Objective To compare the clinical efficacy of allogeneic peripheral blood stem cell transplantation(allo-PBSCT)between severe aplastic anemia(SAA)type Ⅰ(SAAⅠ)and SAA type Ⅱ(SAAⅡ)in North China University of Science and Technology Affiliated Hospital during the same period,so as to provide help for SAA patients to reasonably choose the time of transplantation treatment.Methods The clinical data of SAAⅠ and SAAⅡ patients who underwent allo-PBSCT in North China University of Science and Technology Affiliated Hospital from December 2002 to December 2022 were retrospectively analyzed,including 14 cases of SAAⅠ and 28 cases of SAAⅡ.There were 10 cases of human leukocyte antigen matched sibling donor(MSD group)and 32 cases of haploidentical donor(HID group).All the patients were treated with modified busulfan/cyclophosphamide+rabbit anti-human thymocyte immunoglobulin regimen,cyclosporin A(CsA)+methotrexate(short-term)+mycophenolate mofetil was used for the prevention of graft-versus-host disease,and CsA blood trough concentration was maintained at 150-300 μg/L.The overall survival(OS)rate and event-free survival(EFS)rate after transplantation were compared and observed.Results Follow-up to February 2023,the median follow-up time was 64(7-143)months,7 patients died in the whole group,all of whom were SAAⅡ patients(1 patient in MSD group and 6 patients in HID group),the predicted 3-year OS rate of the whole group was 83.3%(SAAⅠ:100.0%,SAAⅡ:75.0%),and the difference between the two groups was statistically significant(P=0.047).The OS rate was 81.3%in HID group and 90.0%in MSD group,and there were no significant differences between the two groups(P=0.541).The predicted 3-year EFS rate of the whole group was 45.2%(SAAⅠ:64.3%,SAAⅡ:35.7%),and the difference between the two groups was statistically significant(P=0.043).The EFS rate was 43.8%in HID group and 50.0%in MSD group,and there was no significant difference between the two groups(P=0.641).Conclusion For SAA patients with transplantation indications,early allogeneic hematopoietic stem cell transplantation can improve their clinical prognosis.
关键词
重型再生障碍性贫血/慢性再生障碍性贫血/异基因外周血造血干细胞移植
Key words
severe aplastic anemia/chronic aplastic anemia/allogeneic peripheral blood stem cell transplantation