title: Danazol as first-line therapy for aplastic anemia.
abstract: Immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG) plus cyclosporine A (CsA) is the standard treatment for aplastic anemia (AA) patients not eligible for allogeneic hematopoietic stem cell transplantation (HSCT). In the absence of ATG + CsA, androgens continue to be a treatment option. We documented the clinical evolution of AA patients treated with danazol instead of ATG + CsA. AA patients lacking both, human leukocyte antigen-matched donor and access to IST, were treated with danazol and modern support therapy and compared with those receiving a HSCT. Overall survival (OS), response rates, and death risk odds were calculated. Fifty AA patients were studied. Thirteen received a HSCT and 37 danazol and support therapy. Median daily dose of danazol was 400 mg (300 to 600 mg), administered during a median of 12 months. Five-year OS was higher for patients receiving HSCT (92%) compared to the danazol group (41%) (P = 0.001). Overall response rate was 46% (17/37) in the danazol-treated group and the median time to initial response was 3 months (1-27). Tendency to achieve remission was similar among severity groups (P = 0.094). The only adverse side effect recorded on the danazol group was an episode of gastrointestinal bleeding. No patient treated with danazol suffered clonal evolution of his/her disease. Although ATG plus CsA is the therapy of choice for AA patients without a donor when neither HSCT nor IST is available, danazol remains an acceptable therapeutic option for AA patients.
authors.affiliation: Department of Hematology, Internal Medicine Division, Dr. José Eleuterio González University Hospital of the School of Medicine of the Universidad Autónoma de Nuevo León, Monterrey, Mexico. carjaime@hotmail.com
authors.forename: José Carlos
authors.initials: JC
authors.lastname: Jaime-Pérez
authors.forename: Perla R
authors.initials: PR
authors.lastname: Colunga-Pedraza
authors.forename: Cynthia D
authors.initials: CD
authors.lastname: Gómez-Ramírez
authors.forename: César H
authors.initials: CH
authors.lastname: Gutiérrez-Aguirre
authors.forename: Olga G
authors.initials: OG
authors.lastname: Cantú-Rodríguez
authors.forename: Luz C
authors.initials: LC
authors.lastname: Tarín-Arzaga
authors.forename: David
authors.initials: D
authors.lastname: Gómez-Almaguer
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000728:Androgens / Q000009:adverse effects / Q000627:therapeutic use*; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000628:therapy; D002648:Child; D003613:Danazol / Q000009:adverse effects / Q000627:therapeutic use*; D003906:Developing Countries; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008800:Mexico; D008875:Middle Aged; D012074:Remission Induction; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016019:Survival Analysis; D055815:Young Adult
chemical_list: D000728:Androgens; D003613:Danazol