抗胸腺细胞球蛋白联合环孢菌素A治疗儿童再生障碍性贫血疗效及相关因素分析

摘要

Objective To analyze the influential factors of immunosuppressive therapy (1ST) with antithymocyte globulin ( ATG) and cyclosporine ( CSA) in pediatric aplastic anemia ( AA) patients. Methods Forty children diagnosed as A A received 1ST with ATG and CSA. The correlation between response rate and many clinical factors including peripheral blood cell counts and absolute lymphocyte counts (ALC) after treatment, the incidence of serum sickness and so on. Results The duration of follow-up was 9 ~44 months (19 months in median). The complete response rate and overall response rate were 45% and 78% respectively. The ALCs in all patients decreased significantly after 1ST. The response rate in patients with ALCs-decrease more than 2 x 10 /L was higher than those with ALCs-decrease less than 2 × 109/L (89% vs 54% , P < 0. 05). Besides, The response rate in patients who received 1ST in 6 months since diagnosis as AA was higher than those who received 1ST after 6 months(92% vs 54% , P < 0. 05 ). There was not difference in patients treated with different kinds of ATG (Genzyme or Fresenius). Conclusions 1ST with ATG and CSA is effective and safe for pediatric AA patients. Patients treated with two common ATG agents could receive the same effect, and those who receive 1ST in 6 months could get better response. It is necessary to measure ALC during the treatment of ATG. Moreover, Further research is needed to determine whether we should increase ATG dose for patients with unobvious ALC decrease.%目的 探索采用抗胸腺细胞球蛋白( ATG)联合环孢菌素A(CSA)的免疫抑制疗法(IST)治疗儿童再生障碍性贫血(再障)的疗效及其相关影响因素,为进一步提高临床疗效提供参考依据.方法 共40例再障患儿(重型再障28例,依赖成分输血的慢性再障12例)接受ATG联合CSA治疗.统计分析治疗前病程和外周血三系下降程度；ATG治疗后外周血淋巴细胞绝对计数(ALC)下降程度、血清病发生率及不同制剂ATG治疗等临床因素与远期疗效的相关性.结果 中位随访时间19(9 ～44)个月,总有效率和显效率分别为78％和45％.疗效相关统计分析显示:(1)ATG治疗后2周内,ALC下降幅度≥2×109/L者的有效率明显高于下降幅度＜2×109/L者,两组总有效率分别为89％和54％ (P ＜0.05).(2)从确诊再障到接受ATG治疗,病程≤6个月者有效率明显高于病程＞6个月者,两组总有效率分别为92％和53％(P＜0.05).(3)采用两种ATG制剂(美国Genzyme或德国Fresenius)各治疗18例和22例,两组疗效差异无显著性(P＞0.05).结论 ATG联合CSA的免疫抑制治疗是儿童再障的有效疗法,目前常用的两种ATG制剂,均能获得显著疗效.再障确诊后早期治疗有助于提高疗效.治疗期间密切观察ALC下降程度,对于ALC下降不明显者,是否需要适当增加ATG剂量,有待进一步研究论证.