Abstract：Objective The purpose of this study is to evaluate the therapeutic effects of combined immunosuppressive (CIS) therapy on severe aplastic anemia (SAA) and refractory aplastic anemia (RAA) in children and to study the relationship between the effectiveness of IS therapy and the immune mediated pathological mechanism. Methods Thirty-six children with SAA (n=33) and RAA (n=3) were given CIS therapy (CIS group, a combination of antithymocyte globulin, cyclosporin A and high-dose immunoglobulin). The therapeutic effects were evaluated and compared with those of 43 children with SAA (n=42) and RAA (n=1) who received the treatment of single IS agent (Control group). Peripheral blood lymphocyte subsets levels were measured with a flow cytometer. Serum interleukin and TNF levels were examined with radioimmunoassay in the two groups. Results The total response rate [ 80.6%(29/36)] in the CIS group was significantly higher than that in the Control group [ 55.8%(24/43)] (P< 0.05). Among the CIS group, 78.8% (26/33) of SAA patients had responses to CIS therapy, which was more than that of the Control group [ 57.1%(24/42)]. All of RAA patients (3/3) were responsive to CIS therapy. The results of immunological function testing showed that the CD4/CD8 ratio decreased and IL-2R, IL-8 and TNF levels increased in the two groups. In the Control group, the children whose disease duration was less than 6 months or the absolute reticulocyte counting was greater than 10×10 9/L demostrated a higher response rate to single IS therapy. The therapeutic effects in the CIS group were not associated with the disease duration and the absolute reticulocyte counts. No severe side effects and treat-related death were found in both groups. Conclusions CIS therapy is effective and safe for childhood SAA and RAA, and the therapeutic effect of CIS is better than single IS agent. Abnormal immune mediated pathological mechanism of AA might be the base of IS therapy. In order to improve the therapeutic effects, it is necessary to make a therapeutic effect prediction for choosing cases and the treatment protocol before treatment of AA with single IS agent.
XIE Xiao-Tian,WANG Yao-Ping,SHI Wei. Effects of combined immunosupressive therapy on severe aplastic anemia and refractory aplastic anemia in children[J]. CJCP, 2004, 6(6): 492-496.
