title: 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.
authors: Department of Pediatrics, University of Calgary, Alberta Children's Hospital, Canada.
authors: Nicola A M
authors: NA
authors: Bobey
authors: Manuel
authors: M
authors: Carcao
authors: Yigal
authors: Y
authors: Dror
authors: Melvin H
authors: MH
authors: Freedman
authors: Niklas
authors: N
authors: Dahl
authors: Richard C
authors: RC
authors: Woodman
mesh_terms: D029503:Anemia, Diamond-Blackfan / Q000097:blood / Q000188:drug therapy*; D016572:Cyclosporine / Q000494:pharmacology / Q000627:therapeutic use*; D004200:Diseases in Twins; D004920:Erythropoiesis / Q000187:drug effects*; D006801:Humans; D007223:Infant; D008297:Male; D013997:Time Factors; D014430:Twins, Monozygotic
chemical_list: D016572:Cyclosporine