Ketoconazole treatment of gonadotropin independent precocious puberty in girls with McCune-Albright syndrome: a preliminary report.

McCune-Albright syndrome (MAS) in girls is characterized by gonadotropin independent precocious puberty (GIPP). This form of GIPP is resistant to therapy with GnRH analogues. As an alternative treatment, we successfully used ketoconazole 200 mg t.i.d. orally in two girls with MAS, GIPP and advanced bone age Ketoconazole led to rapid control of GIPP with cessation of menses and regression of pubertal signs in both patients. Ketoconazole was temporarily interrupted in one patient due to pruritus but later restarted without problem. After 1 year of therapy both patients have remained free of menses, progression of puberty and other side effects. Repeat sonography on ketoconazole revealed continued presence of ovarian cysts. Our preliminary experience indicates the safety and effectiveness of ketoconazole as a therapy for GIPP with potential advantages over previously used modes of treatment. Longer use of ketoconazole to suppress GIPP is required to determine whether this therapy can prolong linear growth with enhancement of final height.