氟达拉滨 (Flu) 和马法兰 (Mel) 降低强度预处理常用于急性髓性白血病 (AML) 和骨髓增生异常综合征 (MDS) 患者的同种异体造血细胞移植 (allo-HCT)。然而，关于 Mel 剂量对异基因 HCT 的毒性和临床结果影响的证据有限。我们回顾性比较了 345 名 AML 或 MDS 患者接受 100 mg/m2（Mel-100，n=62）总 Mel 剂量与 140 mg/m2（Mel-140，n= 283）与流感结合使用。 allo-HCT 的中位年龄为 66 岁，中位随访时间为 36.5 个月。对于 Mel-100 组与 Mel-140 组，任何级别的胃肠道毒性发生率分别为 40.3% 与 67.8% (p<0.001)，第 100 天的 II-IV 级急性移植物抗宿主病 (GVHD) 发生率分别为 21.0% 与 67.8% (p<0.001)。 43.1% (p=0.001) 和 2 年慢性 GVHD 发生率分别为 17.4% 和 27.1% (p=0.033)。在多变量分析中，Mel-140导致胃肠道毒性（HR=1.83，p=0.013）、II-IV级急性GVHD（HR=2.35，p=0.003）和中/重度慢性GVHD（HR=3.13）的风险较高，p=0.007）。总梅尔剂量对口腔粘膜炎、非复发死亡率、复发、无复发生存率和总生存率没有独立影响。虽然我们的观察结果需要独立验证，但我们的研究结果支持将 Mel-100 与 Flu 联合使用，以最大程度地减少与 GVHD 相关的异基因 HCT 毒性和发病率。版权所有 © 2024。由 Elsevier Inc. 出版。

Fludarabine (Flu) and melphalan (Mel) reduced-intensity conditioning is frequently used for allogenic hematopoietic cell transplant (allo-HCT) in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). However, there is limited evidence on the impact of Mel dosing on toxicities and clinical outcomes of allo-HCT. We retrospectively compared 8/8 HLA matched donor allo-HCT outcomes of 345 patients with AML or MDS receiving total Mel dose of 100 mg/m2 (Mel-100, n=62) versus 140 mg/m2 (Mel-140, n=283) in combination with Flu. Median age at allo-HCT was 66 years and median follow-up was 36.5 months. For Mel-100 versus Mel-140 groups, any grade gastrointestinal toxicity rates were 40.3% vs. 67.8% (p<0.001), day 100 grade II-IV acute graft-versus-host disease (GVHD) rates were 21.0% vs. 43.1% (p=0.001) and 2-year chronic GVHD rates were 17.4% vs. 27.1% (p=0.033). In multivariable analysis, Mel-140 resulted in higher risks of gastrointestinal toxicity (HR=1.83, p=0.013), grade II-IV acute GVHD (HR=2.35, p=0.003), and moderate/severe chronic GVHD (HR=3.13, p=0.007). Total Mel dose had no independent impact on oral mucositis, non-relapse mortality, relapse, relapse-free survival, and overall survival. While independent validation of our observation is warranted, our findings support using Mel-100 in combination with Flu to minimize allo-HCT toxicities and morbidities related to GVHD.Copyright © 2024. Published by Elsevier Inc.