(1) Background: Treosulfan and melphalan (TreoMel)-based high-dose chemotherapy (HDCT) has shown promising safety and efficacy as a conditioning regimen for acute myeloid leukemia (AML) patients undergoing autologous stem cell transplantation (ASCT). However, despite intensive first-line induction treatment and upfront consolidation with HDCT and ASCT, AML relapse rates are still high, and further efforts are needed to improve patient outcomes. The aim of this study was to compare two melphalan dose schedules in regard to the safety of TreoMel HDCT and patient outcomes. (2) Methods: We retrospectively analyzed the safety and efficacy of two melphalan dose schedules combined with standard-dose treosulfan in AML patients undergoing HDCT and ASCT at the University Hospital of Bern, Switzerland, between August 2019 and August 2023. Patients received treosulfan 42 g/m2combined with either melphalan 140 mg/m2(TreoMel 140) or melphalan 200 mg/m2(TreoMel 200). Co-primary endpoints were progression-free survival (PFS), overall survival (OS), as well as safety profile. (3) Results: We included a total of 51 AML patients: 31 (60.8%) received TreoMel 140 and 20 (39.2%) TreoMel 200. The patients’ basal characteristics were comparable between both cohorts. No significant differences in the duration of hospitalization or the adverse event profile were identified. There were no statistically significant differences in relapse (0.45 vs. 0.30,p= 0.381) and mortality rates (0.42 vs. 0.15,p= 0.064) between the melphalan 140 mg/m2and 200 mg/m2cohorts, nor for PFS (HR: 0.81, 95% CI: 0.29–2.28,p= 0.70) or OS (HR: 0.70, 95% CI: 0.19–2.57,p= 0.59) for the TreoMel 140 vs. TreoMel 200 cohort. (4) Conclusions: A higher dose of melphalan (TreoMel 200) was well tolerated overall. No statistically significant differences for patient outcomes could be observed, possibly due to the relatively small patient cohort and the short follow-up. A longer follow-up and prospective randomized studies would be required to confirm the safety profile and clinical benefit.
(1) 背景：基于曲奥舒凡与美法仑（TreoMel）的大剂量化疗作为急性髓系白血病患者自体干细胞移植的预处理方案，已显示出良好的安全性和疗效。然而，尽管采用强化一线诱导治疗及大剂量化疗联合自体干细胞移植的前期巩固治疗，急性髓系白血病的复发率仍然较高，仍需进一步努力改善患者预后。本研究旨在比较两种美法仑剂量方案在TreoMel大剂量化疗中的安全性及患者预后差异。(2) 方法：我们回顾性分析了2019年8月至2023年8月期间在瑞士伯尔尼大学医院接受大剂量化疗联合自体干细胞移植的急性髓系白血病患者中，两种美法仑剂量方案联合标准剂量曲奥舒凡的安全性和疗效。患者接受曲奥舒凡42 g/m²联合美法仑140 mg/m²（TreoMel 140）或美法仑200 mg/m²（TreoMel 200）治疗。共同主要终点为无进展生存期、总生存期及安全性特征。(3) 结果：共纳入51例急性髓系白血病患者：31例（60.8%）接受TreoMel 140方案，20例（39.2%）接受TreoMel 200方案。两组患者的基线特征具有可比性。住院时间及不良事件特征均无显著差异。美法仑140 mg/m²组与200 mg/m²组在复发率（0.45 vs. 0.30，p=0.381）和死亡率（0.42 vs. 0.15，p=0.064）方面无统计学显著差异，TreoMel 140组与TreoMel 200组的无进展生存期（风险比：0.81，95%置信区间：0.29-2.28，p=0.70）和总生存期（风险比：0.70，95%置信区间：0.19-2.57，p=0.59）亦无显著差异。(4) 结论：较高剂量的美法仑（TreoMel 200）方案总体耐受性良好。未观察到患者预后存在统计学显著差异，这可能与患者队列规模相对较小及随访时间较短有关。需要更长期的随访及前瞻性随机研究来确认其安全性特征和临床获益。
肿瘤(癌症)患者之家