Yonsei Med J.  2014 Jan;55(1):53-60. 10.3349/ymj.2014.55.1.53.

Efficacy of Short-Term Growth Hormone Treatment in Prepubertal Children with Idiopathic Short Stature

• Kim HS
• Yang SW
• Yoo HW
• Suh BK
• Ko CW
• Chung WY
• Lee KH
• Hwang JS
• Ji HJ
• Ahn H
• Kim DH

• KMID: 1779886
• DOI: http://doi.org/10.3349/ymj.2014.55.1.53

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.

Keyword

Treatment outcome; safety; growth hormone; child; short stature; idiopathic

MeSH Terms

Child
Female
Growth Disorders/blood/*drug therapy
Growth Hormone/*therapeutic use
Humans
Insulin-Like Growth Factor Binding Protein 3/blood
Insulin-Like Growth Factor I/metabolism
Male
Treatment Outcome
Growth Hormone
Insulin-Like Growth Factor Binding Protein 3
Insulin-Like Growth Factor I

Figure

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  Fig. 1 Subject distribution. FAS, full analysis set; PPS, per-protocol set.

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  Fig. 2 Mean height velocity (cm/year). Data from per-protocol set; error bar indicates standard deviation. *Significant change from baseline (p<0.0001, Wilcoxon's signed rank test). **Significant change from baseline (lower end of one-sided 95% confidence interval=5.21 cm/year>2 cm/year).

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  Fig. 3 Mean height SDS and change in height SDS. Data from per-protocol set. *Significant change from baseline (p<0.0001, Wilcoxon's signed rank test). SDS, standard deviation score.

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