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

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.

A Sustained Release Human Growth Hormone (LB03002):Efficacy and Safety Following Six-month Treatment in Children with Growth Hormone Deficiency (GHD) / 대한소아내분비학회지

BACKGROUND: LB03002[somatropin(rDNA origin) for injectable suspension] is a sustained release formulation of human growth hormone to be administered by once-a-week subcutaneous injections. Less frequent administration could provide a considerable improvement on compliance and convenience. OBJECTIVE: To determine the efficacy and safety of a LB03002 administered in children with GHD once weekly for 6 months. DESIGN: Open-label, active-controlled, randomised, parallel group, phase II study. PATIENTS: A total of forty-two naive or previously treated, pre-pubertal children with GHD, confirmed by two different GH provocation tests, were randomised and received either LB03002(0.3 or 0.5 mg/kg/week) or Eutropin(TM)(daily rhGH, 0.3 mg/ kg/week divided 6 times a week) for 6 months. RESULTS: The pre-treatment(HV0) and 6-month annualised height velocity(HV6) are shown(mean+/-SD) in the table below: ----------------------------------------------------------------------- LB03002 LB03002 EutropinTM 0.3 mg/kg/week 0.5 mg/kg/week 0.3 mg/kg/week ----------------------------------------------------------------------- N 10 13 13 HV0 3.1+/-1.0 3.9+/-1.5 3.0+/-1.1 HV6 9.3+/-2.3 10.2+/-2.3 11.1+/-2.5 ----------------------------------------------------------------------- Mean IGF-I and IGFBP-3 levels were significantly elevated from baseline values in all the study groups. LB03002 at all dose groups was safe and well tolerated. No clinically relevant adverse events or abnormal laboratory parameters were observed and there were no remarkable differences between groups or changes over time within groups regarding parameters for glucose and lipid metabolism including fasting glucose and haemoglobin A1c. Injection site reactions were mostly mild to occasionally moderate and resolved within 2 to 3 days post-dose without intervention. CONCLUSIONS: Treatment with LB03002 by weekly administration of the doses tested in the study resulted in comparable safety and efficacy to daily rhGH in pre-pubertal children with GHD.