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Sustained cyclosporine-induced erythropoietic response in identical male twins with diamond-blackfan anemia.

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.
AuthorsNicola A M Bobey, Manuel Carcao, Yigal Dror, Melvin H Freedman, Niklas Dahl, Richard C Woodman
JournalJournal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 25 Issue 11 Pg. 914-8 (Nov 2003) ISSN: 1077-4114 [Print] United States
PMID14608205 (Publication Type: Case Reports, Journal Article)
Chemical References
  • Cyclosporine
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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