Objective: To retrospectively analyze the incidence and clinical significance of platelet transfusion refractoriness (PTR) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) . Methods: A cohort of 55 CMML patients received allo-HSCT at Peking University Institute of Hematology during 2004-2021 were retrospectively assessed. The incidence of PTR within 30 days after allo-HSCT was retrospectively analyzed, and the impact on clinical outcomes and bleeding event were compared between patients with platelet transfusion refractoriness (PTR) or effective platelet transfusion (EPT) . Results: The incidence of PTR after allo-HSCT in CMML patients was 25.5% (14/55) . PTR patients had a lower rate of platelet engraftment than EPT patients (28.6% vs 100%) , and the median time of engraftment was 67 (33-144) days and 21 (9-157) days respectively (P<0.010) . There was no significant difference between two groups in acute graft-versus-host disease (aGVHD) and chronic graft-versus-host disease (cGVHD) (P=0.183, P=0.455) . After following-up a median of 684 (24-3978) days, the 1-year overall survival (OS) and 1-year leukemia free survival (LFS) in PTR and EPT patients were (35.4±13.9) % vs (75.1±7.8) % (P=0.037) and (28.1±13.3) % vs (65.3±8.2) % (P=0.072) , respectively. The transplant-related mortality (TRM) were (48.2±2.4) % and (9.0±0.25) %, respectively (P=0.009) . Bleeding events occurred in five patients (35.7%) of PTR and 2 patients (4.9%) of EPT (P=0.009) . Conclusion: In CMML patients with allo-HSCT, the incidence of PTR is 25.5%, which was associated with delayed platelet engraftment, increased bleeding events, inferior OS and increased TRM.
目的： 分析血小板输注无效在慢性粒-单核细胞白血病（CMML）患者异基因造血干细胞移植（allo-HSCT）后的发生率及其临床意义。 方法： 回顾性分析2004年至2021年在北京大学血液病研究所接受allo-HSCT后的55例CMML患者，根据移植后30 d内是否发生血小板输注无效分为血小板输注无效组和血小板输注有效组，比较两组造血重建、移植相关并发症及移植后1年生存情况。 结果： ①55例CMML患者中，男28例（50.9%），女27例（49.1%），中位年龄为44（12~63）岁。②14例（25.5%）患者移植后发生血小板输注无效，41例（74.5%）患者血小板输注有效。③血小板输注无效组血小板植入率明显低于血小板输注有效组（28.6%对100%，χ(2)=26.835，P<0.001），血小板植入的中位时间分别为67（33~144）d、21（9~157）d（χ(2)=2.696，P=0.010）。血小板输注无效组、有效组急性移植物抗宿主病（aGVHD）和慢性移植物抗宿主病（cGVHD）发生率差异无统计学意义（χ(2)=3.399，P=0.183；χ(2)=1.573，P=0.455）。④血小板输注无效组、有效组移植后1年总生存（OS）率分别为（35.4±13.9）%、（75.1±7.8）%（χ(2)=4.366，P=0.037），无白血病生存（LFS）率分别为（28.1±13.3）%、（65.3±8.2）%（χ(2)=3.226，P=0.072），移植相关死亡率（TRM）分别为（48.2±2.4）%、（9.0±0.25）%（χ(2)=4.747，P=0.009）。⑤血小板输注无效组移植后出血事件发生率高于血小板输注有效组[35.7%（5/14）对4.9%（2/41），χ(2)=6.934，P=0.009]。 结论： CMML患者allo-HSCT后血小板输注无效发生率为25.5%，显著影响血小板植入率和植入速度，且移植后出血事件发生率增加、OS率降低、TRM增高。.
Keywords: Chronic myelomonocytic leukemia; Platelet transfusion refractoriness; allogeneic hematopoietic stem cell transplantation.