阿伐曲泊帕促进儿童异基因造血干细胞移植后血小板植入的回顾性临床研究

Avatrombopag for platelet engraftment after allogeneic hematopoietic stem cell transplantation in children: a retrospective clinical study

摘要:

目的 探讨阿伐曲泊帕在儿童异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）后促进血小板植入的有效性和安全性，并与重组人血小板生成素（recombinant human thrombopoietin, rhTPO）比较。方法 回顾性分析2023年4月—2024年8月于中国医学科学院血液病医院行allo-HSCT的53例患儿，根据围植入期用药情况，将其分为阿伐曲泊帕组（15例）和rhTPO组（38例）。分析比较两组细胞植入情况及影响血小板植入的因素。结果 移植后14、30和60 d时，阿伐曲泊帕组和rhTPO组分别有20%（3/15）和39%（15/38）、60%（9/15）和82%（31/38）、93%（14/15）和97%（37/38）患儿血小板植入。两组间各时间点血小板植入率、累积血小板植入率、总生存率和非复发生存率方面比较，差异均无统计学意义（P>0.05）。多因素Cox生存分析显示，急性移植物抗宿主病是血小板植入延迟的独立危险因素（P=0.043）。结论 儿童allo-HSCT后应用阿伐曲泊帕有效促进血小板植入，其疗效和安全性与rhTPO相当，是有效选择之一。

Abstract:

Objective To evaluate the efficacy and safety of avatrombopag in promoting platelet engraftment after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children, compared with recombinant human thrombopoietin (rhTPO).Methods A retrospective analysis was conducted on 53 pediatric patients who underwent allo-HSCT at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences from April 2023 to August 2024. Based on medications used during the periengraftment period, patients were divided into two groups: the avatrombopag group (n=15) and the rhTPO group (n=38).Results At days 14, 30, and 60 post-transplant, platelet engraftment was achieved in 20% (3/15), 60% (9/15), and 93% (14/15) of patients in the avatrombopag group, and in 39% (15/38), 82% (31/38), and 97% (37/38) in the rhTPO group, respectively. There were no significant differences between the two groups in platelet engraftment rates at each time point, cumulative incidence of platelet engraftment, overall survival, and relapse-free survival (all P>0.05). Multivariable Cox proportional hazards analysis indicated that acute graft-versus-host disease was an independent risk factor for delayed platelet engraftment (P=0.043).Conclusions In children undergoing allo-HSCT, avatrombopag effectively promotes platelet engraftment, with efficacy and safety comparable to rhTPO, and represents a viable therapeutic option.