Department of Hematology, Guangzhou First People's Hospital, Institute of Blood Transfusion and Hematology, Guangzhou Medical University, Guangzhou 510180, China.
Department of Hematology, Guangzhou First People's Hospital, Institute of Blood Transfusion and Hematology, Guangzhou Medical University, Guangzhou 510180, China.
Transplant Cell Ther. 2024 Nov;30(11):1099.e1-1099.e10. doi: 10.1016/j.jtct.2024.08.016. Epub 2024 Aug 24.
The aim of the present study was to examine the efficacy of the modified post-transplant cyclophosphamide (PTCy) regimen, which involved reducing the Cy dose to 40 mg on days +3 and +4 in patients with severe aplastic anemia (SAA) subjected to unrelated donor allogeneic hematopoietic stem cell transplantation (URD-HSCT). For this purpose, a prospective single-center trial was conducted and the clinical outcomes were collected from 30 patients with SAA treated with the modified PTCy regimen for URD-HSCT. The median time to neutrophil and platelet engraftment was 13 days (range, 11 to 16) and 12 days (range, 5 to 33), respectively. The cumulative incidence of neutrophil and platelet engraftment was 93.1% ± 0.3% and 96.6% ± 0.2%, respectively. The 2-year overall survival (OS) was 97% (95% confidence interval [CI]: 90%-100%] and 2-year graft-versus-host disease (GVHD) and rejection-free survival (GRFS) was 93% (95% CI: 85%-100%). The incidence rates of acute GVHD (aGVHD) and chronic GVHD (cGVHD) were 13.8 ± 0.4% and 10.3 ± 0.3%, respectively, and no patients developed grades III-IV aGVHD. However, only one patient developed a moderate extensive cGVHD. The incidence of reconstitution varies among different subsets of immune cells after URD-HSCT. Natural killer (NK) cells recover first, followed by CD8 T and CD19 B cells, and finally CD4 T cells. In conclusion, the present study demonstrates that the modified PTCy regimen, with a reduced dose of 40 mg on days +3 and +4, may be an effective regimen for URD-HSCT in patients with SAA and reduce the occurrence of the GVHD.
本研究旨在探讨改良的移植后环磷酰胺（PTCy）方案的疗效，该方案涉及在接受无关供体异基因造血干细胞移植（URD-HSCT）的严重再生障碍性贫血（SAA）患者中，将环磷酰胺剂量减少至第 3 天和第 4 天的 40mg。为此，进行了一项前瞻性单中心试验，从 30 例接受改良 PTCy 方案治疗的 SAA 患者中收集了 URD-HSCT 的临床结果。中性粒细胞和血小板植入的中位时间分别为 13 天（范围为 11 至 16 天）和 12 天（范围为 5 至 33 天）。中性粒细胞和血小板植入的累积发生率分别为 93.1%±0.3%和 96.6%±0.2%。2 年总生存率（OS）为 97%（95%置信区间[CI]：90%-100%），2 年移植物抗宿主病（GVHD）和无排斥反应的存活率（GRFS）为 93%（95%CI：85%-100%）。急性 GVHD（aGVHD）和慢性 GVHD（cGVHD）的发生率分别为 13.8%±0.4%和 10.3%±0.3%，无一例患者发生 3 级或 4 级 aGVHD。然而，仅有 1 例患者发生了中度广泛的 cGVHD。URD-HSCT 后不同免疫细胞亚群的重建率存在差异。自然杀伤（NK）细胞首先恢复，随后是 CD8 T 和 CD19 B 细胞，最后是 CD4 T 细胞。总之，本研究表明，在 SAA 患者中，减少剂量至第 3 天和第 4 天的 40mg 的改良 PTCy 方案可能是一种有效的 URD-HSCT 方案，并降低 GVHD 的发生。
