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Published on: March 17, 2020
[Prognostic factors of immunosuppressive therapy in children acquired aplastic anemia]
Shu-chun Wang1, Xiao-juan Chen, Yao Zou
1Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China.
Insights
Infections during immunosuppressive therapy (IST) for pediatric severe aplastic anemia (SAA) reduce response and increase mortality. A higher nucleated erythrocyte population in bone marrow indicates a good prognosis for these children.
Area of Science:
- Pediatric Hematology
- Immunosuppressive Therapy
- Aplastic Anemia Research
Background:
- Severe aplastic anemia (SAA) is a rare but serious condition in children.
- Immunosuppressive therapy (IST) is a primary treatment for SAA in non-transplant candidates.
- Identifying prognostic factors is crucial for optimizing treatment strategies.
Purpose of the Study:
- To investigate prognostic factors of immunosuppressive therapy (IST) in children with acquired severe aplastic anemia (SAA).
- To evaluate the efficacy and outcomes of IST regimens in pediatric SAA patients.
Main Methods:
- Retrospective analysis of 56 children with SAA treated with rabbit anti-thymocyte globulin (R-ATG) and cyclosporine A (CSA).
- Standardized dosing and therapeutic drug monitoring for CSA were employed.
- Patients also received stanozolol or testosterone propionate.
Main Results:
- The overall response rate to IST was 62.5%, with a complete remission rate of 37.5%.
- Five-year overall survival was 66.27%.
- Infections during R-ATG treatment correlated with significantly lower response rates and higher mortality. Patients with a nucleated erythrocyte population >=10% in bone marrow showed a good prognosis, while those with granulocytes >=10% had lower mortality.
Conclusions:
- Presence of infections during R-ATG therapy is a negative prognostic factor in pediatric SAA.
- A higher proportion of nucleated erythrocytes in bone marrow is associated with a favorable prognosis.
- These findings can aid in risk stratification and treatment decisions for pediatric SAA.
Objective:
To investigate prognostic factors of immunosuppressive therapy (IST) in children acquired severe aplastic anemia(SAA).
Methods:
Data of 56 consecutive children cases with SAA who had received rabbit anti-thymocyte globulin (R-ATG) [3-5 mg/( kg x d) x 5 d] and cyclosporine A (CSA) from January 2000 to June 2006 were retrospectively analyzed. No repeated courses of R-ATG were given for nonresponders. All the patients also received stanozolol or testosterone propionate. The dose of CSA was adjusted to maintain trough drug levels above 100 microg/L and peak drug levels above 300 microg/L.
Results:
The overall response rate to the immunosuppressive therapy (IST) was 62.5% and the complete remission rate was 37.5%. The 5-year overall survival for IST regimens was 66.27% +/- 6.84%, patients who had infections when using ATG had significantly lower response and higher mortality. Patients whose nucleated erythrocyte population in bone marrow was > or =10% had good prognosis. Patients whose granulocytes population in bone marrow was > or =10% had lower mortality.
Conclusion:
Patients who had infections when using ATG had significantly lower response and higher mortality. Patients whose nucleated erythrocyte population in bone marrow was > or =10% had good prognosis.
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