State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, 70585Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Clin Appl Thromb Hemost. 2022 Jan-Dec;28:10760296211068037. doi: 10.1177/10760296211068037.
Secondary failure of platelet recovery (SFPR) is a life-threatening complication that may affect up to 20% of patients after allogeneic hematopoietic stem cell transplantation (HSCT). In this study, to evaluate the efficacy of recombinant human thrombopoietin (rhTPO), we retrospectively analyzed 29 patients who received continuous rhTPO for the treatment of SFPR. Overall response and complete response were observed in 24 (82.8%) patients and 10 (34.5%) patients, at a median time of 21.5 days (range, 3-41 days) and 39.5 days (range, 7-53 days) after initiation of rhTPO treatment, respectively. Among the responders, the probability of keeping overall response and complete response at 1 year after response was 77.3% and 80.0%, respectively. In multivariate analysis, higher CD34 cells (≥3 × 10/kg) infused during HSCT (HR: 7.22, 95% CI: 1.53-34.04, P = 0.01) and decreased ferritin after rhTPO treatment (HR: 6.16, 95% CI: 1.18-32.15, P = 0.03) were indicated to associate with complete response to rhTPO. Importantly, rhTPO was well tolerated in all patients without side effects urging withdrawal and clinical intervention. The results of this study suggest that rhTPO may be a safe and effective treatment for SFPR.
血小板恢复的继发失败（SFPR）是一种危及生命的并发症，可能影响到所有异基因造血干细胞移植（HSCT）患者的 20%。在这项研究中，为了评估重组人血小板生成素（rhTPO）的疗效，我们回顾性分析了 29 例接受连续 rhTPO 治疗 SFPR 的患者。在 rhTPO 治疗开始后 21.5 天（范围，3-41 天）和 39.5 天（范围，7-53 天）时，分别观察到 24 例（82.8%）和 10 例（34.5%）患者的总反应和完全反应。在反应者中，反应后 1 年保持总反应和完全反应的概率分别为 77.3%和 80.0%。多因素分析显示，HSCT 期间输注的 CD34 细胞较高（≥3×10/kg）（HR：7.22，95%CI：1.53-34.04，P=0.01）和 rhTPO 治疗后铁蛋白降低（HR：6.16，95%CI：1.18-32.15，P=0.03）与 rhTPO 完全反应相关。重要的是，rhTPO 在所有患者中均耐受良好，无副作用促使停药和临床干预。这项研究的结果表明，rhTPO 可能是治疗 SFPR 的一种安全有效的方法。
