异基因外周造血干细胞移植治疗儿童重型再生障碍性贫血

目的探讨异基因外周血造血干细胞移植(allo—PBSCT)对儿童重型再生障碍性贫血(SAA)的疗效。方法8例接受allo—PBSCT治疗的SAA 患儿均为血缘供体移植，男6例，女2例，7～14岁，中位年龄为11岁。4例患儿为急性SAA I型，其中2例移植前合并重型感染且未能完全控制；另4例患儿为SAA Ⅱ型，患儿移植前大多经包括环孢菌素A(CSA)在内的多种治疗无效。诊断至移植的时间为1～45个月，中位时间为13个月。预处理方案由低剂量环磷酰胺（CTX）和抗淋巴细胞球蛋白（ALG）或抗胸腺细胞球蛋白（ATG）组成，采用CsA+短程甲氨蝶呤(MTX)方案预防移植物抗宿主病(GVHD)。以DNA短串联重复序列多态性分析(STR PCR)检测学植入依据。结果患者移植后均获造血重建，中性粒细胞绝对值≥0.5×109／L和血小板计数≥20×109／L的中位时间分别为移植后+12.75d(+10～+15d)和+19.88d(+15～+32d)。8例患儿均为完全供体型植入，其中1例移植后10个月出现外周血白细胞、红细胞、血小板计数逐渐下降，STR PCR显示移植排斥，经ATG、CsA 等药物治疗逐渐恢复自身造血。1例患儿出现Ⅱ度急性GVHD，1例出现局限性慢性GVHD。随访15～50个月，中位数为29个月，8例SAA患儿均存活。结论采用以免疫抑制为主的ATG/ALG+CTX预处理方案进行allo—PBSCT能够有效治疗儿童SAA，移植相关并发症少，值得临床推广应用。

Effects of allogeneic peripheral blood stem cell transplantation for severe aplastic anemia in childpatients

Objetive To investigate the effects of allogeneic peripheral blood stem cell transplantation (allo-PBSCT) for severe aplastic anemia(SAA) in children. Methods Eight child patients, six boys and two girls, with a median age of 11 years (range:7-14) with SAA (four patients with SAA-Ⅰ and four with SAA- Ⅱ ) received allo-PBSCT of related donors. Most in this group had been treated with CSA and other treatments which proved to be ineffective before transplantation. The period from diagnosis to transplantation was 1-45 months, with a median time of 13 months. Being conditioned with a decreased dosage of immu-nosuppressive agents of CTX and anti-lymphocyte globulin( ALG) or anti-thymocyte globulin(ATG), patients were given CsA and MTX to prevent graft-versus-host disease (GVHD).The engraftment was identified by means of STR-PCR. Results All acquired fast reconstruction was with full donor ceD grafting. The median periods for neutrophil ≥0.5 × 10~9/L and platelet ≥20×10~9/L were 12.75 (10-15)and 19.88( 15-32) days, respectively. One patient had a decreased pancytopenia 10 months after transplantation; STR-PCR indicated graft rejection. This patient was treated with ATG and CsA, and then hematopoiesis of the patient gradually recovered. One patient developed grade 11 aGVHD and one developed limited cGVHD. All the patients survived with a median period of 29 months (15- 50) at the end of follow-up. Conclusion Allo-PBSCT with a conditioning regimen of ATG plus CTX is an effective therapy with few complications for child patients with SAA and is worth popularizing in clinical practice.