Xu Zheng-Li, Ji Jie, Wang San-Bin, Li Nai-Nong, Zhou Jian, Lin Ming-Hao, Xu Lan-Ping, Wang Yu, Zhang Xiao-Hui, Huang Xiao-Jun
Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University, Beijing.
Department of Hematology and Institute of Hematology, Stem Cell Transplantation and Celluar Therapy Division, Clinic Trial Center, West China Hospital, Sichuan University, Chengdu.
Haematologica. 2025 Mar 1;110(3):629-639. doi: 10.3324/haematol.2024.286040.
This study aimed to demonstrate the clinical outcomes of granulocyte colony-stimulating factor (G-CSF)/antithymocyte globulin (ATG), posttransplantation cyclophosphamide (PTCy) and PTCy combined with low-dose ATG (PTCy with ATGlow)-based haploidentical transplantation protocols in patients with hematologic malignancies. The comparisons were conducted via propensity score matching (PSM) analysis to balance the basic characteristics among different groups and were based on the transplantation data reported to the Chinese Bone Marrow Transplantation Registry Group (CBMTRG) from January 2020 to December 2022. For each patient in the PTCy or PTCy with ATGlow group, patients (at a 1:2 ratio) from the G-CSF/ATG group were selected. In total, the PTCy group (N=122) was matched with the G-CSF/ATG group 1 (N=230), and the PTCy+ATGlow group (N=123) was matched with the G-CSF/ATG group 2 (N=226). Compared with those in the PTCy group, the incidences of 28- day neutrophil engraftment (P=0.005), 100-day platelet engraftment (P=0.002), median time to neutrophil engraftment (P<0.001) and platelet engraftment (P=0.011) were significantly greater in the G-CSF/ATG group. No significant differences were observed in acute graft-versus-host disease (aGVHD) incidence or relapse incidence. In addition, patients in the G-CSF/ ATG group had lower non-relapse mortality (NRM; P<0.001), better 3-year overall survival (OS; P<0.001) and leukemia-free survival (P<0.001) rates than those in the PTCy group. Similarly, the G-CSF/ATG group achieved lower NRM (P<0.001) and better 3-year leukemia-free survival (P=0.002) than the PTCy+ATGlow group. In conclusion, G-CSF/ATG-based haplo-HSCT may be a preferential choice for the Chinese population with hematologic malignancies. In the future, a randomized controlled study is needed for further confirmation.
本研究旨在论证粒细胞集落刺激因子(G-CSF)/抗胸腺细胞球蛋白(ATG)、移植后环磷酰胺(PTCy)以及基于PTCy联合低剂量ATG(PTCy联合低剂量ATG)的单倍体相合移植方案在血液系统恶性肿瘤患者中的临床疗效。通过倾向评分匹配(PSM)分析进行比较,以平衡不同组间的基本特征,比较基于2020年1月至2022年12月向中国骨髓移植登记组(CBMTRG)报告的移植数据。对于PTCy组或PTCy联合低剂量ATG组中的每例患者,按1:2的比例从G-CSF/ATG组中选择患者。总共,PTCy组(N=122)与G-CSF/ATG组1(N=230)进行匹配,PTCy+低剂量ATG组(N=123)与G-CSF/ATG组2(N=226)进行匹配。与PTCy组相比,G-CSF/ATG组28天中性粒细胞植入率(P=0.005)、100天血小板植入率(P=0.002)、中性粒细胞植入中位时间(P<0.001)和血小板植入中位时间(P=0.011)均显著更高。急性移植物抗宿主病(aGVHD)发生率或复发率未观察到显著差异。此外,G-CSF/ATG组患者的非复发死亡率(NRM;P<0.001)更低,3年总生存率(OS;P<0.001)和无白血病生存率(P<0.001)均优于PTCy组。同样,G-CSF/ATG组的NRM低于PTCy+低剂量ATG组(P<0.001),3年无白血病生存率更高(P=0.002)。总之,基于G-CSF/ATG的单倍体造血干细胞移植可能是中国血液系统恶性肿瘤患者的优先选择。未来,需要进行随机对照研究以进一步证实。