Objective: To investigate the efficacy, safety and the risk factors affecting prognosis of high-risk acute myeloid leukemia (AML) patients treated by cladribine-based intensified conditioning regimen.
Methods: The clinical data of 28 patients with high-risk AML treated by cladribine in combination with busulfan plus cyclophosphamide (BuCy) intensified conditioning regimen before allogeneic hematopoietic stem cell transplantation (allo-HSCT) in Zhujiang Hospital, Southern Medical University from October 2016 to June 2020 were analyzed retrospectively. The overall survival (OS) rate, cumulative progression-free survival (PFS) rate, relapse rate, non-relapse mortality (NRM), regimen related toxicity (RRT) and risk factors affecting prognosis of the patients were analyzed.
Results: The 1-year OS and PFS of the patients after implantation was (78.8±8.6)% and (79.8±8.1)%, while the 1-year cumulative relapse rate and NRM of the patients was 9.3% and 22.0%, respectively. The 1-year expected OS of MRD- high-risk patients before HSCT was 100%. The 1-year expected OS and PFS of the patients in pre-transplant relapse group was (46.9±18.7)% and (50.0±17.7)%, respectively. The incidence of I/II grade RRT was 39.3%. NO III/IV grade RRT were found in 28 patients. Multivariate analysis showed that pre-transplant relapse was the independent risk factor affecting OS and PFS of the patients.
Conclusion: The intensified conditioning regimen of cladribine in combination with BuCy can reduce the relapse rate of high-risk AML transplantation, and its RRT is mild, exhibiting good safety. MRD- high-risk patients before HSCT can achieve better transplant benefits, but the prognosis of patients with relapse before transplantation is not significantly improved. Therefore, for non-relapsed high-risk AML patients, this intensified conditioning regimen deserves to be considered.
题目: 含克拉屈滨的强化预处理方案在异基因造血干细胞移植治疗 高危急性髓系白血病患者中的疗效和安全性分析.
目的: 探讨含克拉屈滨的强化预处理方案在治疗高危急性髓系白血病（AML）患者中的疗效、安全性及影响预后的危险因素.
方法: 回顾性分析2016年10月至2020年6月于南方医科大学珠江医院行异基因造血干细胞移植前接受克拉屈滨联合白消安及环磷酰胺（BuCy）强化预处理方案的28例高危AML患者临床资料，分析移植后累积总生存率（OS）、累积无进展生存率（PFS）、复发率、非复发死亡率（NRM）、预处理相关毒性反应（RRT）以及影响预后的危险因素.
结果: 移植后1年的预期累积OS和PFS分别为（78.8±8.6）%和（79.8±8.1）%，1年累积复发率和NRM分别为9.3%和22.0%。移植前MRD-的高危患者移植后1年预期累积OS为100%，移植前复发组1年的预期累积OS和PFS分别为（46.9±18.7）%和（50.0±17.7）%。I/II级RRT发生率为39.3％，28例患者均未发生III/IV级RRT。多因素分析显示，移植前复发是影响患者OS和PFS的独立不良预后因素.
结论: 克拉屈滨联合BuCy的强化预处理方案降低了高危AML移植后复发率，且其预处理相关毒性轻、安全性良好。移植前MRD-的高危患者可取得较好的移植获益，但移植前复发患者预后的改善并不明显，因此，对于非复发的高危AML患者，可以考虑应用该强化方案作预处理.
Keywords: allogeneic hematopoietic stem cell transplantation; cladribine; high-risk acute myeloid leukemia; intensified conditioning regimen.