艾曲泊帕治疗异基因造血干细胞移植后持续性血小板减少的多中心临床研究

Efficacy of eltrombopag on persistent thrombocytopenia after allogeneic hematopoietic stem cell transplantation:a multi-center clinical study

目的:观察艾曲泊帕治疗异基因造血干细胞移植(allo-HSCT)后持续性血小板减少的有效性及安全性。方法:回顾性分析2018年1月1日至2020年5月31日于郑州大学第一附属医院、河南省人民医院、河南省肿瘤医院血液科确诊为allo-HSCT后持续性血小板减少并应用艾曲泊帕治疗的40例患者的临床资料。结果:40例患者中,14例为移植后原发性血小板减少(PT),26例为移植后继发性血小板减少(SFPR)。开始艾曲泊帕治疗的中位时间为移植后73(53,111)d,起始剂量25 mg/d,最大剂量(25~75)mg/d。28例(70.0%)患者经艾曲泊帕治疗有效(OR),其中21例(52.5%)患者为完全有效(CR),7例(17.5%)患者为部分有效(PR),12例(30.0%)患者无效。艾曲泊帕疗效与治疗前骨髓中巨核细胞数目密切相关,治疗前骨髓中巨核细胞数目正常患者与减少患者相比,OR率及CR率更高(88.2%vs 56.5%,P=0.030;70.6%vs 39.1%,P=0.049),获得OR及CR所需时间更短[(18.87±11.05)d vs(29.15±14.55)d,P=0.043;30(15,38)d vs 48(36,82)d,P=0.025]。治疗过程中9例(22.5%)患者出现1~2级肝损伤,但无患者因此停药。结论:艾曲泊帕是治疗allo-HSCT后持续性血小板减少的一种安全而有效的方法。

Aim:To observe the efficacy and safety of eltrombopag in the treatment of persistent thrombocytopenia after allogeneic hematopoietic stem cell transplantation(allo-HSCT).Methods:A total of 40 patients with persistent thrombocytopenia after allo-HSCT were enrolled in this retrospective study,who came from three medical centers[(the First Affiliated Hospital of Zhengzhou University(n=24),Henan Provincial People′s Hospital(n=10)and Henan Cancer Hospital(n=6),from January 1,2018 to May 31,2020].Results:Out of the 40 patients,14 were post-transplant primary thrombocytopenia(PT)and 26 were post-transplant secondary failure of platelet recovery(SFPR).The median time to start the treatment of eltrombopag was 73(53,111)days after transplantation,with the initial dose of 25 mg/d and the maximum dose of(25-75)mg/d.28 patients(70.0%)were overall response(OR),among whom,21(52.5%)were complete response(CR),7(17.5%)were partially response(PR).Besides,12 patients(30.0%)were no response(NR)to the treatment.The efficacy of eltrombopag was closely related to the number of megakaryocytes in bone marrow before treatment.The OR and CR rates were higher in patients with a normal number of megakaryocytes in bone marrow before treatment compared with those with reduced numbers(88.2%vs 56.5%,P=0.030;70.6%vs 39.1%,P=0.049),and the time required to obtain OR and CR was shorter in patients with a normal number of megakaryocytes[(18.87±11.05)days vs(29.15±14.55)days,P=0.043;30(15,38)days vs 48(36,82)days,P=0.025].During the treatment,9 patients(22.5%)had grade 1-2 liver injury,but none of them discontinued the drug due to serious adverse reactions.Conclusion:Eltrombopag is a safe and effective treatment option for persistent thrombocytopenia after allo-HSCT.