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目的探讨脐血移植(UCBT)与外周血造血干细胞移植(PBSCT)治疗重型再生障碍性贫血的临床疗效,同时通过对比性研究,为患者选择合适的治疗方案。方法 PBSCT组以环磷酰胺(CTX)、抗胸腺细胞球蛋白(ATG)、氟达拉滨(FLU)为主要预处理药物,术后以环孢素A(CsA)+骁悉(MMF)+甲氨蝶呤(MTX)预防移植物抗宿主病(GVHD);UCBT组以CTX+ATG为主要预处理药物,术后以CsA预防GVHD。结果两组移植病例中粒系恢复情况相当,但UCBT的红系、巨核系恢复较慢。PBSCT组植入率为85.71%。而UCBT组仅有1例完全植入(亲缘供者),植入率仅为3.12%。PBSCT组完全缓解22例,部分缓解1例,死亡5例,总有效率78.57%;脐血移植完全缓解25例,无效2例,死亡5例,总有效率78.12%。结论 UCBT虽植入率低但临床疗效与PBSCT相当,是重型再生障碍性贫血患者无合适人类白细胞抗原配型时有效的治疗方案。
Objective To investigate the clinical efficacy of umbilical cord blood transplantation (UCBT) and peripheral blood stem cell transplantation (PBSCT) in the treatment of severe aplastic anemia. At the same time, we select the appropriate treatment for patients through comparative study. Methods CTCT, ATG and FLU were used as the main pretreatment drugs in PBSCT group. After CsA + MMF + Methotrexate (MTX) was used to prevent graft versus host disease (GVHD). UCBT group was given CTX + ATG as the main pretreatment drug and CsA was used to prevent GVHD after operation. Results There was no significant difference between the two groups in the recovery of myeloid lineages, but the erythroid and megakaryocyte lines of UCBT recovered more slowly. PBSCT group implantation rate was 85.71%. In UCBT group, only 1 patient was completely implanted (relative donor), and the implantation rate was only 3.12%. In PBSCT group, 22 cases were completely relieved, 1 case partially relieved and 5 died. The total effective rate was 78.57%. Complete cord blood transplantation was relieved in 25 cases, 2 cases were ineffective and 5 cases died. The total effective rate was 78.12%. Conclusion Although the implantation rate of UCBT is low, the clinical efficacy is comparable to that of PBSCT. It is an effective treatment for patients with severe aplastic anemia without proper HLA matching.