Objective: To evaluate the efficacy and safety of matched sibling donor allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of myelofibrosis (MF). Methods: In this case series, the clinical data of 18 patients with MF who received allo-HSCT in the Department of Hematology, Peking University People's Hospital from December 2008 to December 2023 were retrospectively studied. Kaplan-Meier survival analysis and competitive risk model were used to evaluate the probabilities of 3-year overall survival (OS), disease-free survival (DFS), cumulative incidence of relapse (CIR), and transplant related mortality (TRM). The transplant related complications were also analyzed. Results: Among the 18 patients included, there were 12 males and 6 females, with a median age of 50 (range: 28-64) years. All 18 patients achieved neutrophil engraftment, and the time of neutrophil engraftment [M (Q1, Q3)] was 16.0 (11.8, 18.0) days. Twelve patients achieved platelet engraftment, and the platelet engraftment time was 21.0 (16.2, 43.2) days. Six patients had grade Ⅱ to Ⅳ acute graft-versus-host disease (GVHD), and six patients had chronic GVHD. The 3-year OS rate and DFS rate after transplantation were 62.2% and 52.2%, respectively. The 3-year CIR and TRM were 29.7% and 24.6%, respectively. Four patients died during follow-up, with the main cause of death being infections. Conclusion: Matched sibling allo-HSCT is a feasible option for the treatment of MF.
目的： 评价同胞相合异基因造血干细胞移植（allo-HSCT）治疗骨髓纤维化（MF）的疗效及安全性。 方法： 本研究为回顾性病例系列研究，收集2008年12月至2023年12月北京大学人民医院血液科接受同胞相合allo-HSCT治疗的18例MF患者的临床资料，利用Kaplan-Meier生存分析或竞争风险模型计算移植后3年总体生存（OS）率、无病生存（DFS）率、累积复发率（CIR）及移植相关死亡率（TRM），同时统计分析移植相关并发症发生情况。 结果： 18例患者中男性12例，女性6例，中位年龄为50岁（范围28~64岁）。18例患者均实现了粒细胞植入，中性粒细胞植入时间［M（Q1，Q3）］为16.0（11.8，18.0）d；12例患者血小板植入，血小板植入时间为21.0（16.2，43.2）d。6例患者发生Ⅱ~Ⅳ度急性移植物抗宿主病（GVHD），6例患者发生慢性GVHD。移植后3年OS率、DFS率分别为62.2%、52.2%，3年CIR及TRM分别为29.7%、24.6%。随访期内共4例患者死亡，主要死亡原因为感染。 结论： 同胞相合allo-HSCT是治疗MF的可行选择。.