异基因造血干细胞移植对重型再生障碍性贫血的疗效

THERAPEUTIC EFFICACY OF ALLOGENEIC HEMOTOPOIETIC STEM CELL TRANSPLATION FOR PATIENTS WITH SEVERE APLASTIC ANEMIA

目的探讨异基因造血干细胞移植（Allo-HSCT）治疗重型再生障碍性贫血（SAA）临床效果。方法 6例接受Allo-HSCT的SAA病人均为血缘供者,其中SAA-Ⅰ型4例,SAA-Ⅱ型2例。预处理方案为氟达拉滨＋环磷酰胺＋兔抗人胸腺细胞球蛋白。移植物抗宿主病（GVHD）预防采用环孢菌素A＋短程甲氨蝶呤＋骁悉方案。结果 6例病人移植后均获造血重建,其中性粒细胞〉0.5×109/L和血小板〉20×109/L的中位时间分别为移植后13.51、7.5 d。经微卫星技术检测造血干细胞植入率为100%。移植后1例出现Ⅱ度肠道急性GVHD,1例出现Ⅰ度皮肤急性GVHD,1例出现皮肤广泛型慢性GVHD。随访4~82个月（中位时间30个月）,5例SAA病人存活,1例皮肤慢性广泛型GVHD并发肺部混合感染死亡。结论 Allo-HSCT是治疗SAA的可靠有效方法。

Objective To evaluate the effect of allogeneic hemotopoietic stem cell transplantation（Allo-HSCT） in the treatment of patients with severe aplastic anemia（SAA）. Methods Six SAA patients received Allo-HSCT from consanguineous donors,of whom,four were type SAA-Ⅰ,and two were SAA-Ⅱ.Preparative regime was a combination of fludarabine（Flu）,cyclophosphamide（CTX） and rabbit anti-human thymocyte globulin（ATG）.A combination of cyclosporine A（CSA） with short-range methotrexate（MTX） and mycophenolate mofetil（MMF） was adopted for prophylaxis of graft-versus-host-disease（GVHD）.Results All of the six patients showed reconstruction of hematogenesis after Allo-HSCT.The median time of neutrophils reached 0.5×109/L was 13.5 days,and that of platelets reached 20×109/L was 17.5 days.Implantation rate of hematopoietic stem cells was 100% in all patients detected by microsatellite technology（STR-PCR）.After transplantation,one patients appeared acute grade Ⅱintestinal GVHD,one acute gradeⅠskin GVHD,and one with chronic extensive skin GVHD.Five patients survived during a follow-up of 4-82 months,median being 30 months,one with chronic extensive skin GVHD died of pulmonary mixed infection. Conclusion Allogeneic hematopoietic stem cell transplantation is a reliable and effective therapy for severe aplastic anemia.