单倍体造血干细胞移植治疗伴阵发性睡眠性血红蛋白尿克隆的重型再生障碍性贫血的临床效果  

Clinical Effect of Haploidentical Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia with Paroxysmal Nocturnal Hemoglobinuria Clones

作　　者：刘珍珍 卢润青 谢新生[1] 姜中兴[1] 何飞[1] 樊冀鑫 赵海秋 郭荣[1] LIU Zhenzhen;LU Runqing;XIE Xinsheng;JIANG Zhongxing;HE Fei;FAN Jixin;ZHAO Haiqiu;GUO Rong(Department of Hematology,the First Affiliated Hospital of Zhengzhou University,Zhengzhou 450052,China)

机构地区：[1]郑州大学第一附属医院血液内科,河南郑州450052

出　　处：《河南医学研究》2021年第19期3463-3467,共5页Henan Medical Research

基　　金：国家自然科学基金(81070445);河南省自然科学基金(182300410301);河南省科技攻关计划项目(201403029);河南省医学科技攻关计划省部共建备选项目(2018010001)。

摘　　要：目的观察单倍体造血干细胞移植(haplo-HSCT)治疗伴阵发性睡眠性血红蛋白尿(PNH)克隆的重型再生障碍性贫血(SAA)的临床效果。方法回顾性分析2018年2月至2020年3月于郑州大学第一附属医院接受haplo-HSCT治疗的5例伴PNH克隆的SAA患者的临床资料。预处理方案为BU+FC+ATG。采用环孢素A联合霉酚酸酯、短程甲氨蝶呤预防移植物抗宿主病(GVHD)。观察5例患者的移植相关并发症、造血重建、PNH克隆演变等指标。结果5例伴PNH克隆的SAA患者均为男性,中位年龄21岁,初诊时PNH克隆大小中位数为57.33%,诊断至移植中位时间为4个月,期间给予环孢素A、成分输血、雄激素及细胞生长因子等治疗,移植前PNH克隆大小中位数为61.34%。输注的中位单核细胞数为7.72×10^(8)kg^(-1),中位CD34+细胞数为6.40×10^(6)kg^(-1)。5例患者移植后均快速获得造血重建,中性粒细胞及血小板中位植入时间分别为14、16 d。造血重建后植活细胞嵌合鉴定证实均为完全植入。PNH克隆均在移植后1个月复查时转阴,未转化为临床型PNH。中位随访时间为15个月,1例患者于移植后48 d出现继发性植入功能不良,1例发生慢性GVHD,1例出现移植后淋巴细胞增殖性疾病,对症支持治疗后均好转,无移植相关死亡。结论haplo-HSCT对于伴PNH克隆的SAA患者可能具有较好的疗效,且PNH克隆在移植后短期内转阴,但仍需要更多的临床研究加以验证。Objective To observe the clinical effect of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)for severe aplastic anemia(SAA)with paroxysmal nocturnal hemoglobinuria(PNH)clones.Methods The clinical data of 5 patients with SAA with PNH clones undergoing haplo-HSCT in the First Affiliated Hospital of Zhengzhou University from February 2018 to March 2020 were retrospectively analyzed.The pretreatment scheme was BU+FC+ATG.Prophylaxis of graft versus host disease(GVHD)was performed with cyclosporine A,mycophenolate mofetil and short-term methotrexate,and the transplantation related complications,hematopoietic reconstitution and PNH clone evolution were observed.Results The 5 patients were all male,with the median age of 21 years old.The median PNH clone size was 57.33%.The median time from diagnosis to transplantation was 4 months,and cyclosporine A,blood component transfusion,androgen and cell growth factor were applied during this period.The median PNH clone size before transplantation was 61.34%.The median counts of mononuclear cell and CD34+cell were 7.72×10^(8)kg^(-1)and 6.40×10^(6)kg^(-1),respectively.All the 5 patients achieved rapid hematopoietic reconstitution after transplantation.The median implantation time of neutrophils was 14 days,and the median time of platelets was 16 days.The full donor chimerism was attained in all the 5 patients after transplantation.PNH clones became negative within one month after transplantation and did not convert to clinical PNH.During the follow-up at a median time of 15 months,one patient got poor graft function 48 days after transplantation,and one patient developed chronic GVHD,and one patient developed post-transplant lymphoproliferative disorder.The patients improved after symptomatic and supportive treatment.No transplantation related mortality was found in the 5 patients.Conclusion Haplo-HSCT may be an effective treatment for patients with SAA with PNH clones,and PNH clones become negative within a short period after transplantation,but more clinical

关 键 词：单倍体造血干细胞移植 重型再生障碍性贫血 阵发性睡眠性血红蛋白尿克隆 

分 类 号：R551[医药卫生—血液循环系统疾病]