静脉滴注白消安和氟达拉滨预处理方案行异基因造血干细胞移植治疗髓系血液病疗效观察

The clinical research of allogeneic hematopoietic stem cell transplantation using intravenous Bu/Flu conditioning regimen in the treatment of myeloid hematopathy

目的探讨静脉滴注白消安(Bu)和氟达拉滨(Flu)作为预处理方案,进行异基因造血干细胞移植治疗髓系血液病的疗效。方法选取2003年10月至2005年4月成都军区昆明总医院血液科髓系血液病患者9例,其中急性非淋巴细胞白血病(ANLL)3例,慢性粒细胞白血病(CML)5例,骨髓增生异常综合征(MDS)1例,均进行同胞白细胞抗原(HLA)全相合异基因造血干细胞移植。预处理方案采用移植前第6天至移植前第3天静脉滴注白消安3.2mg/(kg.d),共4d;移植前第6天至移植前第2天静脉滴注氟达拉滨30mg/(m2.d),共5d。环孢素A和霉酚酸酯(骁悉)联合应用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>0.5×109/L的平均时间为12d;血小板(PLT)>20×109/L的平均时间为14d。中位观察时间为31个月。除轻微胃肠道反应外,无严重的预处理相关毒性,移植后1个月检测证实均为供者型完全植入。发生急性GVHD2例,慢性GVHD1例。9例患者中8例无病存活。结论静脉滴注Bu/Flu预处理方案,移植相关毒性小,治疗髓系血液病安全有效。

Objective To evaluate the efficacy of intravenous Bu/Flu conditioning regimen for the treatment of myeloid hematopathy in patients receiving allogeneic hematopoietic stem cell transplantation. Methods Nine patients with acute nonlymphocytic leukemia （ ANLL, n = 3 ） , chronic myelogenous leukemia （ CML, n = 5 ） , and myelodysplastic syndrome （MDS ,n = 1 ）were treated by allogeneic hematopoietic stem cell transplantation using intravenous Bu/Flu conditioning regimen, which consisted of busulfex [ 3. 2 mg/（ kg· d ） for 4 days ], and fludarabine [ 30 mg/（ m^2 ·d ） for 5 days ]. All patients received cyclosporin A （ CsA ） and mycophenolet mofetil （ MMF ） for prophylaxis of graft-versus-host disease （GVHD）. Results The intravenous Bu/Flu regimen was very well tolerated, with no severe regimen related toxicity. In all the 9 patients absolute neutrophil count 〉 0. 5 × 10^9/L was achieved in 10 to 16 （ median 12 ） days and platelet count 〉 20 ×10^9/L in 12 to 19（ median 14 ）days after transplantation. Complete chimerism was achieved in all the patients at one month after HSCT. Two patients had acute GVHD and one had chronic GVHD. With a median follow-up of 31 （19 -37 ）months ,8 of 9 patients were alive and dlsease-free. Conclusion The intravenous Bu/Flu 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 Bu/Flu conditioning regimen is a safe and effective option for the patients with myeloid hematopathy.