Eur J Endocrinol. 2022 Sep 1:EJE-22-0449. doi: 10.1530/EJE-22-0449. Online ahead of print.
ABSTRACT
OBJECTIVE: To evaluate the safety and efficacy of weekly PEG-rhGH in children with idiopathic short stature (ISS) in China.
DESIGN AND METHODS: A multicenter, phase II study randomized subjects 1:1:1 to weekly subcutaneous injections of PEG-rhGH 0.1 (low dose [LD]) or 0.2 mg/kg/week (high dose [HD]) or control for 52 weeks. The primary endpoint was change in HT-SDS from baseline to week 52. Secondary endpoints were height velocity (HV), bone maturity, IGF-1 SDS, and IGF-1/IGFBP-3 ratio.
RESULTS: A total of 360 children with ISS were recruited in the study (n=120 in each group). At week 52, the mean change from baseline in HT-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; inter-group P<0.0001). Statistically significant changes in HV, 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.
PMID:36130048 | DOI:10.1530/EJE-22-0449
