Effect of Antiandrogen, Aromatase Inhibitor, and Gonadotropin-releasing Hormone Analog on Adult Height in Familial Male Precocious Puberty

Objective Antiandrogen, aromatase inhibitor, and gonadotropin-releasing hormone analog (GnRHa) treatment normalizes growth rate and bone maturation and increases predicted adult height (AH) in boys with familial male-limited precocious puberty (FMPP). To evaluate the effect of long-term antiandrogen, aromatase inhibitor, and GnRHa on AH, boys with FMPP who were treated were followed to AH. Study design Twenty-eight boys with FMPP, referred to the National Institutes of Health, were started on antiandrogen and aromatase inhibitor at 4.9 +/- 1.5 years of age; GnRHa was added at 6.9 +/- 1.5 years of age. Treatment was discontinued at 12.2 +/- 0.5 years of age (bone age, 14.4 +/- 1.3). AH was assessed at 16.4 +/- 1.3 years of age (bone age, 18.5 +/- 0.6). Results AH (mean +/- SD) for all treated subjects was 173.6 +/- 6.8 cm (-0.4 +/- 1.0 SD relative to adult US males). For 25 subjects with pretreatment predicted AH, AH significantly exceeded predicted AH at treatment onset (173.8 +/- 6.9 vs 164.9 +/- 10.7 cm; P < .001), but fell short of predicted AH at treatment discontinuation (177.3 +/- 9.0 cm; P < .001). For 11 subjects with maternal or sporadic inheritance, the mean AH was 3.1 cm (0.4 SD score) below sex-adjusted midparental height (175.4 +/- 5.8 vs 178.5 +/- 3.1 cm [midparental height]; P = .10). For 16 subjects with affected and untreated fathers, AH was significantly greater than fathers' AH (172.8 +/- 7.4 vs 168.8 +/- 7.2 cm; P < .05). Conclusions Long-term treatment with antiandrogen, aromatase inhibitor, and GnRHa in boys with FMPP results in AH modestly below sex-adjusted midparental height and within the range for adult males in the general population.