Related Experiment Video
Updated: May 1, 2026
Comparative Analysis of Human Growth Hormone in Serum Using SPRi, Nano-SPRi and ELISA Assays
Published on: January 7, 2016
One-Year Data from a Long-Term Phase IV Study of Recombinant Human Growth Hormone in Short Children Born Small for
Hans-Peter Schwarz1, Dorota Birkholz-Walerzak2, Mieczyslaw Szalecki3
1Department of Endocrinology, von Haunersches Kinderspital, University Hospital Munich, Munich, Germany.
Insights
Recombinant human growth hormone (rhGH) effectively treats short children born small for gestational age (SGA), improving height without significantly impacting diabetes risk in the first year. This study confirms rhGH safety and efficacy in this population.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Metabolic Health
Background:
- Long-term phase IV study investigating recombinant human growth hormone (rhGH) efficacy and safety in short children born small for gestational age (SGA).
- Focus on the impact of rhGH on diabetes incidence, with this report presenting the first interim analysis after one year of treatment.
Purpose of the Study:
- To evaluate the long-term effects of rhGH on carbohydrate metabolism in short SGA children.
- To assess the efficacy of rhGH on height parameters, growth factors, and safety profile.
- To determine the incidence of diabetes mellitus during rhGH treatment.
Main Methods:
- Prospective, open-label, multicentre study enrolling 278 pre-pubertal children with SGA.
- rhGH treatment administered at 0.035 mg/kg/day.
- Primary endpoints included fasting glucose, 2-hour oral glucose tolerance test (OGTT), and HbA1c; secondary endpoints included height parameters, IGF-I, IGFBP-3, and adverse events.
Main Results:
- No cases of diabetes mellitus were observed in the first year of rhGH treatment.
- Carbohydrate metabolism markers (fasting glucose, HbA1c, 2-h OGTT) remained stable.
- Significant improvements in height standard deviation score (HSDS), height velocity (HV), and HVSDS were observed. Mean HSDS improved from -3.39 to -2.57, and mean HV increased from 4.25 cm/year to 8.99 cm/year.
- Mean IGF-I SDS and IGFBP-3 SDS increased. Most treatment-emergent adverse events (69.3%) were mild-to-moderate and unrelated to rhGH.
Conclusions:
- rhGH treatment is effective in improving growth parameters in short children born SGA.
- rhGH treatment demonstrates a safe profile concerning carbohydrate metabolism after one year.
- This interim analysis supports the efficacy and safety of rhGH for short SGA children.
Background:
This prospective, open-label, non-comparative, multicentre, long-term phase IV study is examining the efficacy and safety of somatropin [recombinant human growth hormone (rhGH)] in short children born small for gestational age (SGA) and its impact on the incidence of diabetes. This report is the first interim analysis of patients who have completed 1 year of treatment.
Methods:
A total of 278 pre-pubertal patients were enrolled. Key eligibility criteria included height standard deviation score (HSDS) <-2.5; parental adjusted SDS <-1; birth weight and/or length <-2 SD and failure to show catch-up growth by ≥4 years of age. Patients were treated with rhGH 0.035 mg/kg/day. The primary objective was to evaluate the long-term effect of rhGH on carbohydrate metabolism [including fasting glucose, stimulated glucose (2-h oral glucose tolerance test, OGTT) and glycated haemoglobin (HbA1c)]. Secondary objectives included evaluation of height parameters [body height, HSDS, height velocity (HV), HVSDS]; insulin-like growth factor 1 (IGF-I) and insulin-like growth factor-binding protein 3 (IGFBP-3) serum levels during treatment; and incidence and severity of adverse events (AEs).
Results:
None of the children developed diabetes mellitus within the first year of treatment. Mean levels of fasting glucose, HbA1c and 2-h OGTT values remained stable during the study period. Treatment with rhGH was effective, as documented by all height parameters. Mean HSDS improved from -3.39 at baseline to -2.57 at Year 1. Mean HV increased markedly from 4.25 cm/year at baseline to 8.99 cm/year during the first year. Similarly, mean peak-centred HVSDS increased from -2.13 at baseline to +4.16 at Year 1. Mean IGF-I SDS and IGFBP-3 SDS also increased within the first year (by +1.80 and +0.41, respectively). 13 patients (4.7%) did not respond adequately to treatment (HVSDS <1); they were withdrawn from the study. In total, 192 children (69.3%) experienced treatment-emergent AEs; most (98.7%) were mild-to-moderate, and the majority (96.5%) were unrelated to study treatment.
Conclusion:
This interim analysis shows that short children born SGA can be effectively and safely treated with rhGH and that rhGH treatment has no major impact on carbohydrate metabolism after the first year of treatment.
Related Concept Videos
01:24Pharmacokinetics in Pediatric Patients: Drug Metabolism
01:10Nature and Nurture
01:23Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
01:26Pharmacokinetics in Pediatric Patients: Drug Excretion