免疫抑制治疗获得性重型再生障碍性贫血患儿疗效分析

目的 分析免疫抑制治疗儿童获得性重型再生障碍性贫血(severe aplastic anemia,SAA)的近远期疗效.方法 回顾性分析2000年1月至2006年6月在我院应用联合免疫抑制治疗的获得性重型再生障碍性贫血患儿.112例患儿随机分3组:Ⅰ组(26例):单用环孢素A(CSA)组;Ⅱ组(30例):CSA+丙种球蛋白[400 mg/(kg·d)×5 d];Ⅲ组(56例):兔抗胸腺细胞球蛋白(R-ATG)[3～5 mg(kg·d)×5 d]+CSA.所有患儿治疗均同时加用司坦唑醇或丙酸睾丸酬.CSA血药浓度调整到谷浓度100 ug/L以上,峰浓度300 ug/L以上.结果Ⅰ组免疫抑制治疗的总反应率为26.92%;Ⅱ组免疫抑制治疗的总反应率为33.33%;Ⅲ组免疫抑制治疗的总反应率为62.5%,明显高于Ⅰ组(P=0.001);比较Ⅰ组与Ⅱ组的总反应率差异无统计学意义.Ⅰ、Ⅱ和Ⅲ组5年总生存率分别为(20.50±15.41)%、(39.77±9.77)%和(66.27±6.84)%.结论对无HLA匹配同胞供者的重型获得性再生障碍性贫血患儿ATG联合CSA是最理想的治疗方法 .

Efficacy of immunosuppresive therapy in children with acquired aplastic anemia

Objective This study was designed to evaluate the efficacy of immunosuppressive therapy (IST) regimens as treatment of children with acquired severe aplastic anemia(SAA). Methods Data of consecutive 112 children with SAA who had no HLA-matched sibling seen from January 2000 to June 2006 were retrospectively analyzed. The patients were randomized to receive one of the following IST regimens: cyclosporine A (CSA) alone(IST regimen Ⅰ);CSA and intravenous immunoglobulin (IVIG) [400 mg/(kg·d)×5 d] (IST regimen Ⅱ);rabbit anti-T-lymphocyte globulin (R-ATG)[3-5mg/ (kg·d)×5 d] and CSA (IST regimen Ⅲ). No repeated courses of R-ATG were given for nonresponders. All the patients also received stanozolol or testosterone propionate. The dose of CSA was adjusted to maintain trough drug levels above 100 ug/L and peak drug levels above 300 ug/L Results The overall rate of response to IST regimen Ⅰ was 26.92% and to IST regimen Ⅱ was 33.33%. The response to IST regimen Ⅲ (62.5% ) was significantly higher (P=0.001). The response to IST regimen Ⅰ and IST regimen Ⅱ had no significient difference. The 5-year overall survival for IST regimens Ⅰ, Ⅱ, and Ⅲ was 20.50%± 15.41%, 39.77%±9.77%, and 66.27%±6.84%, respectively. Conclusion If patients had no HLA-matched sibling, the combination of R-ATG and CSA remains the best combination for the treatment of children with SAA, providing a survival advantage at 5 years.