This study was aimed to investigate the therapeutic efficacy of rabbit anti-thymocyte globulin (r-ATG) combined with cyclosporine A (CsA) and to analyse the efficacy-related factors in children with aplastic anemia (AA). Twenty five AA children treated with r-ATG [3. 5 mg/( kg · d) × 5 days] combined with CsA were analyzed retrospectively. The lymphocyte subgroups, CD4 + /CD8 ratio and expression of CD55, CD59 on surface of neutrophils and erythrocytes in peripheral blood were detected by direct immunofluorescence method and flow cytometry; the responsive time, effective rate, adverse effects and infections after immunosuppressive therapy (IST) were analyzed; the distribution of T-lymphocyte subgroups in IST-effective and IST-unefective groups was compared, and therapeutic efficacy-related factors were evaluated. The results showed that the response to treatments was found in 21 out of 25 cases, the total responsive rate was 84. 0% ; the response time was 3-6 months, average of 4 months; the effective rates in month 3,6,9, 12 after treatment were 56. 0% , 72. 0% , 80.0% and 84. 0% respectively. The AA children with age ≥5 years old, course of disease <6 months and absolute neutrophil value ≥1.5 × 109/L on 30 days after IST had good curative effect; the effective rate in AA children with age ≥5 years old, course of disease <6 months, high or reverse ratio of CD4+ /CD8+ and absolute neutrophil value ≥ 1.5 × 109/L after IST was higher than that in AA children with age<5 yeaars old, conrse of disease ≥6 months, normal ratio of CD4/CD8+ and absolute neutrophil value after IST < 1. 5 × 109/L (94.4% vs 57.1 % , 90.4% vs 50.0% , 94.1 % vs 62.5% , 94.1 % vs 62. 5% ) (P <0. 05 ). The high effective rate was observed in AA children with decrease of CD55 and CD59 expression, but there was no significant difference (P>0. 05) as compared with normal expression of CD55,CD59. It is concluded that the treatment using r-ATG (3.5 mg/kg · d × 5 d) combined with CsA is a safe and effective for children with AA. Age, course of disease and absolute neutrophil value on 30 days after IST are the main factors affecting curative affect.%本研究探讨兔源抗胸腺细胞球蛋白(r-ATG)联合环孢菌素(CsA)治疗儿童再生障碍性贫血(AA)的疗效并对疗效相关性因素进行分析.以应用r-ATG(剂量为3.5 mg/(kg·d)×5d]联合CsA治疗的25例AA患儿为对象,采用直接免疫荧光法和流式细胞术检测外周血淋巴细胞亚群及CD4+/CD8+比值和外周血粒细胞及红细胞膜表面CD55、CD59的表达情况,观察免疫抑制治疗(IST)起效时间、有效率、不良反应及感染发生情况,IST有效组和无效组T淋巴细胞亚群分布情况,并比较及分析与疗效相关的因素.结果表明,25例中对治疗有反应者21例,总反应率为84.0％,起效时间为3-6个月,平均为4个月；r-ATG联合CsA治疗后第3、6、9、12月末的有效率分别为56.0％、72.0％、80.0％和84.0％.年龄≥5岁、病程＜6个月、CD4+/CD8+比例超高或比例倒置和IST治疗后30 d中性粒细胞绝对值≥1.5×109/L的患儿,其IST治疗有效率高于年龄＜5岁、病程≥6个月、CD4+/CD8+比例正常和IST治疗后30 d中性粒细胞绝对值＜1.5×109/L者(94.4％ vs 57.1％;90.4％ vs 50.0％;94.1％ vs 62.5％；94.1％ vs 62.5％),两组比较差异均有统计学意义(P＜0.05).CD55、CD59表达降低患儿的IST治疗有效率较高,但与CD55、CD59表达正常相比无明显差别(P＞0.05).结论:r-ATG[剂量为3.5 mg/(kg·d) ×5 d]联合CsA治疗是治疗儿童AA的一种安全有效的方法,对年龄≥5岁、病程＜6个月、CD4+/CD8+比例异常的AA患儿IST治疗疗效较好,对IST治疗后30 d中性粒细胞绝对值≥1.5×109/L的AA患儿疗效亦较佳,年龄、病程长短和CD4+/CD8+比值可作为预测AA患儿IST疗效的指标.
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