Background: Studies of haploidentical-related donor (HRD) stem cell transplantation using a combination of peripheral blood stem cells (PBSCs) and bone marrow as the graft have reported encouraging results for patients with hematological diseases. However, few studies specifically reported transplantation of only PBSCs from HRDs among patients with relapsed or refractory acute myeloid leukemia (AML). Here, the long-term outcomes and side effects of unmanipulated HRD PBSC transplantation (HRD-PBSCT) for relapsed/refractory AML were analyzed.
Methods: We performed a retrospective analysis of the outcomes in relapsed/refractory AML patients who underwent PBSCT from HRDs (n = 36).
Results: Thirty-one (86.1%) patients in the HRD-PBSCT group achieved platelet recovery. The cumulative incidence of acute graft-versus-host disease (aGVHD) in the HRD-PBSCT group was 40.00%, and the cumulative incidence of grades 2-4 aGVHD in this group was 13.33%. A total of 13 patients in the HRD-PBSCT group had recurrent disease at a median of 183 days after transplantation (range: 10-1700 days), reaching cumulative incidences of relapse of 50.28% at 5 years. On multivariate analysis, donor age and patient age >40 years were independent risk factors for inferior disease-free survival or overall survival (P < 0.05). The results of the present study demonstrate rapid and complete neutrophil engraftment, a low incidence of grade 2-4 aGVHD, and promising survival rates in patients after HRD-PBSCT. Thus, granulocyte colony-stimulating factor-primed PBSCs may be a reliable graft source in unmanipulated HRD-HSCT under myeloablative conditioning when no matched sibling donor is available.
Conclusions: Our results support the feasibility, effectiveness, and tolerability of PBSCs as a graft source in unmanipulated HRD transplantation under myeloablative conditioning in patients with leukemia.
单倍体相合外周血干细胞移植治疗复发难治急性髓系白血病的疗效和安全性研究摘要背景: 目前的报道关于采用外周血干细胞和骨髓联合输注的单倍体相合移植治疗恶性血液病取得了很好的疗效，而单用外周血干细胞的单倍体相合移植治疗复发难治急性髓系白血病的研究较少。本文分析了单用外周血干细胞单倍体相合移植治疗复发难治急性髓系白血病长期疗效和毒副反应。 方法: 回顾性分析接受单倍体相合外周血干细胞的36例复发难治急性髓系白血病的疗效。 结果: 36例患者中有31例单倍体移植患者血小板植入，植入率为86.7%。急性移植物抗宿主病的累积发生率为40%，2-4度急性移植物抗宿主病的累积发生率为13.33%。移植后13例患者出现疾病复发，平均复发时间为移植后183天（10 -1700天），5年的累积复发率为50.28%。多因素分析显示，供者年龄或患者年龄≥ 40岁是复发难治急性髓系白血病患者单倍体移植预后不良（总生存率和无疾病生存率）的独立危险因素(P < 0.05)。 单倍体相合外周血干细胞移植治疗复发难治急性髓系白血病，移植后中性粒细胞和血小板重建迅速，2-4度急性移植物抗宿主病发生率低，总体生存预后较好。由此，当复发难治急性髓系患者缺乏同胞全合供者时，粒细胞集落刺激因子动员的外周血干细胞单倍体相合移植可以作为治疗该类患者的较好选择。 结论: 本研究证明了粒细胞集落刺激因子动员的外周血干细胞用于单倍体相合清髓移植治疗急性白血病的可行性、有效性和安全性。.
Keywords: Acute Myeloid Leukemia; Haploidentical Transplantation; Peripheral Blood Stem Cell Transplantation; Recurrence.