of platelet recovery(SFPR).The median time[M(Q,Q:)]from transplantation to initiation of treatment was 79 days(50 days,89 days),and the median duration of treatment was 19.5 days(15 days,30 days).0f the total cohort,treatment was effective in 13 cas

关 键 词：血小板减少症 造血干细胞移植 血小板生成素 

分 类 号：R558.2[医药卫生—血液循环系统疾病] R457.7[医药卫生—内科学]