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Diamond-blackfan anemia and cyclosporine therapy revisited
A J Alessandri1, P C Rogers, L D Wadsworth
1Department of Paediatrics, University of British Columbia and British Columbia's Children's Hospital, Vancouver, Canada.
Insights
Cyclosporine A (CSA) offers a promising treatment for Diamond-Blackfan anemia when steroids fail, enabling patients to become transfusion-independent. However, CSA discontinuation can lead to relapse, necessitating ongoing therapy.
Area of Science:
- Hematology
- Pediatric Hematology
- Immunosuppressive Therapy
Background:
- Diamond-Blackfan anemia (DBA) is a rare congenital red blood cell aplasia.
- Prednisone is a common treatment, but can cause significant side effects.
- Alternative therapies are needed for patients intolerant to steroids.
Observation:
- A pediatric patient with DBA, diagnosed in infancy, initiated cyclosporine A (CSA) at 9 years and 8 months due to prednisone intolerance.
- The patient demonstrated a significant positive response to CSA, allowing for steroid cessation within 5 months.
- The patient has remained transfusion-independent for over 4 years.
Findings:
- CSA therapy led to sustained transfusion independence in a patient with Diamond-Blackfan anemia.
- Discontinuation attempts of CSA resulted in anemia relapse, particularly during viral infections.
- Recurrent oral aphthous ulceration was a manageable side effect of CSA treatment.
Implications:
- Cyclosporine A represents a viable therapeutic option for Diamond-Blackfan anemia patients who experience adverse effects from steroid therapy.
- Early consideration of CSA may prevent the need for transfusion programs or stem cell transplantation.
- Long-term CSA therapy appears necessary for sustained remission in some DBA patients.
Abstract:
A girl with Diamond-Blackfan anemia diagnosed in infancy started cyclosporine A (CSA) therapy at 9 years and 8 months of age after experiencing unacceptable side effects while receiving prednisone. Since then, she has been followed-up for more than 4 years. She exhibited a dramatic response to CSA, with weaning and then cessation of steroid therapy after 5 months. She has remained transfusion-independent. Attempts to discontinue CSA therapy have been unsuccessful. Relapse of the anemia has occurred in the context of viral infections with missed CSA doses. The major clinical problem during treatment has been recurrent oral aphthous ulceration, which responds to topical therapy. She is currently maintained on CSA 100 mg twice daily with a hemoglobin of 10.2 g/dL and a reticulocyte count of 1.6%. A trial of CSA therapy should be considered in patients with Diamond-Blackfan anemia in whom steroid therapy has failed before a transfusion program is instituted or alternative donor stem cell transplantation is entertained.
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