Efficacy of short-term growth hormone treatment in prepubertal children with idiopathic short stature.

Abstract

PURPOSE: It has been reported that daily recombinant human growth hormone (GH)

treatment showed beneficial effects on growth in prepubertal children with

idiopathic short stature (ISS). The present study aimed to validate the GH

(Eutropin(R)) effect on growth promotion and safety after short-term GH

treatment. MATERIALS AND METHODS: This study was an open-label, multicenter,

interventional study conducted at nine university hospitals in Korea between 2008

and 2009. Thirty six prepubertal children with ISS were enrolled in this study to

receive 6-month GH treatment. Yearly growth rate, height standard deviation score

(SDS), and adverse events were investigated during treatment. RESULTS: After 26

weeks of GH treatment, the height velocity significantly increased by 6.36+/-3.36

cm/year (p<0.001). The lower end of one-sided 95% confidence interval was 5.22

cm/year, far greater than the predefined effect size. The gain in height SDS at

week 26 was 0.57+/-0.27 (p<0.0001). Bone age significantly increased after GH

treatment, however, bone maturation rate (bone age for chronological age) showed

limited advancement. This 26-week GH treatment was effective in increasing serum

levels of insulin-like growth factor (IGF)-I and IGF binding protein (IGFBP)-3

from baseline (p<0.0001). Eutropin was well tolerated and there were no

withdrawals due to adverse events. No clinically significant changes in

laboratory values were observed. CONCLUSION: This 6-month daily GH treatment in

children with ISS demonstrated increased height velocity, improved height SDS,

and increased IGF-I and IGFBP-3 levels with a favorable safety profile.