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目的探讨异基因造血干细胞移植(allo-HSCT)治疗儿童重型再生障碍性贫血(SAA)的疗效及并发症。方法 4例SAA患儿,均接受氟达拉滨、环磷酰胺、抗胸腺细胞球蛋白预处理;其中3例患儿行HLA全相合同胞骨髓造血干细胞移植(BMT),1例患儿行HLA全相合同胞外周血造血干细胞移植(PBSCT)。同胞供者采集重组人粒细胞集落刺激因子5μg.kg-1.d-1,动员骨髓及外周血干细胞。采用环孢素+短疗程小剂量甲氨蝶呤方案预防移植物抗宿主病,前列腺素E预防肝静脉闭塞综合征,更昔洛韦预防巨细胞病毒感染,美司那及水化碱化预防出血性膀胱炎。通过DNA短串联重复序列多态性分析检测植入情况。结果 2例BMT患儿及1例PBSCT患儿完全植入;1例BMT患儿嵌合植入。中性粒细胞>0.5×109L-1中位时间12 d(9～15 d),血小板>20×109L-1中位时间19 d(12～30 d)。结论 allo-HSCT是治疗儿童SAA的有效方法,维持造血功能以及移植后并发症的发生及防治,仍是目前重点讨论的课题。
Objective To investigate the efficacy and complications of allo-HSCT in the treatment of pediatric severe aplastic anemia (SAA). Methods Four children with SAA received pretreatment with fludarabine, cyclophosphamide and anti-thymocyte globulin. Three of the three children underwent HLA-identical myeloid bone marrow stem cell transplantation (BMT) All matched sibling peripheral blood stem cell transplantation (PBSCT). Sibling donor collected recombinant human granulocyte colony-stimulating factor 5μg.kg-1.d-1 mobilization of bone marrow and peripheral blood stem cells. Cyclosporine + short-course, low-dose methotrexate regimen for the prevention of graft-versus-host disease, prostaglandin E for the prevention of hepatic veno-occlusive syndrome, ganciclovir for the prevention of cytomegalovirus infection, and prevention of metasex and hydration and alkalinization Hemorrhagic cystitis. Implantation was detected by DNA short tandem repeat polymorphism analysis. Results Two cases of BMT children and one case of PBSCT were completely implanted. One case of BMT children received chimerism implantation. Neutrophils> 0.5 × 109L-1 median time 12 d (9 ~ 15 d), platelets> 20 × 109 L-1 median time 19 d (12 ~ 30 d). Conclusion Allo-HSCT is an effective method for the treatment of children with SAA. Maintaining the hematopoietic function and the occurrence and prevention of complications after transplantation are still the key topics for discussion.