Bicalutamide plus anastrozole for the treatment of gonadotropin-independent precocious puberty in boys with testotoxicosis: a phase II, open-label pilot study (BATT)
OBJECTIVE: To evaluate the efficacy, tolerability, and pharmacokinetics of bicalutamide plus anastrozole in young males with testotoxicosis. METHODS: This was a multicenter, open-label, single-arm, 12-month, Phase II pilot trial in 14 males (2-9 years) with testotoxicosis treated with bicalutamide (12.5, 25, 50, or 100 mg) and anastrozole (0.5 or 1 mg) daily. The primary outcome was change in growth rate. RESULTS: At 1 year, the mean (standard deviation) change from baseline in growth rate was -1.6 ( 5.1) cm/year and -0.1 ( 1.8) SD units, and in bone maturation was -2.3 ( 0.5) years. The bone age/chronological age ratio was reduced from 2.1 ( 0.6) at baseline to 1.0 ( 0.4) (p = 0.00013). Steady-state trough R-bicalutamide and anastrozole concentrations were attained by Day 21 and 8, respectively. Gynecomastia (42.9%) and breast tenderness (12.5%) were the most common treatment-related adverse events. CONCLUSIONS: Treatment of testotoxicosis with bicalutamide plus anastrozole resulted in slower growth rate.
Reiter EO, Mauras N, McCormick K, Kulshreshtha B, Amrhein J, De Luca F, O'Brien S, Armstrong J, Melezinkova H. Bicalutamide plus anastrozole for the treatment of gonadotropin-independent precocious puberty in boys with testotoxicosis: a phase II, open-label pilot study (BATT) J Pediatr Endocrinol Metab 2010 Oct;23(10):999-1009.