同胞相合异基因造血干细胞移植治疗骨髓纤维化的临床观察  

Clinical outcomes of allogeneic hematopoietic stem cell transplantation from matched sibling donor for myelofibrosis

作　　者：马瑞 唐博瑞 韩婷婷[1] 罗雪宜 韩伟[1] 陈瑶[1] 莫晓冬[1] 许兰平[1] 张晓辉[1] 王昱[1] 黄晓军[1] 孙于谦[1] Ma Rui;Tang Borui;Han Tingting;Luo Xueyi;Han Wei;Chen Yao;Mo Xiaodong;Xu Lanping;Zhang Xiaohui;Wang Yu;Huang Xiaojun;Sun Yuqian(Peking University People′s Hospital,Peking University Institute of Hematology,Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation,Beijing 100044,China)

机构地区：[1]北京大学人民医院、北京大学血液病研究所、造血干细胞移植治疗血液病北京市重点实验室,北京100044

出　　处：《中华内科杂志》2024年第10期961-967,共7页Chinese Journal of Internal Medicine

基　　金：国家自然科学基金(8227010768);科技部重点研发计划(2021YFC2500300)。

摘　　要：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。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(Q 1,Q 3)]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 gradeⅡtoⅣacute 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.

关 键 词：骨髓纤维化 异基因造血干细胞移植 同胞相合供者 

分 类 号：R457.7[医药卫生—治疗学] R551.3[医药卫生—临床医学]