异基因造血干细胞移植治疗儿童再生障碍性贫血临床分析

目的探讨异基因造血于细胞移植治疗儿童再生障碍性贫血（简称再障）的疗效。方法6例再障患儿中，5例行G．CSF动员的HLA全相合同胞供体骨髓联合外周血造血干细胞移植，平均输入骨髓单个核细胞数4．61（1．5～13．2）×10^8／kg，平均输入外周血单个核细胞数9．36（2．05～16．75）×10^8／kg。预处理方案采用FLU＋CTX＋ATG。1例实行无关供体HLA全相合骨髓移植，输入骨髓单个核细胞数16．8×10^8／kg。预处理方案采用FLU＋CTX＋TBI。急性GVHD预防均采用CSA＋MTX。结果1例移植失败，未植入，移植后第23天自体造血功能恢复。余5例均植入。所有患者均无GVHD的发生。1例病史4年的轻型再障患儿在移植后1年4个月时移植排斥，再障复发合并重症感染治疗无效死亡；余5例随访至2009年2月均无病存活。结论HLA全相合的同胞间骨髓联合外周血造血干细胞移植可作为治疗儿童再障的首选治疗方法；免疫抑制治疗失败且无合适同胞供者的再障患儿可以选择无关供体HLA全相合骨髓移植。

Clinical analysis of six children with aplastic anemia after allogeneic hematopoietic stem cell transplanta-tion

Objective To explore the efficacy of allogeneic hematopeietic stem cell transplantation in children with aplastic anemia. Methods In six children with aplastic anemia, five children received G-CSF primed allogeneic bone marrow plus peripheral stem cell transplantation from HLA-matched siblings, the nucleat- ed cell dose of BM and PBSC transplanted was respectively 4. 61 ( 1.5 ～ 13.2)×108/kg and 9. 36(2. 05 ～ 16. 75 )×108/kg. All five cases used FLU + CTX + ATG as the conditioning regimens. One child received unre-lated HLA-matched bone marrow transplantation, the nucleated cell dose of BM transplanted was 16. 8 x 10S/kg, and used FLU + CTX + TBI as the conditioning regimen. All cases received CSA + MTX + MMF as acute GVHD prophylaxis. Results One child got graft failure and the hematopoiesis recovered at 23 days by itself. The other five children achieved hematopoietic recovery after transplantation. None of them got GVHD after transplanta-tion. One child who had got MAA for four years happened graft rejection,got disease recurrence and finally died of serious infection. Other five children got disease-free survival with a follow-up to Feb 2009. Conclusion AI-logeneic bone marrow plus peripherial stem cell transplantation from HLA-matched siblings could be considered as the preferred treatment for children with aplastic anemia. It is feasible for children with aplastic anemia to re-ceive bone marrow transplant from unrelated HLA-matched donors when the patient was failed in immunosup-pressive therapy and without an available sibling donor.