title: Danazol therapy for aplastic anemia refractory to immunosuppressive therapy.
abstract: Although there are anecdotal reports of the efficacy of danazol in the treatment of aplastic anemia (AA), there has been no systematic study to clarify its efficacy and toxicity. Therefore, we assessed the efficacy of danazol for treatment of patients with AA refractory to immunosuppressive therapy (IST) and those who relapsed after IST, in a prospective clinical trial. Sixteen patients (12 males and four females; six severe cases and 10 moderate cases) were treated with 300 mg of danazol daily for 12 weeks. All patients completed the treatment period without occurrence of severe toxicity. Three female patients achieved partial remission, whereas only two of the 12 male patients did so. None of the responders had shown a response to previous IST or an increase in the percentage of paroxysmal nocturnal hemoglobinuria (PNH)-type cells which are known to be a marker for a good response to IST. These findings indicate that danazol is effective for a subset of AA patients, and particularly for female patients with AA refractory to IST.
authors.affiliation: Department of Internal Medicine, NTT Kanazawa Hospital, Kanazawa, Ishikawa, Japan.
authors.forename: Tatsuya
authors.initials: T
authors.lastname: Chuhjo
authors.forename: Hirohito
authors.initials: H
authors.lastname: Yamazaki
authors.forename: Mitsuhiro
authors.initials: M
authors.lastname: Omine
authors.forename: Shinji
authors.initials: S
authors.lastname: Nakao
mesh_terms: D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000628:therapy; D001803:Blood Transfusion; D056486:Chemical and Drug Induced Liver Injury / Q000209:etiology; D003131:Combined Modality Therapy; D003613:Danazol / Q000627:therapeutic use*; D004351:Drug Resistance; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000175:diagnosis / Q000188:drug therapy; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D003613:Danazol