Long-acting growth hormone therapy in Chinese children with idiopathic short stature: efficacy evaluation and influencing factors

OBJECTIVES: Exogenous growth hormone (GH) supplementation is the primary strategy for treating idiopathic short stature (ISS). Although long-acting growth hormone (LAGH) has attracted considerable interest due to its reduced injection frequency, data on its comparative efficacy and long-term safety in Chinese children with ISS remain limited. This study aims to assess LAGH's efficacy in this population and identify the factors influencing therapeutic outcomes. METHODS: This study was a single-center, retrospective cohort study. It included 180 ISS children who received LAGH treatment in our hospital from January 2019 to December 2024. Based on age, gender, and height standard deviation score (HtSDS), a 1:1 propensity score matching (PSM) was conducted to select concurrent ISS patients receiving short-acting growth hormone (daily rhGH) treatment. Treatment efficacy was evaluated according to the changes in HtSDS and growth velocity (GV) before and after treatment, and multivariate regression analysis was used to identify factors influencing clinical efficacy. RESULTS: The average age of children in the LAGH group was 8.37 ± 2.49 years. After treatment, the LAGH group showed significantly higher HtSDS (-1.47 ± 0.31 vs. -1.63 ± 0.48, p<0.001) and GV (10.06 ± 1.69 vs. 9.24 ± 1.34, p<0.001) compared to the control group for 12 months. Pearson correlation analysis revealed that IGF-1 most correlated with GV (r=0.58, p<0.001). Multivariate regression showed GH efficacy positively correlated with ΔIGF-1 (β=0.42, p<0.001) and IGFBP-3 (β=0.28, p=0.002), negatively with age (β=-0.35, p<0.001). CONCLUSIONS: LAGH demonstrates favorable therapeutic effects in ISS, and it holds the potential to benefit a greater number of Chinese pediatric patients affected by this condition.