Successful treatment of severe aplastic anemia in children using standardized immunosuppressive therapy with antithymocyte globulin and cyclosporine A.
BACKGROUND
Given the heterogeneity of published data in US children, we sought to evaluate outcomes of a standardized immunosuppressive therapy (IST) regimen for severe aplastic anemia (SAA) at The Children's Hospital (Denver, CO).


METHODS
We retrospectively analyzed the records of 16 children diagnosed from 1990 to 2003 and treated by IST, among whom 14 received the standardized regimen of antithymocyte globulin (ATG) and cyclosporine A (CsA). Serial hematologic parameters, complications, transfusion requirements, and time to response were assessed.


RESULTS
One child who died from a pre-existing Aspergillus infection prior to expected IST response was excluded from the analysis. Overall (transfusion-independent) response to IST was 100% (13/13), without any relapses or clinically evident leukemic/myelodysplastic transformations after a median follow-up time of 4.4 years (range: 10 months-13.3 years).


CONCLUSIONS
This report documents excellent outcome using combination ATG and CsA IST for pediatric SAA.
Goldenberg
Neil A
NA
Division of Hematology, Oncology, and BMT, Department of Pediatrics, UCHSC/The Children's Hospital, Denver, Colorado 80128, USA. neil.goldenberg@uchsc.edu
Graham
Douglas K
DK
Liang
Xiayuan
X
Hays
Taru
T
D000293:Adolescent; D000741:Anemia, Aplastic / Q000150:complications / Q000209:etiology / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005938:Glucocorticoids / Q000627:therapeutic use; D006525:Hepatitis, Viral, Human / Q000209:etiology; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use* / Q000633:toxicity; D007223:Infant; D012189:Retrospective Studies; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D016572:Cyclosporine