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Published on: January 7, 2016
Long-acting PEGylated growth hormone in children with idiopathic short stature
Xiaoping Luo1, Sha Zhao2, Yu Yang3
1Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, Wuhan, Hubei, China.
Insights
Weekly PEGylated-recombinant human growth hormone (PEG-rhGH) significantly improved height and growth markers in children with idiopathic short stature (ISS). Both low and high doses were safe and well-tolerated over 52 weeks.
Area of Science:
- Pediatrics
- Endocrinology
- Pharmacology
Background:
- Idiopathic short stature (ISS) affects children's growth and development.
- Growth hormone therapy is a standard treatment for ISS.
- Novel formulations like PEGylated-recombinant human growth hormone (PEG-rhGH) aim to improve treatment efficacy and convenience.
Purpose of the Study:
- To evaluate the safety and efficacy of weekly PEG-rhGH in Chinese children with ISS.
- To compare the effects of two different doses of PEG-rhGH against a control group.
Main Methods:
- A multicenter, phase II, randomized controlled study.
- 360 children with ISS were randomized to receive weekly subcutaneous injections of PEG-rhGH (0.1 or 0.2 mg/kg) or a control for 52 weeks.
- Primary endpoint: change in height standard deviation score (HT-SDS); Secondary endpoints: height velocity (HV), bone maturity, IGF-1 SDS, and IGF-1/IGFBP-3 ratio.
Main Results:
- Significant improvements in ΔHT-SDS were observed in both PEG-rhGH groups (0.56 ± 0.26 for low-dose, 0.98 ± 0.35 for high-dose) compared to control (0.20 ± 0.26) (P < 0.0001).
- Statistically significant improvements in HV, IGF-1 SDS, and IGF-1/IGFBP-3 ratio were noted in both treatment groups (P < 0.0001), showing dose-dependent responses.
- Treatment-emergent adverse events (TEAEs) were similar across groups (86.5% HD, 84.6% LD, 91.3% control), with 8.7% experiencing drug-related TEAEs.
Conclusions:
- Fifty-two-week treatment with weekly PEG-rhGH (0.1 or 0.2 mg/kg) significantly improved HT-SDS and other growth parameters in children with ISS.
- The treatment demonstrated a dose-dependent efficacy and was well-tolerated with a similar safety profile across doses.
- Weekly PEG-rhGH is a safe and effective therapeutic option for children with ISS in China.
Objective:
To evaluate the safety and efficacy of weekly PEGylated-recombinant human growth hormone (PEG-rhGH) in children with idiopathic short stature (ISS) in China.
Design And Methods:
This was a multicenter, phase II study in which all subjects were randomized 1:1:1 to weekly s.c. injections of PEG-rhGH 0.1 (low-dose (LD) group) or 0.2 mg/kg/week (high-dose (HD) group) or control for 52 weeks. The primary end point was change (Δ) in height s.d. score (HT-SDS) from baseline to week 52. Secondary end points were height velocity (HV), bone maturity, insulin-like growth factor-1 (IGF-1) SDS, and IGF-1/insulin-like growth factor-binding protein-3 (IGFBP-3) molar ratio.
Results:
A total of 360 children with ISS were recruited in the study (n = 120 in each group). At week 52, ΔHT-SDS was 0.56 ± 0.26, 0.98 ± 0.35, and 0.20 ± 0.26 in the LD, HD, and control groups, respectively (within-group P < 0.0001; intergroup P < 0.0001). Statistically significant values of ΔHV, IGF-1, IGF-1/IGFBP-3 ratio, and IGF-1 SDS at week 52 from baseline were observed in both treatment groups (P < 0.0001). There were clear dose-dependent responses for all auxological variables. PEG-rhGH was well tolerated throughout the treatment period with treatment-emergent adverse events (TEAEs) reported in 86.5%, 84.6%, and 91.3% of children in the HD, LD, and control groups, respectively. The incidence of TEAEs was similar in all treatment groups despite the difference in doses. A total of 27 (8.7%) children experienced drug-related TEAEs.
Conclusion:
Fifty-two-week treatment with PEG-rhGH 0.1 or 0.2 mg/kg/week achieved significant improvement in HT-SDS and other growth-related variables, including HV, IGF-1 SDS, and IGF-1/IGFBP-3 ratio, in a dose-dependent manner. Both doses were well tolerated with similar safety profiles.
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