title: Therapy for aplastic anemia.
abstract: A 24-year-old man from Ecuador presents to your clinic with dyspnea on exertion, bruising, and petechiae. He is noted to be pancytopenic with ANC 430, hemoglobin 7.4 g/dL (reticulocyte count 0.9%), and platelets 18 000. His BM biopsy is hypocellular for age. Ultimately, he is diagnosed with severe aplastic anemia. He is the only child of 2 South American parents without any matches in the unrelated donor registry, including cord blood. He is red cell- and platelet transfusion-dependent. He has been recommended therapy with antithymocyte globulin and cyclosporine but declined it. He seeks recommendations about new alternatives to this regimen to improve his chance of response.
authors.affiliation: Department of Oncology, Johns Hopkins University, Baltimore, MD 21205, USA. adezern1@jhmi.edu
authors.forename: Amy E
authors.initials: AE
authors.lastname: DeZern
authors.forename: Eva C
authors.initials: EC
authors.lastname: Guinan
mesh_terms: D000741:Anemia, Aplastic / Q000188:drug therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D006801:Humans; D008297:Male; D016032:Randomized Controlled Trials as Topic; D020123:Sirolimus / Q000627:therapeutic use; D016559:Tacrolimus / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D016572:Cyclosporine; D020123:Sirolimus; D016559:Tacrolimus