Sustained cyclosporine-induced erythropoietic response in identical male twins with diamond-blackfan anemia.
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.
Bobey
Nicola A M
NA
Department of Pediatrics, University of Calgary, Alberta Children's Hospital, Canada.
Carcao
Manuel
M
Dror
Yigal
Y
Freedman
Melvin H
MH
Dahl
Niklas
N
Woodman
Richard C
RC
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
D016572:Cyclosporine