替代供者异基因造血干细胞移植治疗重型再生障碍性贫血19例疗效分析

Outcome analysis of alternative donor allogeneic hematopoietic cell transplantation in the treatment of 19 severe aplastic anemia patients

目的评价替代供者异基因造血干细胞移植（ADallo-HSCT）治疗重型再生障碍性贫血（SAA）患者的疗效。方法回顾性分析我院2003年5月至2012年12月接受ADallo-HSCT治疗的19例SAA患者临床资料，其中接受亲缘半相合供者移植12例，无关供者移植7例；预处理方案为环磷酰胺＋抗人胸腺细胞球蛋白＋氟达拉滨士阿糖胞苷土白消安或马法兰；移植物抗宿主病（GVHD）预防方案采用环孢素或他克莫司、短疗程甲氨蝶呤、霉酚酸酯三药联合应用；异基因骨髓移植组患者回输CD34＋细胞中位数3．10（2．11-4．38）×10^6／kg；异基因外周血干细胞移植组患者回输CD34＋细胞中位数4．90（2．08～6．88）×10^6／kg。结果19例患者移植后均获得造血重建，其中12例发生急性GVHD，7例发生慢性GVHD，1例出现移植物排斥。所有患者移植后中位随访13（3-115）个月，13例生存，预期5年总体生存率为（67．5~11．0）％。结论ADallo—HSCT可以作为无HLA匹配同胞供者SAA患者的一种替代治疗手段。

Objective To evaluate the efficacy of alternative donor allogeneic hematopoietic stem cell transplantation （AD allo-HSCT） in the treatment of severe aplastic anemia （SAA）. Methods Retrospective analysis of the clinical data of 19 SAA patients received AD aIlo-HSCT from May 2003 to December 2012. Of them, 12 received haploidentical H SCT （haplo-HSCT）, 7 received unrelated donor transplantation. The conditioning regimen was CY＋ATG＋Flu＋Ara-C＋Bu/Mel, the GVHD preventing regimen was MMF＋MTX＋CSA/FKS06; the median reinfusion quantity of CD34＋ was 3. 10 （2.11-4.38）× 10^6/kg in allo- BMT and 4.90 （2.08-6.88） × 10^6/kg in allo-PBSCT. Results Hematopoiesis reconstitution was achieved in all 19 patients. Twelve patients developed acute graft-versus-host disease （aGVHD）, and 7 developed chronic GVHD （cGVHD）. Graft rejection （GR） was occurred in one patient. The median follow-up time was 13 （3-115） months. Thirteen patients survived, and the prospective 5-year overall survival rate is （67.5± 11.0）%. Conclusion AD allo-HSCT can be used as an alternative therapy for SAA patients without HLA matched sibling donor.