(1) Background: Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) is the only treatment with the potential for cure in patients with myelofibrosis (MF). However, the risk of graft rejection, which is particularly high in MF, and the risk of significant non-relapse mortality must be considered. (2) Methods: In this retrospective, single-center study, we compared allo-HSCT outcomes in 36 adult patients with MF transplanted at two-time intervals (2001–2015 versus 2016–2021). (3) Results: The estimated median overall survival was 48.9 months (95%CI 0.00–98.2) in the cohort transplanted before 2016 and not reached in the more recent years (p= 0.04) due to markedly lower non-relapse mortality (p= 0.02). The 3-year relapse incidence was low in both cohorts (11.1% and 12.5%,p> 0.99). When comparing only subgroups within the more recent cohort based on the presence or absence of total body irradiation (TBI) or the use of sequential regimens, OS and PFS were comparable. (4) Conclusion: Pretreatment with ruxolitinib, intensified conditioning, and the preferential use of haploidentical related instead of mismatched unrelated donors for patients lacking an HLA-identical donor are most likely responsible for the improved outcome after allo-HCT in MF in recent years.
（1）背景：异基因造血干细胞移植（allo-HSCT）是骨髓纤维化（MF）患者唯一可能实现治愈的治疗手段。然而，必须考虑移植排斥风险（在MF患者中尤其高）以及显著的非复发死亡率风险。（2）方法：在这项回顾性单中心研究中，我们比较了36例成年MF患者在两个时间段（2001–2015年与2016–2021年）接受allo-HSCT的结局。（3）结果：2016年前移植组的中位总生存期估计为48.9个月（95%CI 0.00–98.2），而近年移植组尚未达到中位生存期（p=0.04），这主要归因于非复发死亡率显著降低（p=0.02）。两组3年复发率均较低（11.1%与12.5%，p>0.99）。在近年队列中，根据是否接受全身照射（TBI）或采用序贯预处理方案进行亚组比较时，总生存期（OS）与无进展生存期（PFS）结果相当。（4）结论：近年来MF患者allo-HSCT结局的改善，最可能归因于卢索替尼预处理、强化预处理方案的应用，以及对缺乏HLA全相合供者的患者优先选择单倍体相合亲缘供者而非不相合无关供者的策略。
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