异基因造血干细胞移植治疗10例重型再生障碍性贫血

Allogeneic hematopoietic stem cell transplantation for severe aplastic anemia in 10 cases

背景:再生障碍性贫血目前的一线治疗方案为免疫抑制治疗或造血干细胞移植治疗,其中同胞全合异基因造血干细胞疗效最好。近年来,随着各移植中心技术的进步,单倍体移植也越来越多应用于再生障碍性贫血的治疗。目的:探讨异基因造血干细胞移植治疗重型再生障碍性贫血的临床效果。方法:回顾性分析2014年3月至2018年2月进行异基因造血干细胞移植的10例重型再生障碍性贫血患者的临床资料,行同胞相合异基因造血干细胞移植患者7例,行单倍体异基因造血干细胞移植患者3例。结果与结论:①所有患者移植后均获得造血重建,粒系植入中位时间为移植后10(9-18) d,血小板植入中位时间为移植后15.5(8-39) d,红系植入中位时间为移植后16(11-35) d;移植后1个月骨穿检查均获得疾病缓解;②Ⅱ-Ⅳ度急性移植物抗宿主病发生率40%,Ⅲ-Ⅳ度急性移植物抗宿主病发生率20%。巨细胞病毒血症发生率90%,EB病毒血症发生率60%;③中位随访时间7.3(2.5-49.7)个月,总生存率达80%,同胞相合移植者总生存率为100%;④结果表明,同胞相合异基因造血干细胞移植是治愈重型再生障碍性贫血的首选方法;缺乏同胞相合供者时,单倍体造血干细胞移植也可以作为治疗重型再生障碍性贫血的一种重要移植方式。

BACKGROUND: Nowadays the first-line therapy for aplastic anemia is immunosuppressive therapy or hematopoietic stem cell transplantation. Human leukocyte antigen(HLA)-matched sibling donor allogeneic hematopoietic stem cell transplantation achieves best outcomes. With the development of hematopoietic stem cell transplantation technique, HLA haploidentical hematopoietic stem cell transplantation is popularized in the treatment of aplastic anemia.OBJECTIVE: To evaluate the clinical efficacy of allogeneic hematopoietic stem cell transplantation for severe aplastic anemia.METHODS: Ten cases of severe aplastic anemia who had been treated with HLA matched sibling(n=7) or haploidentical allogeneic hematopoietic stem cell transplantation(n=3) from March 2014 to February 2018 were retrospectively analyzed and reviewed.RESULTS AND CONCLUSION:(1) Hematopoiesis reconstitution was achieved in all the 10 cases. The median time of neutrophils, platelets and reticulocytes was 10(9-18), 15.5(8-39) and 16(11-35) days after transplantation, respectively. One month after transplantation, remission of aplastic anemia was achieved in each case, as confirmed by bone marrow biopsy.(2) Grade II-IV acute graft-versus-host disease occurred in 40% patients, grade III-IV acute graft-versus-host disease in 20% patients, cytomegalovirus viremia in 90% patients, and epstein-barr virus infection in 60% patients.(3) The median follow-up time was 7.3(2.5-49.7) months. The overall survival rate was 80%, and the survival rate of sibling recipients was 100%. To conclude, HLA-matched sibling donor allogeneic hematopoietic stem cell transplantation is the first choice to cure severe aplastic anemia, and haploidentical hematopoietic stem cell transplantation can also be used as an important transplantation method for severe aplastic anemia when lack of matched sibling donors.