Sanz Jaime, Labopin Myriam, Pabst Thomas, Versluis Jurjen, Van Gorkom Gwendolyn, Meijer Ellen, Gedde-Dahl Tobias, Montoro Juan, Arcese William, Pérez-Simón Jose Antonio, Schaap Nicolaas, Maertens Johan, Vrhovac Radovan, Lanza Francesco, Gorin Norbert Claude, Mohty Mohamad, Ciceri Fabio
Hematology Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain.
CIBERONC, Instituto Carlos III, Madrid, Spain.
Bone Marrow Transplant. 2023 Nov;58(11):1197-1202. doi: 10.1038/s41409-023-02075-4. Epub 2023 Aug 8.
We retrospectively compared the impact of the conditioning regimen in adult patients with acute myeloid leukemia (AML) in first complete remission (CR1) that received high-dose myeloablative chemotherapy followed by autologous stem cell transplantation (ASCT) from 2010 to 2021 with either high-dose cytarabine, etoposide and busulfan (BEA), busulfan with cyclophosphamide (BUCY) or busulfan and high-dose melphalan (BUMEL) registered in the EBMT database. Overall 1560 patients underwent ASCT, of which 156, 1143 and 261 received BEA, BUCY and BUMEL, respectively. Compared to BUCY and BUMEL, BEA patients were younger (p < 0.001) and less frequently had NPM1 mutations (p = 0.03). Transplant outcomes at 5 years with BEA, BUCY and BUMEL were: cumulative incidence of relapse 41.8%, 46.6% and 51.6%; non-relapse mortality (NRM) 1.5%, 5.2% and 7.3%; probability of leukemia-free survival (LFS) 56.7%, 48.2% and 41.1%; and overall survival (OS) 71.3%, 62.3% and 56%, respectively. In multivariable analysis the BEA regimen showed significant improvement in OS compared to BUCY (hazard ratio [HR] 0.65; 95% CI, 0.42-0.83; p = 0.048) and BUMEL (HR 0.59; 95% CI, 0.37-0.94; p = 0.029). In conclusion, high-dose myeloablative combination chemotherapy with BEA offered improved outcomes compared to classical BUCY or BUMEL in patients with AML in CR1 undergoing ASCT.
我们回顾性比较了2010年至2021年间接受大剂量清髓性化疗后进行自体干细胞移植（ASCT）的首次完全缓解（CR1）的成年急性髓系白血病（AML）患者中，采用大剂量阿糖胞苷、依托泊苷和白消安（BEA）、白消安联合环磷酰胺（BUCY）或白消安和大剂量美法仑（BUMEL）的预处理方案的影响。EBMT数据库记录显示，共有1560例患者接受了ASCT，其中分别有156例、1143例和261例接受了BEA、BUCY和BUMEL方案。与BUCY和BUMEL相比，接受BEA方案的患者更年轻（p < 0.001），NPM1突变的频率更低（p = 0.03）。接受BEA、BUCY和BUMEL方案的患者5年移植结局如下：复发累积发生率分别为41.8%、46.6%和51.6%；非复发死亡率分别为1.5%、5.2%和7.3%；无白血病生存概率分别为56.7%、48.2%和41.1%；总生存率分别为71.3%、62.3%和56%。多变量分析显示，与BUCY方案相比，BEA方案的总生存率有显著改善（风险比[HR] 0.65；95%置信区间，0.42 - 0.83；p = 0.048），与BUMEL方案相比也有显著改善（HR 0.59；95%置信区间，0.37 - 0.94；p = 0.029）。总之，对于接受ASCT的CR1期AML患者，与经典的BUCY或BUMEL方案相比，采用BEA的大剂量清髓性联合化疗可提供更好的结局。
