ABSTRACT
BACKGROUND: Allogeneic stem cell transplantation (ASCT) represents the only option with a potential cure rate of 30% to 50% in myelodysplastic syndrome (MDS); however, < 5% of patients are optimal candidates for this management. Therapeutic options are limited in patients unsuitable for ASCT. Evidence that androgens might be beneficial in MDS is controversial. We aimed to document the clinical outcomes of patients diagnosed with MDS treated with danazol as first-line therapy. PATIENTS AND METHODS: We retrospectively reviewed patients diagnosed in our center with MDS according to the World Health Organization 2008 criteria and treated with danazol between 2005 and 2015. Response was defined according to international working group criteria. RESULTS: We included 42 patients treated exclusively with danazol. Median dose was 400 mg/d (range, 100-600 mg/d). Median follow-up was 12 (range, 3-76) months. Twenty-four of these patients (60%) achieved clinical response. Median overall survival was 24 months (95% confidence interval, 5.1-42). Responders were older than nonresponders (P = .025) and had higher baseline hemoglobin concentration (P = .009). No patients discontinued danazol because of toxicity. Fifteen patients died (35.7%) and 5 progressed to acute myeloid leukemia. CONCLUSION: Danazol as first-line therapy is an acceptable treatment option with low side effects for patients with MDS who cannot receive ASCT.
