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Analysis of differences in response to long-acting growth hormone therapy and influencing factors between patients with idiopathic short stature (ISS) and growth hormone deficiency (GHD)

   
@article{TP155988,
	author = {Fang Tang and Li Yan and Peng Gou and Zhonghui Li and Xinran Cheng},
	title = {Analysis of differences in response to long-acting growth hormone therapy and influencing factors between patients with idiopathic short stature (ISS) and growth hormone deficiency (GHD)},
	journal = {Translational Pediatrics},
	volume = {15},
	number = {6},
	year = {2026},
	keywords = {},
	abstract = {Background: Long-acting growth hormone [pegylated recombinant human growth hormone (PEG-rhGH)] is increasingly used for pediatric short stature, yet its differential effects between growth hormone deficiency (GHD) and idiopathic short stature (ISS) remain unclear. This study aims to compare the 52-week efficacy and safety of PEG-rhGH in prepubertal children with GHD versus ISS within a Chinese clinical cohort.Methods: A single-center retrospective cohort study enrolled prepubertal children (3–10 years for girls, 3–11 years for boys) with GHD/ISS (June 2023–June 2024). All received once-weekly PEG-rhGH [starting dose: 0.2 mg/kg/week, adjusted by insulin-like growth factor 1 standard deviation score (IGF-1 SDS)/height velocity (HV)] and completed 12-month follow-up. Primary endpoint: 12-month Δheight standard deviation score (ΔHt-SDS); secondary endpoints: HV, ΔIGF-1 SDS, target height achievement rate. Safety was assessed via adverse events (AEs) and metabolic/hepatorenal parameters. Regression analyses identified influencing factors.Results: There were 132 children (68 GHD, 64 ISS) included, with balanced baselines. At 12 months: (I) efficacy: GHD had higher ΔHt-SDS (0.92±0.31 vs. 0.65±0.27, P},
	issn = {2224-4344},	url = {https://tp.amegroups.org/article/view/155988}
}