Huang Aijie, Sun Guangyu, Tang Baolin, Han Yongsheng, Wan Xiang, Yao Wen, Song Kaidi, Cheng Yaxin, Wu Weiwei, Tu Meijuan, Wu Yue, Pan Tianzhong, Zhu Xiaoyu
Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China.
Anhui Provincial Key Laboratory of Blood Research and Applications, Hefei, Anhui 230001, China.
Chin Med J (Engl). 2025 May 5;138(9):1072-1083. doi: 10.1097/CM9.0000000000003216. Epub 2024 Sep 10.
Delayed platelet engraftment is a common complication after umbilical cord blood transplantation (UCBT), and there is no standard therapy. Avatrombopag (AVA) is a second-generation thrombopoietin (TPO) receptor agonist (TPO-RA) that has shown efficacy in immune thrombocytopenia (ITP). However, few reports have focused on its efficacy in patients diagnosed with thrombocytopenia after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
We conducted a retrospective study at the First Affiliated Hospital of the University of Science and Technology of China to evaluate the efficacy of AVA as a first-line TPO-RA in 65 patients after UCBT; these patients were compared with 118 historical controls. Response rates, platelet counts, megakaryocyte counts in bone marrow, bleeding events, adverse events and survival rates were evaluated in this study. Platelet reconstitution differences were compared between different medication groups. Multivariable analysis was used to explore the independent beneficial factors for platelet implantation.
Fifty-two patients were given AVA within 30 days post-UCBT, and the treatment was continued for more than 7 days to promote platelet engraftment (AVA group); the other 13 patients were given AVA for secondary failure of platelet recovery (SFPR group). The median time to platelet engraftment was shorter in the AVA group than in the historical control group (32.5 days vs . 38.0 days, Z = 2.095, P = 0.036). Among the 52 patients in the AVA group, 46 achieved an overall response (OR) (88.5%), and the cumulative incidence of OR was 91.9%. Patients treated with AVA only had a greater 60-day cumulative incidence of platelet engraftment than patients treated with recombinant human thrombopoietin (rhTPO) only or rhTPO combined with AVA (95.2% vs . 84.5% vs . 80.6%, P <0.001). Patients suffering from SFPR had a slightly better cumulative incidence of OR (100%, P = 0.104). Patients who initiated AVA treatment within 14 days post-UCBT had a better 60-day cumulative incidence of platelet engraftment than did those who received AVA after 14 days post-UCBT (96.6% vs . 73.9%, P = 0.003).
Compared with those in the historical control group, our results indicate that AVA could effectively promote platelet engraftment and recovery after UCBT, especially when used in the early period (≤14 days post-UCBT).
血小板植入延迟是脐带血移植（UCBT）后常见的并发症，且尚无标准治疗方法。阿伐曲泊帕（AVA）是第二代血小板生成素（TPO）受体激动剂（TPO-RA），已在免疫性血小板减少症（ITP）中显示出疗效。然而，很少有报告关注其在异基因造血干细胞移植（allo-HSCT）后诊断为血小板减少症患者中的疗效。
我们在中国科学技术大学附属第一医院进行了一项回顾性研究，以评估AVA作为一线TPO-RA对65例UCBT后患者的疗效；将这些患者与118例历史对照进行比较。本研究评估了缓解率、血小板计数、骨髓巨核细胞计数、出血事件、不良事件和生存率。比较了不同用药组之间的血小板重建差异。采用多变量分析探讨血小板植入的独立有益因素。
52例患者在UCBT后30天内给予AVA，并持续治疗超过7天以促进血小板植入（AVA组）；另外13例患者因血小板恢复继发失败（SFPR）给予AVA。AVA组血小板植入的中位时间短于历史对照组（32.5天对38.0天，Z = 2.095，P = 0.036）。在AVA组的52例患者中，46例达到总体缓解（OR）（88.5%），OR的累积发生率为91.9%。仅接受AVA治疗的患者60天血小板植入累积发生率高于仅接受重组人血小板生成素（rhTPO）或rhTPO联合AVA治疗的患者（95.2%对84.5%对80.6%，P <0.001）。患有SFPR的患者OR累积发生率略高（100%，P = 0.104）。在UCBT后14天内开始AVA治疗的患者60天血小板植入累积发生率高于在UCBT后14天之后接受AVA治疗的患者（96.6%对73.9%，P = 0.003）。
与历史对照组相比，我们的结果表明AVA可有效促进UCBT后血小板植入和恢复，尤其是在早期（UCBT后≤14天）使用时。
