造血干细胞移植治疗重型再生障碍性贫血  

Hematopoietic stem cell transplantation for aplastic anemia

作　　者：曹星玉[1] 吴彤[1] 卢岳[1] 赵艳丽[1] 周葭蕤 魏志杰[1] 刘德琰[1] 熊敏[1] 张建平[1] 陆道培[1] 

机构地区：[1]河北燕达医院陆道培血液肿瘤中心,河北省廊坊市065201

出　　处：《中华器官移植杂志》2013年第2期71-74,共4页Chinese Journal of Organ Transplantation

摘　　要：目的探讨不同类型的造血干细胞移植（HSCT）治疗重型再生障碍性贫血（sAA）的预处理方案、预后及并发症发生情况等。方法回顾性分析2001年9月至2010年11月间41例sAA患者接受HSCT的病例资料及其临床特点，移植类型包括同基因移植6例，同胞全相合移植8例，非亲缘移植18例，亲缘半相合移植7例，自体脐带血移植1例及非亲缘脐带血移植1例。所有受者均采用以环磷酰胺＋抗胸腺细胞球蛋白为主的预处理方案，其中23例加用氟达拉滨（Flu），2例加用全身照射，5例加用Flu和全身照射，1例加用Flu和白消安。结果移植后白细胞和血小板均植活的受者共有38例，其中6例（15．8％）发生排斥反应，分布于同基因移植、同胞全相合移植、非亲缘移植和亲缘半相合移植等多种移植类型；4例发生Ⅲ～Ⅳ度aGVHD，分别为同胞全相合移植1例，非亲缘移植1例，亲缘半相合移植2例；25例发生巨细胞病毒（CMV）血症，8例发生EB病毒（EBV）血症，其中6例为非亲缘移植，1例同胞全相合移植及1例亲缘半相合移植，发生EBV感染的受者均伴有CMV感染。所有受者5年预期存活率为75％，其中同基因移植、同胞全相合移植及非亲缘移植受者的5年预期存活率分别为75％、100％和70％，亲缘半相合移植受者的4年预期存活率57％。结论sAN患者接受同胞全相合移植后长期存活率高，可以作为sAA的首选治疗方式。而非亲缘移植和亲缘半相合移植的预后还有改善的空间，需要通过选择合适的供者和预处理方案来降低移植后相关并发症的发生率，从而改善预后。Objective To study complications and prognosis about sAA-HSCT in order to improve the survival of the patients with sAA. Methods Clinical characteristics of 41 patients with sAA who underwent HSCT between September 2001 and November 2010 were retrospectively analyzed. The patients received stem cells from syngeneic siblings （n = 6）, identical siblings （IS, n = 8）, unrelated donors （URD, n = 18） ： haploidentical family members （Haplo, n = 7）, unrelated cord blood （n = 1 ）, or autologous cord blood （n = 1）. All patients received conditioning regimen including cyclophosphamide and antithymocyte globulin （Cy/ATG）. Patients received Cy/ATG only （n = 10）, Cy/ATG plus fludarabine （n = 23）, Cy/ATG plus TBI （n = 2）, Cy/ATG plus fludarabine and TBI （n = 5） or Cy/ATG plus fludarabine and busulfan （n = 1）. Results Thirty-eight patients had neutrophil and platelet recovery. Secondary graft rejection occurred in 15. 8% of patients. Four patients had acute GVHD of grade Ⅲ or Ⅳ. CMV was diagnosed in 25 cases and 8 of them had EBV viremia. The estimated 5-year overall survival （OS） for all patients was 75%. Probability of 5-year OS in patients subject to syngeneic sibling HSCT, identical sibling HSCT and URD-HSCT was 75%, 100% and 70% respectively. The 4-year OS in patients who underwent haplo-HSCT was 57%. Conclusion IS- HSCT is a preferred option for sAA. We need to do more to improve the outcome of AA-HSCT with URD or haplo-identical donors.

关 键 词：贫血 再生障碍性 造血干细胞移植 

分 类 号：R556.5[医药卫生—血液循环系统疾病]