摘要
目的:评价FCA(氟达拉滨+减量环磷酰胺+抗胸腺细胞球蛋白)作为重型再生障碍性贫血(SAA)患者异基因造血干细胞移植(allo-HSCT)预处理方案的疗效及安全性。方法:对2014年11月—2020年5月在徐州医科大学附属医院骨髓移植中心接受allo-HSCT治疗的27例SAA患者进行回顾性研究,其中男13例(48.1%),女14例(51.9%);中位年龄23(3~48)岁;骨髓联合外周血造血干细胞移植23例(85.2%),脐血联合外周血造血干细胞移植2例(7.4%),外周血造血干细胞移植2例(7.4%);HLA全合同胞供者(MSD)15例(55.6%),HLA单倍体相合亲缘供者(HFD)12例(44.4%)。预处理方案:氟达拉滨120 mg/m^(2),-5~-2 d;环磷酰胺120 mg/kg,-5~-2 d;抗胸腺细胞球蛋白(兔抗人)10 mg/kg,-5~-2 d。采用环孢素A+短程甲氨蝶呤+霉酚酸酯方案预防移植物抗宿主病(GVHD)。结果:27例患者中性粒细胞、血小板中位植入时间分别为13(10~25)d、15(11~111)d。3例(11.1%)患者发生Ⅲ~Ⅳ度急性GVHD,1例(3.7%)患者发生广泛型慢性GVHD。27例患者中7例死亡,生存的20例患者均获得造血重建,并在移植后不同时间点检测STR-PCR结果均为完全供者嵌合。移植过程中17例(63.0%)患者发生CMV血症;20例(74.1%)发生EBV血症,其中1例(3.7%)进展为EBV相关移植后淋巴细胞增殖性疾病;10例(37.0%)患者在骨髓抑制期发生败血症;3例(11.1%)发生肺部感染;1例(3.7%)发生中枢神经系统感染;1例(3.7%)发生肛周感染。存活患者的中位随访时间39.1(3.5~70.2)个月,预期5年总生存率及无失败生存率均为73.3%。MSD组及HFD组的5年总生存率及无失败生存率差异均无统计学意义(均为86.2%和57.1%;χ^(2)=2.962,P=0.085)。结论:氟达拉滨联合减量环磷酰胺及抗胸腺细胞球蛋白预处理方案行HLA同胞全相合及亲缘单倍体相合allo-HSCT治疗SAA均安全有效。
Objective:To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation(allo-HSCT)for severe aplastic anemia(SAA)with pre-transplant conditioning using FCA(fludarabine+reduced-dose cyclophosphamide+anti-thymocyte globulin).Methods:The clinical data of 27 SAA patients(male 13,female 14)who received allo-HSCT from November 2014 to May 2020 in the bone marrow transplantation center of Affiliated Hospital of Xuzhou Medical University were analyzed retrospectively.Among them,23 patients(85.2%)received bone marrow combined mobilized peripheral blood stem cells transplantation,2 patients(7.4%)received cord blood combined mobilized peripheral blood stem cells transplantation and 2 patients(7.4%)underwent mobilized peripheral blood stem cells transplantation.Fifteen patients(55.6%)received MSD-HSCT and 12 patients(44.4%)received HFD-HSCT.The conditioning regimen was consisted of fludarabine 120 mg/m^(2),-5 to-2 d,cyclophosphamide 120 mg/kg,-5 to-2 d;ATG 10 mg/kg,-5 to-2 d.GVHD prophylaxis was performed by cyclosporine+short-term course methotrexate+mycophenolate mofetil.Results:The median durations of myeloid engraftment were 13(10 to 25)days and 15(11 to 111)days for platelets.Three patients(11.1%)had grade III to IV aGVHD.Only 1 patient(3.7%)had extensive cGVHD during the follow-up period.Seven cases of the 27 patients were died.All the 20 surviving patients achieved hematopoietic reconstitution.And the results of STR-PCR detection during the 20 surviving patients at different time points after transplantation were complete donor chimerism.During the transplantation process,17 patients(63.0%)developed CMV viremia,20 patients(74.1%)with EBV viremia,1 patient(3.7%)with EBV progressed post-transplant lymphoproliferative disorders;10 patients(37.0%)with septicemia during bone marrow suppression;3 patients(11.1%)with pulmonary infection;1 patient(3.7%)with central nervous systems infections;1 patient(3.7%)with perianal infection.The median follow-up time of survival patients was 39.1(3.5 to 70.2)months.Both the expected 5-year overall survival rate and failure-free survival rate were 73.3%.There was no significant difference in 5-year overall survival rate and failure-free survival rate between MSD group and HFD group(86.2%and 57.1%;χ^(2)=2.962,P=0.085).Conclusion:For both MSD-HSCT and HFD-HSCT,fludarabine combined with reduced-dose cyclophosphamide and anti-thymocyte globulin pretreatment regimen is safe and effective in the treatment of SAA.
作者
毛晓蕾
李群
王莹
李德鹏
李振宇
邱婷婷
徐开林
MAO Xiaolei;LI Qun;WANG Ying;LI Depeng;LI Zhenyu;QIU Tingting;XU Kailin(Department of Hematology,Affiliated Hospital of Xuzhou Medical University,Xuzhou,221000,China)
出处
《临床血液学杂志》
CAS
2021年第5期314-318,326,共6页
Journal of Clinical Hematology
基金
国家自然科学基金(No:81400129)
中国博士后科学基金(No:2015113010)。
关键词
再生障碍性贫血
预处理
造血干细胞移植
氟达拉滨
环磷酰胺
aplastic anemia
conditioning regimen
hematopoietic stem cell transplantation
fludarabine
cyclophosphamide
