摘要
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(Q_(1),Q_(3))]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(Q_(1),Q_(3))]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(Q_(1),Q_(3))]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。
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(Q_(1),Q_(3))]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-Ⅳacute 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 1489.0(270.3,2794.8)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.
作者
王佳丽
张海啸
魏嘉璘
马巧玲
庞爱明
杨栋林
翟卫华
陈欣
何祎
冯四洲
韩明哲
张荣莉
姜尔烈
Wang Jiali;Zhang Haixiao;Wei Jialin;Ma Qiaoling;Pang Aiming;Yang Donglin;Zhai Weihua;Chen Xin;He Yi;Feng Sizhou;Han Mingzhe;Zhang Rongli;Jiang Erlie(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China)
出处
《中华医学杂志》
CAS
CSCD
北大核心
2023年第45期3665-3669,共5页
National Medical Journal of China
基金
国家自然科学基金(82070192)
细胞生态海河实验室创新基金(22HHXBSS00034)
关键词
原发性骨髓纤维化
异基因造血干细胞移植
疗效
安全性
Primary myelofibrosis
Allogeneic hematopoietic stem cell transplantation
Efficacy
Safety
作者简介
通信作者:张荣莉,Email:zhangrongli@ihcams.ac.cn
