Long-acting PEG-RHGH: insights from five-year outcomes in pediatric patients with growth hormone deficiency from CGLS database

Wei Wu; Nan Li; Xiaoping Luo

doi:10.1530/endoabs.110.P637

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Endocrine Abstracts (2025) 110 P637 | DOI: 10.1530/endoabs.110.P637

ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)

Long-acting PEG-RHGH: insights from five-year outcomes in pediatric patients with growth hormone deficiency from CGLS database

Wei Wu ¹ , Nan Li ² , 3 & Xiaoping Luo ¹

Author affiliations

¹Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Department of Pediatrics, Wuhan, China; ²Peking University Third Hospital, Research Center of Clinical Epidemiology, Beijing, China; ³Peking University, Key Laboratory of Epidemiology of Major Diseases, Ministry of Education, Beijing, China

JOINT2668

Introduction: Growth hormone deficiency (GHD), an endocrine disorder, caused due to insufficient production of growth hormone (GH), thereby affecting children’s growth. Currently, pegylated recombinant human growth hormone (PEG-rhGh) is the only long-acting treatment approved for treating pediatric GHD (PGHD) in China. Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China (CGLS) is a large, surveillance registry database comprising information on participants with short stature treated with recombinant human growth hormone (rhGH) or PEG-rhGH in a real-world setting.

Methods: In this study, we analyzed the data from the CGLS database to evaluate the safety and effectiveness of PEG-rhGH over five-years in children with PGHD in China. Key outcomes such as adverse events (AEs), serious AEs (SAEs), and height gain were assessed.

Results: A total of 1207 participants were included in the safety analysis set, of which 339 received PEG-rhGH for five years and were included for efficacy analysis. Safety assessment indicated that 563 participants exhibited 1328 AEs with an incidence rate of 46. 64%. In addition, SAEs were reported in 0. 99% of participants (n = 12) with none being related to PEG-rhGH treatment. During the treatment period, a significant increase in mean change in height-SD score (ΔHt-SDS) was observed with a mean ΔHt-SDS of 2. 11±0. 87 in five years. Over the treatment course of five years, the mean Ht-SDS showed a consistent increase i. e., from baseline (−2. 43 ± 0. 94) to five years (−0. 33 ± 0. 83). The highest height velocity was highest at 1st year (10. 21 ± 2. 52 cm/y), which stabilized by the fifth year (6. 72 ± 1. 74 cm/y). A more favorable response in height improvements was observed in the age subgroup analysis (2-6, 6-8 and >8 years) when treatment was initiated at an early age.

Conclusion: Data from the CGLS database confirmed that participants with PGHD treated with PEG-rhGH for five years demonstrated a satisfactory safety profile and consistent improvement in height.