Tang Baolin, Huang Lulu, Liu Huilan, Cheng Siqi, Song Kaidi, Zhang Xuhan, Yao Wen, Ning Lijuan, Wan Xiang, Sun Guangyu, Wu Yun, Cheng Jiehui, Long Qi, Sun Zimin, 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, China.
Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Blood Adv. 2020 Aug 25;4(16):3829-3839. doi: 10.1182/bloodadvances.2020002257.
Delayed platelet engraftment is a common complication after umbilical cord blood transplantation (UCBT) accompanied by increased transplant-related complications or death. This study was designed to determine the safety and efficacy of recombinant human thrombopoietin (rhTPO) in promoting platelet engraftment after UCBT. A total of 120 patients scheduled to receive UCBT were randomly assigned to the rhTPO group (300 U/kg once daily from days 14 to 28 after UCBT, n = 60) or the control group (n = 60). The primary outcome was the 60-day cumulative incidence of platelet engraftment after single-unit cord blood transplantation. The 60-day cumulative incidence of platelet engraftment (platelet count ≥20 × 109/L) and the 120-day cumulative incidence of platelet recovery (platelet count ≥50 × 109/L) were both significantly higher in the rhTPO group than in the control group (83.1% vs 66.7%, P = .020; and 81.4% vs 65.0%, P = .032, respectively). In addition, the number of required platelet infusions was significantly lower in the rhTPO group than in the control group (6 vs 8 units, respectively; P = .026). The cumulative incidence of neutrophil engraftment and the probability of 2-year overall survival, disease-free survival, and graft-versus-host disease-free relapse-free survival did not differ between the 2 groups. Other transplant-related outcomes and complications did not differ between the 2 groups, and no severe adverse effects were observed in patients receiving rhTPO. This study demonstrated that rhTPO is well tolerated in patients and could effectively promote platelet engraftment after UCBT. This study was registered on the Chinese Clinical Trial Registry (http://www.chictr.org.cn/index.aspx) as ChiCTR-IPR-16009357.
血小板植入延迟是脐带血移植（UCBT）后常见的并发症，会伴随移植相关并发症增加或死亡风险上升。本研究旨在确定重组人血小板生成素（rhTPO）在促进UCBT后血小板植入方面的安全性和有效性。共有120例计划接受UCBT的患者被随机分为rhTPO组（UCBT后第14天至28天，300 U/kg，每日1次，n = 60）或对照组（n = 60）。主要结局是单单位脐带血移植后60天血小板植入的累积发生率。rhTPO组血小板植入（血小板计数≥20×10⁹/L）的60天累积发生率和血小板恢复（血小板计数≥50×10⁹/L）的120天累积发生率均显著高于对照组（分别为83.1%对66.7%，P = 0.020；以及81.4%对65.0%，P = 0.032）。此外，rhTPO组所需血小板输注次数显著低于对照组（分别为6次对8次；P = 0.026）。两组中性粒细胞植入的累积发生率以及2年总生存、无病生存和无移植物抗宿主病无复发生存的概率无差异。两组间其他移植相关结局和并发症无差异，接受rhTPO的患者未观察到严重不良反应。本研究表明，rhTPO在患者中耐受性良好，可有效促进UCBT后血小板植入。本研究已在中国临床试验注册中心（http://www.chictr.org.cn/index.aspx）注册，注册号为ChiCTR-IPR-16009357。
