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目的:探讨以氟达拉滨(Flu)加抗胸腺细胞球蛋白(ATG)强效免疫抑制联合极小剂量环磷酰胺(CTX)(1 200mg/m2)预处理方案在再障非亲缘移植中的可行性。方法:对1例诊断重型再生障碍性贫血(SAA),CsA治疗4年无效并反复输注红细胞、血小板的26岁男性患者进行非亲缘异基因造血干细胞移植治疗,高分辨HLA基因型10/10相合,供者为女性,血型相同,干细胞来源为G-CSF动员的外周造血干细胞,预处理方案以Flu加ATG强效免疫抑制为基础,联合极小剂量CTX:CTX 300mg/m2×4d,Flu 30mg/m2×4d,ATG3.75mg/kg×4d,输注单个核细胞(MNC)12.12×108/kg,CD34+细胞13.26×106/kg。术后GVHD预防联合应用环孢素A、短程氨甲喋呤、霉酚酸酯。结果:造血重建良好,术后11dANC>0.5×109/L,术后11dPLT>20×109/L,术后16dPLT>50×109/L。移植后输血量:RBC 3U,PLT 4U。术后21d、100d骨髓XX/XY染色体检测为完全供者型嵌合。术后29d出现巨细胞病毒血症,经更昔洛韦治疗3周转阴,无VOD,现随访6个月,出现局限型cGVHD并控制,血常规正常稳定,Kamofsky评分100分。结论:以Flu、ATG强效免疫抑制为基础的预处理进行非亲缘异基因造血干细胞移植治疗SAA安全有效,并发症少。
Objective: To investigate the effect of fludarabine plus anti-thymocyte globulin (ATG) combined with potent immunosuppression and CTX (1 200 mg / m 2) preconditioning in non-paracramatic aplastic Feasibility. Methods: A non-allogeneic allogeneic hematopoietic stem cell transplantation was performed on a 26-year-old male patient diagnosed with severe aplastic anemia (SAA) and CsA for 4 years and repeatedly receiving erythrocytes and platelets. High-resolution HLA genotypes of 10/10 The donor was female and had the same blood group. The stem cells were derived from G-CSF-mobilized peripheral blood stem cells. The pretreatment protocol was based on Flu plus ATG potent immunosuppression. Combined with minimal dose CTX: CTX 300mg / m2 × 4d, Flu 30mg / m2 × 4d, ATG3.75mg / kg × 4d, mononuclear cells (MNC) 12.12 × 108 / kg, and CD34 + cells 13.26 × 106 / kg. Postoperative GVHD prophylaxis combined with cyclosporine A, short-range methotrexate, mycophenolate mofetil. Results: The hematopoietic reconstructed well, with postoperative 11dANC> 0.5 × 109 / L, PLT> 20 × 109 / L after operation and PLT> 50 × 109 / L after operation. Transplanted blood transfusion: RBC 3U, PLT 4U. At 21 days after operation, bone marrow XX / XY chromosomes on day 100 were tested as complete donor chimerism. After 29 days, cytomegalovirus was found. After treatment with ganciclovir for 3 weeks, no VOD was observed. The patients were followed up for 6 months. The patients had localized cGVHD and were controlled. The blood routine was normal and stable. The Kamofsky score was 100 points. Conclusion: Flu, ATG potent immunosuppression-based preconditioning for non-allogeneic allogeneic hematopoietic stem cell transplantation for the treatment of SAA is safe and effective with few complications.