我们报告了一项随机试验（GITMO，AML-R2）的长期结果，比较了白消安和环磷酰胺的 1:1 组合（BuCy2，n = 125）与白消安和氟达拉滨的组合（BuFlu，n = 127）作为接受同种异体造血干细胞移植的急性髓系白血病患者（中位年龄 51 岁，范围 40-65 岁）的预处理方案。中位随访时间为 6 年，证实 BuFlu 接受者的非复发死亡率 (NRM) 显着改善，并在移植后持续长达 4 年（10% 与 20%，p = 0.0388）。这种差异在 51 岁以上的患者中更高（BuFlu 为 11%，BuCy2 为 27%，p = 0.0262）。复发的累积发生率是整个研究人群的首要死因，在两个随机组之间没有差异。同样，即使按中位年龄对患者进行分层，两个队列的无白血病生存期 (LFS) 和总生存期 (OS) 也没有差异。 BuFlu 组与 BuCy2 组的无移植无复发生存率 (GRFS) 在 4 年时分别为 25% 和 20%，在 10 年时分别为 20% 和 17%。因此，NRM 减少所带来的好处不会被复发率增加所抵消。白血病复发仍然是一个主要问题，敦促开发新的治疗方法。© 2024。作者。

We report the long-term results of a randomized trial (GITMO, AML-R2), comparing 1:1 the combination of busulfan and cyclophosphamide (BuCy2, n = 125) and the combination of busulfan and fludarabine (BuFlu, n = 127) as conditioning regimen in acute myeloid leukemia patients (median age 51 years, range 40-65) undergoing allogeneic hematopoietic stem cell transplantation. With a median follow-up of 6 years, significantly better non-relapse mortality (NRM) was confirmed in BuFlu recipients, which is sustained up to 4 years after transplant (10% vs. 20%, p = 0.0388). This difference was higher in patients older than 51 years (11% in BuFlu vs. 27% in BuCy2, p = 0.0262). The cumulative incidence of relapse, which was the first cause of death in the entire study population, did not differ between the two randomized arms. Similarly, the leukemia-free survival (LFS) and overall survival (OS) were not different in the two cohorts, even when stratifying patients per median age. Graft-and relapse-free survival (GRFS) in BuFlu arm vs. the BuCy2 arm was 25% vs. 20% at 4 years and 20% vs. 17% at 10 years. Hence, the benefit gained by NRM reduction is not offsets by an increased relapse. Leukemia relapse remains a major concern, urging the development of new therapeutic approaches.© 2024. The Author(s).