Treatment of Precocious Puberty in McCune-Albright Syndrome

Abstract

McCune-Albright syndrome (MAS) is typically defined as a triad of precocious puberty (PP), café au lait spots and fibrous dysplasia of bone. PP is the most common endocrinological manifestation of this rare disease and is much more common in girls than in boys. The treatment options for PP associated with MAS have evolved over the last twenty plus years. Therapy in girls typically includes the use of an anti-estrogen, while treatment options in boys include an antiandrogen in combination with an aromatase inhibitor (Al). This article will briefly review the older therapies and explain why they have largely been supplanted by newer approaches. We will discuss current pharmacotherapy options for the treatment of PP in MAS and finally describe potential novel therapies that will hopefully enable optimal care for affected patients.