Therapeutic Effects Of Non-Standard Immunosuppressive Therapy In Children With Acquired Aplastic Anemia

ObjectiveTo investigate the efficacy and side effects of non-standard immunosuppressive therapy with cyclosporine A + androgen + intermittent high dose prednisone,cyclosporin A + androgen + low dose prednisone in children with aplastic anemia(AA).And analyze the related influencing factors of curative effect.MethodsThe clinical data of 59 patients with AA admitted from January 1,2013 to May31,2018 in our department of pediatrics were retrospectively analyzed and divided into A and B groups according to different treatment methods.Group A patients(n=18)were treated with cyclosporine A + androgen + intermittent high dose prednisone.Group B patients(n=41)were treated with cyclosporine A + androgen + low dose prednisone.The curative effects of non-severe aplastic anemia and severe aplastic anemia were compared within treatment group,the curative effect and side effects were compared among the groups,and the influencing factors of the curative effect were discussed respectively.ResultsAfter 6 months of drug treatment,59 patients were evaluated for efficacy.1patient attained complete remission,25 cases attained partial remission and treatment was ineffective in 33 cases.The overall effective rate was 44.1%(26/59).In group A,the effective rate was 50%(9/18): 9 patients attained partial remission while other 9patients had ineffective treatment outcome.In group B,the effective rate was 41.5%(17/41): 1 patient attained complete remission,16 patients with partial remission,and treatment was ineffective in 24 patients.As of the end of follow-up,the median follow-up time was 18.5 months for group A and 32 months for group B.The follow-up time of group B was significantly longer than that of group A,however,group A patients had a higher effective rate of 72.2%(13/18)than 58.5 %(24/41)for group B.In addition,effectiveness rate for severe aplastic anemia cases in group A(n=12)was 91.7%(11/12)and in group B(n=27)was 55.6%(15/27).No case of recurrence or death occurred in group A patients but group B patients had 3 cases of recurrence and 1 case of death during treatment.Univariate and binary logistic regression analysis was used to investigate the factors influencing treatment effectiveness in group A and B.Severity of aplastic anemia was significantly associated with curative effect in group A(P<0.05),patients with severe aplastic anemia had better treatment outcome than non-severe aplastic anemia patients.Other factors in group A and related factors in group B had no significant effect on the efficacy(P>0.05).The survival time of all follow-up patients was analyzed by univariate survival analysis,and showed no statistical significance(P> 0.05).Adverse reactions were mild and improved after symptomatic treatment or after the reduction or withdrawal of immunosuppressive drugs for both group A and B.Conclusion1.Non-standard immunosuppressive therapy can be used as an alternative treatment when standard treatments cannot be selected.2.Cyclosporine A + androgen + intermittent high dose prednisone treatment of severe aplastic anemia is cost effective,convenient with high compliance and without serious adverse reactions.3.Patients with aplastic anemia with humoral immune disorders and autoimmune-related antibodies in vivo may respond better to cyclosporine A +androgen + intermittent high dose prednisone.