Objective: To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of primary myelofibrosis (PMF) patients. Methods: A total of 14 cases of PMF who underwent allo-HSCT from December 2008 to December 2022 were analyzed retrospectively, including 8 males and 6 females with a median age [M(Q1, Q3)]of 36 (24, 42) years. Three-year overall survival (OS), disease free survival (DFS), cumulative incidence of relapse (CIR), transplantation-related mortality (TRM) were analyzed. Meanwhile, the complications were followed up by telephone and outpatient appointments for 49.6 (9.0,93.1) months. Results: All patients received myeloablative conditioning regimens (MAC). All patients had successful engraftment, and the median time of neutrophils and platelet engraftment were 13.5 (11.8, 18.0) days and 19.5 (13.5, 24.5) days, respectively. Ⅱ-Ⅳ acute graft versus host disease (GVHD) occurred in 3 cases, while chronic GVHD in 8 cases. The rate of 3-year OS,DFS,CIR and TRM were (92.9±6.9)%, (76.0±12.2)%, (38.6±2.7)% and (7.1±0.5)% respectively after a median follow-up time of 1 489.0 (270.3,2 794.8) days. Two patients died from treatment-related complications, one of which died 39 days after transplantation due to heart failure caused by severe anemia, the other patient died 6 years after relapse due to pulmonary infection. Conclusion: Allo-HSCT can be used as a safe and effective approach to treat PMF.
目的： 评价异基因造血干细胞移植（allo-HSCT）治疗原发性骨髓纤维化（PMF）的疗效及安全性。 方法： 回顾性分析2008年12月至2022年12月中国医学科学院血液病医院接受allo-HSCT治疗的14例PMF患者的临床资料，其中男8例，女6例，年龄［M（Q1，Q3）］为36（24，42）岁。通过电话、查阅门诊及住院病历资料进行随访，随访时间［M（Q1，Q3）］为49.6（9.0，93.1）个月，主要观察指标为移植后3年总生存（OS）率、无病生存（DFS）率、复发率及移植相关死亡率，同时统计分析移植相关并发症发生情况。 结果： 全部14例患者均采用清髓性预处理（MAC）方案。14例患者移植后全部获得造血重建，中性粒细胞植入时间［M（Q1，Q3）］为13.5（11.8，18.0）d，血小板植入时间为19.5（13.5，24.5）d。3例患者发生Ⅱ~Ⅳ度急性移植物抗宿主病（GVHD），8例患者发生慢性GVHD。移植后3年OS率、DFS率、复发率及移植相关死亡率分别为（92.9±6.9）%、（76.0±12.2）%、（38.6±2.7）%、（7.1±0.5）%。共2例患者死于治疗相关并发症，其中1例因重度贫血导致心力衰竭，死于移植后39 d；另1例于移植后6年死于复发后肺部感染。 结论： allo-HSCT是治疗PMF安全有效的方法。.