免疫耐受新方法单倍型造血干细胞移植治疗重型再生障碍性贫血

摘要

BACKGROUND:Alogeneic hematopoietic stem cel transplantation (alo-HSCT) is an effective mean to cure severe aplastic anemia, and especialy haplotype transplantation is regarded as a transplantation system with Chinese characteristics, and rank at the international leading level. OBJECTIVE:To explore the patterns of haplotype alo-HSCT as a new immune tolerance method for severe aplastic anemia and to solve the transplantation rejection and graft-versus-host disease. METHODS:Twelve patients with severe aplastic anemia who underwent haplotype alo-HSCT at the Department of Hematology, General Hospital of Beijing Military Area, China from April 2013 to May 2014 were enroled. Al these patients received the new regimen of inducing immune tolerance through the application of high-dose cyclophosphamide (400 mg/m2, consecutively 3 days before transplantation; 50 mg/kg, consecutively 3 days after haplotype transplantation). RESULTS AND CONCLUSION:The median time of neutrophil recovery was 17 (13-21) days, and the median time of platelet recovery was 21 (15-31) days. After transplantation, there were one case of degree II acute graft-versus-host disease and one case of chronic graft-versus-host disease, both of which were controled. The folow-up time was 6 months at least, and the median time was 11 months. During the folow-up, one case died of rejection reaction and one case died of severe lung infection. These findings indicate that the new method of inducing immune tolerance with high-dose cyclophosphamide after transplantation for severe aplastic anemia has significant effects in reducing graft-versus-host disease and transplantation-related mortality rate.%背景：异基因造血干细胞移植是根治重型再生障碍性贫血的有效手段，尤其单倍型造血干细胞移植是具有中国特色的移植体系，在国际上处于领先水平。目的：探索免疫耐受新方法单倍型异基因造血干细胞移植治疗重型再生障碍性贫血的模式，解决移植后排斥和移植物抗宿主病问题。方法：解放军北京军区总医院血液科在2013年4月至2014年5月期间采用单倍型供者造血干细胞移植治疗重型再生障碍性贫血患者12例，单倍型供者采集经动员的骨髓及外周血干细胞，预处理方案中给予环磷酰胺400 mg/m2，连续用3 d，移植后+3 d用环磷酰胺诱导免疫耐受，剂量为50 mg/kg。结果与结论：粒细胞植活中位时间17(13-21) d，血小板植活中位时间21(15-31) d。全部患者移植后发生Ⅱ度急性移植物抗宿主病1例，慢性广泛性移植物抗宿主病1例，经治疗后控制。所有存活患者最少随访时间在6个月以上，中位随访时间11个月，死亡2例，其中1例死于排斥反应，另1例死于肺部严重感染，其余10例随访期间生存。结果表明诱导免疫耐受新方法单倍型造血干细胞移植可减少移植物抗宿主病和移植相关病死率，取得了显著成效。