Adult height in patients with familial male-limited precocious puberty and the role of an aromatase inhibitor in patient management

Abstract

Aromatase inhibitor therapy is associated with a positive height outcome in FMPP but the outcome with and without intervention is unpredictable. Clinicians need to be cautious when counselling families about the potential height outcome in FMPP.

A Case of Familial Male-limited Precocious Puberty with a Novel Mutation

“…Most recent studies have shown good outcomes with anti-androgens such as bicalutamide and third generation aromatase inhibitors such as anastrozole ( 4 , 6 , 7 , 8 ). These treatments, in conjunction with one another, have shown better adult height potential preservation than either alone ( 5 ). In one study, anti-androgens alone did not prevent early growth plate closure ( 5 ), and in another, anastrozole was ineffective in treating continued aggressiveness ( 7 ).…”

“…These treatments, in conjunction with one another, have shown better adult height potential preservation than either alone ( 5 ). In one study, anti-androgens alone did not prevent early growth plate closure ( 5 ), and in another, anastrozole was ineffective in treating continued aggressiveness ( 7 ). Bicalutamide and anastrozole offer the additional advantage of a prolonged half-life that allows for convenient once-daily dosing while other anti-androgen agents would require multiple daily doses.…”

“…The data is somewhat variable in regards to long-term outcomes, but results are promising. Adult height is improved following treatment, though adults with FMPP are still slightly below average in height ( 5 , 6 ). Long-term effects on fertility are still unknown, however one study did not show any differences in sperm count between patients taking anastrozole and controls ( 14 ).…”

“…Treatment goals are twofold: to decrease the effects of testosterone, typically through the use of anti-androgen agents; and to inhibit the conversion of testosterone to estrogen through the use of aromatase inhibitors. Multiple different medications have been trialed, but most recent studies show promising results using bicalutamide (an anti-androgen agent), and anastrozole (a third-generation aromatase inhibitor) ( 4 , 5 , 6 , 7 , 8 ). Little is known about the long-term effects of treatment because the disorder is so rare.…”

“…Little is known about the long-term effects of treatment because the disorder is so rare. However, studies using bicalutamide and anastrozole have shown improved height potential ( 5 , 6 ). In one study, final adult height was only 0.4 standard deviations below that of the general US male population ( 6 ).…”