Division of Hematology and Medical Oncology, Mayo Clinic, Phoenix, AZ, USA.
Division of Biostatistics, Mayo Clinic, Scottsdale, AZ, USA.
Bone Marrow Transplant. 2019 Feb;54(2):204-211. doi: 10.1038/s41409-018-0226-1. Epub 2018 May 24.
The aim of this study is to compare clinical outcomes of patients who underwent allogeneic stem cell transplantation (HCT) for myelofibrosis with reduced intensity conditioning (RIC) using either Busulfan Fludarabine (BuFlu), Fludarabine Bis-chlorethyl-nitroso-urea/ carmustine Melphalan (FBM) or Fludarabine Melphalan (FluMel) regimens. Sixty-one patients were identified who underwent HCT with one of these RIC regimens. Overall survival (OS) was not different in the 3 groups. However, 100% donor chimerism was seen in more frequently at day +30 and day +100 in patients who received FBM or FluMel than BuFlu, in both CD3 and CD33 fractions. For instance, 100% donor chimerism in CD33 fraction was present in 100% patients in FBM cohort, 90% in FluMel cohort while 44% in BuFlu cohort at day +100. Acute graft-versus host disease, grade 2-4 and grade 3-4, was not statistically different in the 3 groups (BuFlu 47 and 35%, FBM 68 and 27%, FluMel 68 and 46%; p = 0.31 and 0.45). Relapses and non-relapse mortality was also not statistically significantly different. Our study shows similar OS with these 3 RIC regimens in myelofibrosis; although donor chimerism at day +30 and day +100 was better in patients who received FBM and FluMel.
本研究旨在比较接受异基因造血干细胞移植(HCT)治疗骨髓纤维化患者的临床结局,这些患者采用的预处理方案为减低强度预处理(RIC),包括马法兰、氟达拉滨和白消安(BuFlu)、氟达拉滨、双氯乙基亚硝脲和卡莫司汀/马法兰(FBM)或氟达拉滨和马法兰(FluMel)。共确定 61 例患者接受了这些 RIC 方案之一的 HCT。3 组患者的总生存率(OS)无差异。然而,在接受 FBM 或 FluMel 的患者中,CD3 和 CD33 亚群在第 30 天和第 100 天的嵌合率均高于接受 BuFlu 的患者,在 FBM 或 FluMel 组中,100%的患者在第 100 天的 CD33 亚群中达到 100%的供者嵌合率,而 BuFlu 组仅为 44%。3 组患者中 2-4 级和 3-4 级急性移植物抗宿主病(GVHD)的发生率无统计学差异(BuFlu 组分别为 47%和 35%,FBM 组分别为 68%和 27%,FluMel 组分别为 68%和 46%;p=0.31 和 0.45)。复发和非复发死亡率也无统计学差异。本研究表明,在骨髓纤维化患者中,这 3 种 RIC 方案的 OS 相似;尽管在接受 FBM 和 FluMel 的患者中,第 30 天和第 100 天的供者嵌合率更好。