目的研究人类白细胞抗原(HLA)配型不合造血干细胞移植治疗重型再生障碍性贫血(SAA)的疗效和安全性。方法从2006年1月至2010年5月共入选17例SAA患者接受配型不合造血干细胞移植治疗,供受者间HLA2个位点不合8例,3个位点不合9例,以改良马利兰/环磷酰胺+抗人胸腺细胞免疫球蛋白(BU/CY+ATG)为预处理方案,进行骨髓加外周血干细胞移植。结果所有病例均达到完全供者植入。发生Ⅲ~Ⅳ度急性移植物抗宿主病(GVHD)3例,14例可评估病例中,广泛型慢性GVHD1例;中位随访285(60~1670)d,11例患者生存,9例血象恢复正常,另2例脱离输血。6例患者死于移植相关合并症。结论当无HLA配型相合同胞供者时,SAA患者采用HLA配型不合移植是可行的治疗选择。
Objective To study the efficacy and safety of human leukocyte antigen (HLA) mismatched hematopoietie stem cell transplantation (HSCT) on severe aplastic anemia ( SAA ). Methods From January 2006 to May 2010, 17 patients received mismatched HSCT. HLA antigens were 3-loci- mismatched in 9 patients, 2-loci-mismatched in 8. All patients received recombinant human granulocyte colony-stimulating factor (rhG-CSF) primed bone marrow cells plus peripheral blood stem cells after modified busulfan/cyelophosphamide + antithymocyte immunoglobulin (BU/CY + ATG ) conditioning regimen. Results All patients achieved full donor type engraftment. Grade ~I-IV graft versus host disease (GVHD) occurred in 3 patients and extensive chronic GVHD in 1. With a median following-up time of 285 (60-1670) d, 11 patients were alive, 9 of them had normal blood counts and the other 2 were blood transfusion independent. Six patients died of transplant-related complications. Conclusion Mismatched HSCT is a feasible and safe option for SAA patients without sibling identical donors.