2023, Vol. 27 ›› Issue (1): 15-20

Allogeneic hematopoietic stem cell transplantation in the treatment of 24 patients with severe aplastic anemia

Fu Chunmei, Zhang Pu, Wang Yang, Li Xiaolin, Xue Yan, Fu Jie, Zhang Cixian, Yang Yujuan, Duan Yaya, Feng Kai   

Abstract: BACKGROUND: Severe aplastic anemia has severe illness and high mortality, which requires rapid recovery of hematopoietic function. At present, hematopoietic stem cell transplantation is the first-line treatment, human leucocyte antigen-matched sibling donor hematopoietic stem cell transplantation is the first choice, and haploidentical hematopoietic stem cell transplantation has also achieved good results as an alternative treatment.
OBJECTIVE: To investigate the efficacy of allogeneic hematopoietic stem cell transplantation (including matched sibling donor hematopoietic stem cell transplantation and haploidentical hematopoietic stem cell transplantation) in the treatment of severe aplastic anemia.
METHODS: The clinical data of 24 patients with severe aplastic anemia diagnosed from April 2015 to July 2021 undergoing allogeneic hematopoietic stem cell transplantation in Xuzhou Center Hospital were retrospectively analyzed. Among them, 8 patients received matched sibling donor hematopoietic stem cell transplantation and 16 patients received haploidentical hematopoietic stem cell transplantation. The pretreatment regimen of 24 patients with severe aplastic anemia was fludarabine, cyclophosphamide and anti-lymphocytic globulin. The prevention of graft versus host disease was cyclosporine combined with short-range methotrexate in matched sibling donor hematopoietic stem cell transplantation, and in haploid matched hematopoietic stem cell transplantation on the basis of this addition of mycophenolate mofetil.
RESULTS AND CONCLUSION: (1) Among the 24 patients, 2 severe aplastic anemia patients died of severe infection during pretreatment, and the other 22 patients reached hematopoietic reconstitution. The median time of neutrophil implantation was 12.5 (10-18) days, and the median time of platelet implantation was 14.5 (10-26) days. (2) Totally 22 patients with successful implantation developed acute graft versus host disease in 8 patients (36%), grade III/IV acute graft versus host disease in 2 patients, chronic graft versus host disease in 4 patients (18%), and grade III/IV chronic graft versus host disease in 1 patient. (3) Totally 18 patients survived and 6 patients died. The 5-year estimated overall survival rate was 74%. (4) These results indicate that allogeneic hematopoietic stem cell transplantation is an effective treatment for severe aplastic anemia. Human leucocyte antigen-matched sibling donor is the first choice. When human leucocyte antigen-matched sibling donor is not available, related human leucocyte antigen-haplo identical donor will be an alternative method.
Key words: severe aplastic anemia, allogeneic hematopoietic stem cell transplantation, haploid transplantation, human leucocyte antigen-matched sibling, hematopoietic reconstitution, graft versus host disease, outcome