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本研究旨在评价HLA不全相合的亲属供者或HLA相合非血缘供者造血干细胞移植在治疗重型再生障碍性贫血(SAA)的疗效和安全性。在2005年11月至2011年5月期间采用非血缘供者或者单倍体相合供者造血干细胞移植治疗SAA患者20例,其中亲缘HLA不合单倍体相合供者14例,非血缘HLA相合供者6例。预处理采用氟达拉滨(FLU)、环磷酰胺(CTX)和抗胸腺细胞球蛋白(ATG)方案,移植物抗宿主病(GVHD)预防方案为经典的环孢素A(CsA)联合短程甲氨蝶呤(MTX)及霉酚酸酯(MMF)。对单倍体相合供者采集经G-CSF动员的骨髓及外周血干细胞联合应用;非血缘供者单纯采集外周血干细胞。结果表明:所有患者均获供者型造血重建,粒细胞植活中位时间14(11-20)d,血小板植活中位时间17(13-31)d,2例取得完全供者植入后2个月发生排斥,其中1例进行母亲单倍体相合供者二次移植,达到完全供者持久植入;移植后发生Ⅱ度急性GVHD 4例,慢性GVHD发生7例,其中1例为慢性广泛性GVHD;14例无病生存,所有存活患者最少随访时间在8个月以上,中位随访时间为48个月,血象完全恢复,Kaplan-Meier计算的累积无病生存率为68.9%。结论:采用FLU、CTX和抗淋巴细胞免疫球蛋白进行预处理,用HLA不全相合的亲属供者或HLA相合非血缘供者造血干细胞移植治疗SAA,植入率高,感染发生率降低,获得良好的长期生存疗效。
This study was designed to evaluate the efficacy and safety of hematopoietic stem cell transplantation for the treatment of severe aplastic anemia (SAA) in an unrelated HLA-matched matched donor or an HLA matched non-donor. From November 2005 to May 2011, non-blood donor or haploidentical donor hematopoietic stem cell transplantation was used to treat 20 patients with SAA. Among them, 14 were matched for HLA-incompatible haploidentical donor and non-HLA matched for donor 6 cases. Pretreatment with fludarabine (FLU), cyclophosphamide (CTX) and anti-thymocyte globulin (ATG) program, graft versus host disease (GVHD) prevention programs for the classic cyclosporin A (CsA) combined with short-range Methotrexate (MTX) and mycophenolate mofetil (MMF). Haploidentical donor collection of G-CSF-mobilized bone marrow and peripheral blood stem cells were used in combination; non-blood donor pure collection of peripheral blood stem cells. The results showed that all the patients were donated by hematopoietic reconstitution. The median time of granulocyte implantation was 14 (11-20) d and the median time of platelet activation was 17 (13-31) days. Two patients achieved complete donor implantation Two cases were rejected 2 months after transplantation, of which 1 case had a secondary transplant with haploidentical maternal haploidentical donor and achieved complete engraftment. After transplanted, 4 cases had grade Ⅱ acute GVHD and 7 cases had chronic GVHD, of which 1 case was Chronic generalized GVHD. Fourteen patients were disease-free. The minimum follow-up time of all survivors was more than 8 months. The median follow-up time was 48 months. The blood was completely recovered. The cumulative disease-free survival rate calculated by Kaplan-Meier was 68.9%. CONCLUSIONS: FLA, CTX and anti-lymphocyte immunoglobulin were used for pretreatment. HLA-matched donor or HLA-matched non-donor donor hematopoietic stem cell transplantation was used to treat SAA. The implantation rate was high, the incidence of infection was reduced, and the result was good Long-term survival effect.