Clinical Analysis of the Efficacy of Intensive Immunosuppressive Therapy in 68 Children With Severe Aplastic Anemia

Objective 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. Methods 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. Results 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 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. Conclusion 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. Key words: severe aplastic anemia(SAA); immunosuppressive therapy(IST); granulocyte colony stimulating factor(G-CSF); child