重型β地中海贫血异基因造血干细胞移植中不同剂量ATG-F的比较研究  

Pretreatment doses of antithymocyte globubin-fresenius for allogeneic hematopoietic stem cell transplantation for beta-thalassemia major

作　　者：李春富[1] 王彦华[1] 吴学东[1] 裴夫瑜[1] 何岳林[1] 冯晓勤[1] 刘华颖[1] 

机构地区：[1]南方医科大学南方医院儿科,广东广州510515

出　　处：《南方医科大学学报》2012年第5期691-694,共4页Journal of Southern Medical University

基　　金：广东省科技计划项目(2011B031800280)

摘　　要：目的探讨不同剂量的抗人T淋巴细胞兔免疫球蛋白(ATG-F)在重型地中海贫血造血干细胞移植中的应用。方法将重型β地贫患儿进行异基因造血干细胞移植的预处理方案ATG-F分为2个剂量组(低剂量15 mg/kg组和高剂量30 mg/kg组)进行比较分析。预处理方案主要包括环磷酰胺,白消安,氟达拉宾赛替哌,以及ATG-F。结果两组在中性粒细胞及血小板植入时间上无差别。II-IV的急性移植物抗宿主病发生率在低剂量组和高剂量组分别为12.5%,9.4%,低剂量组有更高的发生趋势,III-IV的急性移植物抗宿主病在低剂量和高剂量分别是12.5%和6.3%,但无统计学差异。高剂量的ATG-F组临床拟诊真菌感染患儿较低剂量组显著高(P<0.05)。低剂量组和高剂量组2年的总的生存率分别是90.6%、93.8%。结论两组不同剂量的ATG-F作为重型β地中海贫血造血干细胞移植的预处理是可行的。Objective To investigate the effects of different doses of antithymocyte globubin-fresenius（ATG-F） for allogeneic hematopoietic stem cell transplantation（allo-HSCT） in patients with beta-thalassemia Major.Methods Sixty-four children with beta-thalassemia major undergoing allo-HSCT were divided into two equal groups to receive ATG-F pretreatments at high（30 mg/kg） or low（15 mg/kg） doses as part of the conditioning regimen including mainly cyclophosphamide,busulfan,fludarabine,and thiotepa.The outcomes of the patients were compared between the two groups.Results No obvious difference were noted in the time to leukocyte and platelet engraftment between the two groups.The incidence of grade II-IV acute graft-versus-host disease（aGVHD） appeared to be higher in the low-dose group than in the high-dose group（12.5% vs 9.4%）.The incidence of grade III-IV aGVHD was also higher in the low dose group（12.5% vs 6.3%）,but the difference was not statistically significant.Application of high-dose ATG-F was associated with a higher rate of probable and possible fungal infection（P0.05）.Conclusion The two doses of ATG-F is feasible as a part of the conditioning regimen for allo-HSCT in children with beta-thalassemia major.

关 键 词：重型地中海贫血 造血干细胞移植 ATG-F 移植物抗宿主病 

分 类 号：R556.61[医药卫生—血液循环系统疾病]