异基因造血干细胞移植治疗儿童重型再生障碍性贫血

Allogeneic Hematopoietic Stem Cell Transplantation Therapy on Severe Aplastic Anemia in Children

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 analyze the efficacy and complications of allogeneic hematopoietic stem cell transplantation （ allo - HSCT） in children with severe aplastic anemia （SAA）. Methods Four children with SAA received the preparative regimen of the fludarabine, the antithymocyte globulin and the cyclophosphamide. There were 3 children who received allogeneic bone marrow transplantation （BMT） from human leukocyte antigenmatched sibling donors; there was 1 child who received allogeneic peripheral blood stem cell transplantation （allo- PBSCT） from HLA - matched sibling donor. BMT cells and peripheral blood stem cells were collected after the administration of granulocyte colony - stimulating factor 5 μg · kg ^- 1·d ^- 1. Cyclosporine A and methotrexate were routinely used as the preparative regimen of graft - versus - host disease. The prostaglandin E was used as the preparative regimen for veno - occlusive disease of the liver, the ganciclovir was used as the preparative regimen for cytomegalovirus infection, and the mesna and the hydration alkalization were used as the preparative regimen for the hemorrhagic cystitis. Implantation was detected by DNA analysis of short tandem repeat polymorphism. Results Two children who received BMT and 1 child who received allo -PBSCT were completely implanted; one child who received BMT chimera was also implanted. The median time to achieve an acute neutral granulocyte 〉0.5 ×109L-1was 12 days （9 - 15 days） ,the median day to achieve a platelet count 〉 20 ×109L-1 was 19 days （ 12 -30 days）. Conclusions allo - HSCT is an effective therapy on SAA. At present, maintaining hematopoietic function ＇after stein cell transplantation, preventing and controlling complications are the focus of the study.