探讨强化免疫抑制治疗(immunosuppressive therapy，IST)与单用环孢菌素A(cyclosporine A，CSA)治疗儿童重型再生障碍性贫血(severe aplastic anemia，SAA)间疗效差异，并研究强化免疫抑制治疗前，患儿对粒细胞集落刺激因子(granulocyte colony stimulating factor，G–CSF)反应程度与疗效的关系。

2000年1月至2007年11月在中山大学附属第二医院儿科确诊的重型再生障碍性贫血患儿为68例，其中55例采用强化免疫抑制治疗(IST组)，13例因经济原因采取单用环孢菌素A治疗(CSA组)，比较两组疗效差异(本研究遵循的程序符合中山大学附属第二医院人体试验委员会所制定的伦理学标准，得到该委员会批准，分组征得受试对象的知情同意，并与试验患儿监护人签署临床研究知情同意书)。24例重型再生障碍性贫血患儿在强化免疫抑制治疗前做粒细胞集落刺激因子试验性治疗，根据对粒细胞集落刺激因子的反应程度，将IST组分为G–CSF反应IST亚组(n＝13)与G–CSF无反应IST亚组(n＝11)，比较两亚组强化免疫抑制治疗后6个月及长期疗效差异。

IST组和CSA组患儿治疗的平均显效时间分别为3.4个月(1～13个月)和4.5个月(1～15个月)。IST组和CSA组患儿患儿治疗后第1、第3、第6、第9及第12个月末的有效率分别为70.9%，58.2%，79.5%，87.2%和89.7% vs.46.2%，38.5%，46.2%，61.5%和61.5%。随访1年以上，IST组有效率为89.7%(35/39)，CSA组患儿有效率为61.5%(8/13)，两组比较，差异有显著意义(χ²＝5.419，P<0.05)。G–CSF反应IST亚组及其无反应IST亚组患儿治疗6个月后，有效率分别为76.9%(10/13)和45.6%(5/11)，两亚组比较，差异有显著意义(P<0.05)。G–CSF反应IST亚组患儿总有效率(92.3%，12 /13)较无反应IST亚组(54.5%，6/11)高，且差异有显著意义(P<0.05)。

强化免疫抑制治疗重型再生障碍性贫血的疗效明显优于单用环孢菌素A。强化免疫抑制治疗前，对粒细胞集落刺激因子有反应的患儿，强化免疫抑制治疗的预后相对较好。

To analyze the therapeutic efficacy of intensive immunosuppressive therapy(IST) in children with severe aplastic anemia(SAA) and compared it with only cyclosporine A(CSA) therapy, and evaluate the relation between granulocyte colony stimulating factor (G-CSF) reaction and therapeutic efficacy before intensive immunosuppressive therapy.

The study cohort consisted of 68 consecutive patients diagnosed as severe aplastic anemia in the Department of Pediatrics, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University between January 2000 and November 2007. Fifty-five cases of 68 patients accepted intensive immunosuppressive group (IST group), while other 13 cases received only cyclosporine A (CSA group) because of poor economy. The different therapeutic efficacy of these two regimens was analyzed. The procedure of this study was consistent with ethical standard established by the committee of investigation in human beings of Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, and it was approved by this committee. Informed consent was obtained from all participates.24 cases of severe aplastic anemia received granulocyte colony stimulating factor as experimental treatment before intensive immunosuppressive therapy according to the degree of peripheral blood count's response to granulocyte colony stimulating factor. These 24 cases were divided into reaction sub-IST group (n=13)and non-reaction sub-IST group.(n=11). The differences of therapeutic efficacy at the end of 6 months after treatment and long-term effects were observed.

The average effective time of IST group and CSA group after treatment were 3.4 months(1~13 months) and 4.5 months (1~15 months), respectively. The efficacy rates of IST group and CSA group at the end of 1st, 3rd, 6th, 9th and 12th months after treatment were 70.9%, 58.2%, 79.5%, 87.2% and 89.7% vs. 46.2%, 38.5%, 46.2%, 61.5% and 61.5%, respectively. More than 1 year follow-up of 39 patients in IST group, the total efficacy rate was 89.7%(35/39). More than 1 year follow-up of 13 patients in CSA group, the total efficacy rate was 61.5%(8/13). There had statistically significant difference between two groups(χ²=5.419, P<0.05). The efficacy rates of reaction sub-IST group and non-reaction sub-IST group to granulocyte colony stimulating factor at the end of 6th months after treatment were 76.9% and 45.6%, respectively.There had no significant difference between two groups (P>0.05). The total efficacy rates of reaction sub-IST group and non-reaction sub-IST group to granulocyte colony stimulating factor were 92.3%, (12/13)and 54.5%(6/11), respectively.The former was significant higher than that of the latter. There was statistically significant difference in these two groups(P<0.05). The efficacy of intensive immunosuppressive therapy was better than that of single-use cyclosporine A. The average effective time of IST group and CSA group after treatment were 3.4 months and 4.5 months, respectively. The efficacy of IST group and CSA group at 1 year after treatment were 89.7% and 61.5%, respectively. The total efficacy rate of reaction sub-IST group and non-reaction sub-IST group to granulocyte colony stimulating factor were 92.3% and 54.5%, respectively.

The therapeutic efficacy of intensive immunosuppressive therapy in children with severe aplastic anemia is better than that of cyclosporine A therapy only. Children with severe aplastic anemia who responded well to granulocyte colony stimulating factor had a better prognosis in the intensive immunosuppressive therapy.