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

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。

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

Objective To evaluate the efficacy of Flu/CTX conditioning regimen for the treatment of severe aplastic anemia in pa- tients receiving allogeneic hematopoietic stem cell transplantation. Methods Nine patients with severe aplastic anemia received HLA identi- cal peripheral blood hematopoietic stem cell transplantation (PBSCT) using Flu/CTX conditioning regimen, which consisted of fludarbine [30 mg/(m2 d) for5 days (-7 to -3) ], CTX [50mg/(kg d) for4 days(-5 to-2)]. All patients received cyclosporin A (CsA) and mycophenolet mofetil (MMF) for prophylaxis of graft-versus-host disease(GVHD). Results The Fiu/CTX regimen was very well toler- ated, with no severe regimen related toxicity. In all patients, the median days of neutrephil exceeding 0. 5×109/L and platelet exceeding 20 ×109/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 Fiu/CTX conditioning regimen is a safe and effective treatment method for the patients with severe aplastic anemia.