BU-CTX_2预处理方案异基因造血干细胞移植治疗白血病60例

BU-CTX_2 as conditioning regimen for allogeneic hematopoietic stem cell transplantation in sixty patients with leukemia

目的 评价BU CTX2 预处理方案异基因造血干细胞移植 (allo HSCT)治疗 6 0例白血病的长期疗效。方法 1994年 4月至 2 0 0 0年 8月 6 0例白血病患者接受了allo HSCT ,其中急性髓系白血病 (AML) 2 0例 ,急性淋巴细胞白血病 (ALL) 15例 ,慢性髓系白血病 (CML) 2 5例。 5 3名供者系HLA完全相合同胞 ,4名为HLA 1个主要位点不合同胞 ,3名为HLA完全相合无关供者。用BU CTX2 方案预处理 ,用环孢菌素A +甲氨蝶呤 (5 4例 )或甲泼尼龙 (6例 )预防移植物抗宿主病 (GVHD)。结果 6 0例均植活。 2 2例 (36 .7% )发生急性GVHD ,其中CML组为 4 8.0 % ,AML组 30 .0 % ,ALL组则为 2 6 .7%。中位随访时间 2 4 (9～ 2 4 )个月 ,38例仍无白血病生存 ,2 2例 (36 .7% )死亡 ,其中 1例死于肺部感染 ,3例死于急性GVHD ,7例死于巨细胞病毒感染 ,11例死于白血病复发 ,其中AML 3例 (15 .0 % ) ,ALL 8例(5 3 3% )。 8例ALL均于移植早期复发死亡。 4例ALL长期生存者均发生慢性GVHD。 3年无病生存(DFS)率为 6 3.3% ,其中CML组为 80 .0 % ,AML组 70 .0 % ,ALL组则为 2 6 .7%。结论 BU CTX2 为AML和CML的有效预处理方案 ,白血病复发率低 ,长期生存率高 ,而作为ALL的预处理方案则白血病复发率较高 ,提示BU CTX2 不适合作为ALL首选预处?

Objective To evaluate the long term outcome of 60 leukemia patients undergoing allogeneic hematopoietic stem cell transplantation (allo HSCT ) prepared with busulphan cyclophosphamide (BU CTX 2) conditioning regimen. Methods From April 1994 to August 2000, 60 leukemia patients ( 35 male, 25 female; median age 32 years old), including 20 acute myeloid leukemia (AML,CR 1 n=19,CR 2 n=1), 15 acute lymphocytic leukemia (ALL,CR 1 n=8, CR 2 n=6, CR 3 n=1), and 25 chronic myeloid leukemia (CML,CP 1 n=24, CP 2 n=1) received allogeneic hematopoietic stem cells from HLA identical siblings (n=53),1 locus mismatched siblings (n=4), or HLA identical unrelated donor (n=3). BU CTX 2 was used as conditioning regimen. All patients received cyclosporine A and either methotrexate (n=54) or methylprednisolone (n=6) for graft versus host disease (GVHD) prophylaxis. Results All 60 patients got sustained engraftment. Acute GVHD(aGVHD) occurred in 22 patients (36.7%), while the incidence of aGVHD was 48.0% for the CML, 30.0% for the AML and 26 7% for the ALL patients. Thirty eight patients are still alive in complete remission with a median follow up of 30 (12～84) months and 22 died. The main causes of death were aGVHD in 3, CMV IP in 7, and relapse in 11 patients. The remaining one died of pulmonary infection. Among 11 patients who died of relapse, 8 were ALL relapsed in the early posttransplant stage. All 4 long term survivors of ALL developed chronic GVHD. The probability of DFS at 3 year for CML, AML and ALL patients was 80.0%, 70.0% and 26.7%, respectively. Conclusion BU CTX 2 is an effective conditioning regimen for patients with AML and CML, resulting in a low relapse and high long term survival rate. However, it is not effective enough for patients with ALL, because of a higher incidence of relapse.