Outcome Of Haploidentical Donor Hematopoietic Stem Cell Transplantation Of Severe Aplastic Anemia

Objective:To evaluate the efficacy of haploidentical donor hematopoietic stem cell transplantation in the treatment of severe aplastic anemia.Methods:The clinical data of 76 SAA patients who underwent HID-HSCT from December 2014 to October 2020 were retrospectively analyzed.There were 50 males and 26 females with median age of 16(3?52)years old.There were 49 SAA-? patients,18 SAA-? patients,and 9 hepatitis-associated aplastic anemia patients.There were 15 cases of bone marrow combined with peripheral blood stem cell transplantation and 61 cases of peripheral blood stem cell transplantation.Conditioning regimen:Cyclophosphamide(CY)+Fludarabine(Flu)+ATG regimen for 46 patients,and Busulfan(Bu)+CY+Flu+ATG regimen for 30 patients.Results:Three patients died in the myelosuppressive phase after transplantation;73 patients had a median time of neutrophil engraftment of 12(9-21)days;in addition to 3 patients who died early,8 patients did not get platelet reconstruction after transplantation,and 65 patients had platelet engraftment with a medium time of 14(9 to 90)d.The incidence of primary graft failure was 5.5%and the incidence of secondary graft failure was 5.5%.The incidence of ???° acute graft-versus host disease(aGVHD)was 38.4%,the incidence of III-IV0aGVHD was 16.4%,the incidence of chronic graft anti-host disease(cGVHD)was 35.8%,and the incidence of extensive cGVHD was 22.4%.The medium follow-up time was 19.5(1-75)months,the prospective overall survival(OS)for 5 years was(78.6±5.0)%,the failure-free survival(FFS)was(75.9±5.1)%,and the transplant-related mortality(TRM)was(20.2±4.9)%.Multi-factor analysis showed that the patient older than 35 years old and the pre-transplant ferritin>1500ng/ml,HCT-CI?3,the number of neutrophils>1 ×109/L at the time of onset,and ???°aGVHD are risk factors affecting OS(P=0.013,0.004,0.02,0.014,0.001).Patients with graft failure had lower FFS than other patients(37.5%vs 84.4%,P<0.001).Conclusion:HID-HSCT is an effective method to treat SAA,which is effective in children and adolescents as well as young patients,and the occurrence of severe aGVHD and severe infection is still the main cause of survival rate.The prevention and treatment of severe aGVHD and infection are essential to improve efficacy.Objective:To evaluate the efficacy of syngeneic hematopoietic stem cell transplantation in the treatment of aplastic anemia,evaluate the hematopoietic reconstruction,and the occurrence of graft failure after transplantation.Methods:The clinic data of 11 aplastic anemia patients undergoing syngeneic HSCT were retrospectively analyzed.Results:All of the 11 patients achieved engraftment after the first transplantation,neutrophils engraftment occurred after a median of 10 days(range 8-23),platelet engraftment occurred after a median of 11 days(range 8-28).Three patients had graft failure,and two of them underwent secondary transplantation,1 case achieved long-term stable engraftment,but graft failure occurred again after hematopoietic reconstruction in another case.The median follow-up time was 53(5 to 135)months.All of the 11 patients survived,the graft failure rate was 27.3%(3/11),and the blood routine of 9 patients was normal for a long time.The results showed that the long course of the disease,active bone marrow growth before transplantation,the fewer CD3+T cells in the graft,and the lack of post-transplant immunosuppression were associated with graft failure.Conclusion:Syngeneic hematopoietic stem cell transplantation has a good long-term survival rate in the treatment of aplastic anemia,and graft failure is still the most important problem.Pre-transplantation conditioning and application of cyclosporine after transplantation contribute to long-term stable implantation.