Work

Long-Acting PEGylated Recombinant Human Growth Hormone in Short Children Born Small for Gestational Age in China: 4-Year Results from a Randomized Phase 2 Study

Year: 2026

Type: article

Abstract: INTRODUCTION: Children born small for gestational age (SGA) who failed to achieve catch-up growth are at risk of persistent short stature and associated metabolic complications. Daily recombinant human growth hormone (rhGH) therapy improves growth but is limited by adherence challenges. This study evaluated the long-term efficacy and safety of once-weekly PEGylated recombinant human growth hormone (PEG-rhGH, Jintrolong) in short children born SGA. METHODS: This multicenter, randomized, open-label, dose-response, phase 2 study comprised a 52-week main phase and an ongoing extension (NCT02375620) across 9 and 7 sites in China, respectively. Ninety-six GH-treatment-naive, non-GH-deficient, prepubertal children born SGA (aged 3 to <8 years) were randomized (1:1) to PEG-rhGH 0.1 or 0.2 mg/kg/week for 52 weeks. After 52 weeks, sixty-six children proceeded to the extension phase continuing PEG-rhGH treatment (initial dose: 0.2 mg/kg/week; maximum: 0.4 mg/kg/week) until near-adult height. RESULTS: After 208 weeks, the mean (SD) ΔHT-SDS was 1.746 (0.488) and 1.660 (0.710) in the original 0.2 and 0.1 mg/kg/week groups, respectively. Overall, 90.9% (60/66) of patients achieved height > -2 SDS and 53.0% (35/66) reached their target height SDS. The mean annualized height velocity remained within the normal range. Long-term PEG-rhGH treatment was well tolerated, with most treatment-emergent adverse events being mild and consistent with known GH-related effects. CONCLUSION: Once-weekly PEG-rhGH provided sustained improvement in height and growth velocity over 4 years of treatment in short children born SGA. Its favorable safety and adherence profile supports long-acting GH as an alternative to daily GH therapy for managing growth failure in SGA children.

Source: Hormone Research in Paediatrics

DOI: 10.1159/hrp/adaag021