Danazol as first-line therapy for aplastic anemia

• Published in: Annals of hematology 2011-05, Vol.90 (5), p.523-527
• Main Authors: Jaime-Pérez, José Carlos, Colunga-Pedraza, Perla R., Gómez-Ramírez, Cynthia D., Gutiérrez-Aguirre, César H., Cantú-Rodríguez, Olga G., Tarín-Arzaga, Luz C., Gómez-Almaguer, David
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Aged, 80 and over; Androgens - adverse effects; Androgens - therapeutic use; Anemia, Aplastic - drug therapy; Anemia, Aplastic - therapy; Child; Danazol - adverse effects; Danazol - therapeutic use; Developing Countries; Female; Hematology; Hematopoietic Stem Cell Transplantation; Humans; Longitudinal Studies; Male; Medicine; Medicine & Public Health; Mexico; Middle Aged; Oncology; Original Article; Remission Induction; Retrospective Studies; Severity of Illness Index; Survival Analysis; Young Adult
• ISSN: 0939-5555; 1432-0584
• DOI: 10.1007/s00277-011-1163-x

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.