ArticleTranslational pediatrics2026
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 in Translational pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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 Conclusions: GHD children respond better to PEG-rhGH than ISS children; both have good safety. Diagnosis type is the key efficacy factor, supporting subtype-specific PEG-rhGH strategies.
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