替代供者异基因造血干细胞移植治疗范可尼贫血五例分析  

Outcomes of alternative donor allogeneic hematopoietic stem cell transplantation for Fanconi anemia： a five cases report

作　　者：汪文静 孙于谦[1] 唐菲菲[1] 韩婷婷[1] 莫晓冬[1] 王景枝[1] 张晓辉[1] 黄晓军[1] 许兰平[1] 

机构地区：[1]北京大学人民医院北京大学血液病研究所造血干细胞移植治疗血液病北京市重点实验室,100044

出　　处：《中华内科杂志》2018年第1期54-56,共3页Chinese Journal of Internal Medicine

基　　金：国家自然科学基金重点项目（81530046、81370666）

摘　　要：回顾性分析采用氟达拉滨（FLU）＋环磷酰胺（CY）＋抗胸腺免疫球蛋白（ATG）,即FCA预处理方案,并行替代供者造血干细胞移植治疗范可尼贫血的疗效及安全性.2例采用单倍体相合骨髓联合外周血干细胞移植,3例采用非血缘供者外周血干细胞移植.患者耐受性良好,移植后均达到迅速造血重建,1例死于颅内感染,4例存活者均已脱离输血支持且保持完全供者嵌合.Five patients with Fanconi anemia who received hematopoietic cell transplantation were retrospectively analyzed. The conditioning regimens included fludarabine, cyclophosphamide and anti-thymocyte globulin. Two patients received both bone marrow and peripheral blood stem cells as the source of stem cell grafts from haploidentical matched related donors, while the others received peripheral blood stem cells from unrelated donors.All patients tolerated well and reached hematopoietic reconstitution. One patient died of intracranial infection.During follow-up,4 patients survived independent of transfusion with full donor chimerism.

关 键 词：范可尼贫血 造血干细胞移植 单倍体 非血缘供者 

分 类 号：R556[医药卫生—血液循环系统疾病]