Abstract |
After failing a trial of corticosteroid therapy, molecularly proven identical twins were treated for transfusion-dependent Diamond-Blackfan anemia with cyclosporine A, resulting in a robust erythropoietic response and a reversal of anemia. Clonogenic assays of marrow hematopoietic progenitors from both patients showed exuberant growth of BFU-E colonies but absent CFU-E. Clinically, the response has been sustained, and both patients have continued cyclosporine therapy and have been transfusion-independent for more than 27 months.
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Authors | Nicola A M Bobey, Manuel Carcao, Yigal Dror, Melvin H Freedman, Niklas Dahl, Richard C Woodman |
Journal | Journal of pediatric hematology/oncology
(J Pediatr Hematol Oncol)
Vol. 25
Issue 11
Pg. 914-8
(Nov 2003)
ISSN: 1077-4114 [Print] United States |
PMID | 14608205
(Publication Type: Case Reports, Journal Article)
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Chemical References |
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Topics |
- Anemia, Diamond-Blackfan
(blood, drug therapy)
- Cyclosporine
(pharmacology, therapeutic use)
- Diseases in Twins
- Erythropoiesis
(drug effects)
- Humans
- Infant
- Male
- Time Factors
- Twins, Monozygotic
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