FC预处理方案异基因外周血干细胞移植治疗重型再生障碍性贫血  

Allogeneic hematopoietic stem cell transplantation using Flu/CTX conditioning regimen for severe aplastic anemia

作　　者：王三斌[1] 胡灯明[1] 彭利晖[1] 刘林[1] 谢政军[1] 尹波[1] 孙晓娟[1] 

机构地区：[1]成都军区昆明总医院血液科,云南昆明650032

出　　处：《中国医师杂志》2008年第7期892-894,共3页Journal of Chinese Physician

摘　　要：目的探索氟达拉滨（Flu）＋环磷酰胺（CTX）预处理方案（Fc预处理方案）异基因造血干细胞移植治疗重型再障的可行性。方法9例重型再障患者接受HLA全相合并基因造血干细胞移植。预处理方案采用FC方案：即氟达拉滨（Flu）30mg／m^2，静脉滴注，1／d，d-6-d-2，共5d；环磷酰胺（CTX）50mg／kg，静脉滴注，1／d，d-5-d-2，共4d。环孢素A（CsA）＋骁悉（MMF）联舍方案预防移植物抗宿主病（GVHD）。结果9例患者均成功植入，中性粒细胞≥0．5×10^9／L的时间中位时间为12（10-16）d，PLT≥20×10^9／L的时间中位时间为14（12-19）d。移植后30d检测证实均为供者型完全嵌合状态。1例发生肠道Ⅱ度急性GVHD，1例发生皮肤I度急性GVHD，1例出现慢性GVHD，Ⅱ度以上（含Ⅱ度）急性GVHD总发生率仅为11．1％。中位随访时间39个月，9例患者均无病存活。结论Fc预处理方案植入率高，预处理相关并发症少，无病生存率高，临床疗效好。Objective To evaluate the efficacy of Flu/CTX conditioning regimen for the treatment of severe aplastic anemia in patients receiving allogeneic hematopoietic stem cell transplantation. Methods Nine patients with severe aplastic anemia received HLA identical peripheral blood hematopoietic stem cell transplantation （PBSCT） using Flu/CTX conditioning regimen, which consisted of fludarbine [30 mg/（m^2·d） for 5 days （ -7 to -3） ], CTX [50mg/（kg·d） for 4 days（ - 5 to -2） ]. All patients received cyclosporin A （CsA） and mycophenolet mofetil （MMF） for prophylaxis of graft-versus-host disease（GVHD）. Results The Flu/CTX regimen was very well tolerated, with no severe regimen related toxicity. In all patients, the median days of neutrophil exceeding 0.5×10^9/L and platelet exceeding 20×10^9/L were 12 days （range 10-16 days） and 16 days （range 14-19 days）, respectively. Complete chimerism was achieved in all pa- tients at one month after PBSCT. Two patients had acute GVHD and one had chronic GVHD. In the 39-month median follow-up duration, all patients were alive in disease-free situation. Conclusion The Flu/CTX conditioning regimen may reduce transplantation-related toxicities and can achieve full chimerism and high long-term disease-free survival. Allogeneic hematopoietic stem cell transplantation using intravenous Flu/CTX conditioning regimen is a safe and effective treatment method for the patients with severe aplastic anemia.

关 键 词：干细胞移植 环磷酰胺 阿糖腺苷 贫血 再生障碍性/治疗 

分 类 号：R556[医药卫生—血液循环系统疾病]