A randomized pilot trial of growth hormone with anastrozole versus growth hormone alone, starting at the very end of puberty in adolescents with idiopathic short stature

BACKGROUND When given during the course of puberty, anastrozole (A), an aromatase inhibitor, has been shown to increase the predicted adult height (PAH) of GH-deficient (GHD) boys treated with recombinant human growth hormone (rhGH). Our study questioned whether this treatment could retain some of its effects in non-GHD adolescent boys if started only at the very end of puberty, a time when rhGH treatment is denied to short adolescents who have almost reached their final height. OBJECTIVE To explore the effect on adult height of a combination of rhGH and A, compared with rhGH alone, at the end of puberty in boys with idiopatic short stature (ISS). METHODS A prospective randomized study comparing rhGH + A and rhGH was conducted in 24 healthy adolescent boys aged 15.2 ± 1.2 yrs with serum testosterone at adult levels and a faltering growth velocity <3.5 cm/yr leading to a predicted adult height (PAH) <2.5 SDS. Treatments were stopped when growth velocity became <10 mm in 6 months or when height was close to 170 cm. A historical group of ISS adolescents (N = 17) matched for puberty and growth was used for comparison. RESULTS IGF1 levels remained within normal limits in all treated patients. Mean treatment duration was 19 months in the rhGH + A group and 11.5 months in the rhGH group (P = 6.10(-4)). Adult height reached 168.4 ± 2.6 cm in the rhGH + A group and 164.2 ± 5.6 cm in the rhGH group (P < 0.02). Adult height was 160.1 ± 2.8 cm in the historical controls. CONCLUSION A combination of rhGH and A, started at the very end of puberty, seems to allow boys with ISS to reach a greater adult height than rhGH alone. Larger trials are needed to confirm this preliminary observation.