Evaluation of the efficacy of immunosuppressive therapy with rabbit-antithymocyte globulin combined with Cyclosporine in children with severe aplastic anemia
10.3760/cma.j.issn.2095-428X.2015.03.008
- VernacularTitle:兔抗胸腺细胞球蛋白联合环孢素免疫抑制治疗儿童重型再生障碍性贫血的疗效
- Author:
Yan SU
;
Runhui WU
;
Jie MA
;
Xuan ZHOU
;
Rui ZHANG
- Publication Type:Journal Article
- Keywords:
Antithymocyte globulin;
Child;
Severe aplastic anemia
- From:
Chinese Journal of Applied Clinical Pediatrics
2015;30(3):189-193
- CountryChina
- Language:Chinese
-
Abstract:
Objective To evaluate the efficacy and adverse reaction of the first line therapy with rabbit-antithymocyte globulin (ATG) combined with Cyclosporine (CsA) in children with acquired severe aplastic anemia.Methods A retrospective analysis was performed.The clinical data included 44 children with acquired severe aplastic anemia treated with ATG combined with CsA in Beijing Children's Hospital Affiliated to Capital Medical University between June 2006 and December 2013.The SPSS 19.0 statistical package was used for data analysis.Results Sixteen cases were severe aplastic anemia,and 28 cases were very severe aplastic anemia.The median age of treatment with ATG was 8 years old and 10 months.The median follow-up period was 24 months.The hematologic response rate was 66％ (29/44 cases) in 6 months after ATG treatment,and the overall response rate was 77％ (34/44 cases) at the endpoint of follow-up.The median time of partial remission (PR) was 3 months,of complete remission (CR) was 12 months.Eighty-five percent (29/34 cases)effective cases achieved PR during 6 months,while 56％ (15/27 cases)cases achieved CR in 12 months.There was no significant difference between the response rate with ATG treatment and gender,severity of disease,age,interval from clinical onset to ATG treatment,and ratio of CD4 +/CD8 +.The response rate in children without serum sickness was superior to that with serum disease(P =0.006).Thirty-four cases (77％) manifested immediate side-effects with ATG infusion.The side-effects included fever,chill,rash,gastrointestinal distress and irritable cough.The overall 2-year survival rate was 87.3％ (39/44 cases) and the 2-year transplantation-free survival rate was 82.5％ (37/44 cases) (x2 =0.34,P =0.56).Conclusions The response rate with rabbit-ATG combined with CsA in children with acquired severe aplastic anemia was 77％.The adverse reactions were minor.The immunosuppressive therapy should begin as soon as possible in children without chance for hematopoietic stem cell transplantation.
