ArticleEuropean journal of pediatrics2024
Effect of long-acting PEGylated growth hormone for catch-up growth in children with idiopathic short stature: a 2-year real-world retrospective cohort study.
Article in European journal of pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 2 of them syntheses that pooled it.
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Who cites it
14 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Weekly long-acting growth hormone versus daily growth hormone therapy on quality of life, efficacy, and safety in children with short stature: a systematic review and meta-analysis.Frontiers in endocrinology · 2026Pooled it
- Comparison between long-acting pegylated and daily recombinant human growth hormone for pediatric growth hormone deficiency a systematic review.Scientific reports · 2025Pooled it
- Association Between Socioeconomic Indicators and Growth Hormone Treatment Patterns for Idiopathic Short Stature in China: A Large-Scale Real-World Study.Advances in therapy · 2026Article
- 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).Translational pediatrics · 2026Article
- Metabolic and bone effects of long-acting polyethylene glycol-recombinant human growth hormone in growth hormone deficiency during the transition period: a prospective study.Annals of pediatric endocrinology & metabolism · 2026Article
- Can long-acting growth hormone improve adherence in pediatric growth hormone deficiency?Endocrine · 2026Review
- Weekly pegylated recombinant human growth hormone for prepubertal children with growth hormone deficiency: adherence advantage drives superior short-term efficacy over daily rhGH.Translational pediatrics · 2026Article
- Long-Acting PEGylated Growth Hormone in Children with Idiopathic Short Stature: 2-Year Results.Hormone research in paediatrics · 2026Article
- Identification of a novel and a recurrentFrontiers in genetics · 2026Article
- Case Report: Possible improvement of near-adult height with PEG-rhGH and letrozole in a late-puberty boy with atypical Noonan syndrome and bone age/height age discrepancy: a novel therapy.Frontiers in pediatrics · 2026Article
- Efficacy and Safety of PEGylated Recombinant Human Growth Hormone in Children With Growth Hormone Deficiency and Idiopathic Short Stature: A Real-World Cohort Study.International journal of endocrinology · 2026Article
- Comparative Efficacy and Safety of Once-Weekly Pegylated Recombinant Human Growth Hormone Versus Daily Growth Hormone Therapy in Children: A Systematic Review and Meta-Analysis.Journal of clinical medicine · 2025Review
- History and Current Status of Growth Hormone Treatment in Children.Paediatric drugs · 2025Article
- Idiopathic Short Stature in the Genomic Era: Integrating Auxology, Endocrinology, and Emerging Genetic Insights.Children (Basel, Switzerland) · 2025Review
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Several evidence gaps exist regarding the use of long-acting polyethylene glycol recombinant human growth hormone (PEG-rhGH) in children with idiopathic short stature (ISS), particularly studies conducted in real-world settings, with long-term follow-up, involving varied dosing regimens, and in comparison with daily rhGH. The study aimed to evaluate the effectiveness, safety, and adherence of once-weekly PEG-rhGH for catch-up growth in children with prepubertal ISS compared to daily rhGH. A real-world retrospective cohort study was conducted in prepubertal children with ISS in China. Children who voluntarily received once-weekly PEG-rhGH or daily rhGH were included and were followed up for 2 years. Ninety-five children were included, 47 received PEG-rhGH 0.2-0.3 mg/kg weekly and 48 received daily rhGH. Outcome measures included effectiveness in catch-up growth, adverse events, and treatment adherence. Height velocity increased significantly in both groups during rhGH therapy. In children who received PEG-rhGH treatment, height velocity was 10.59 ± 1.37 cm/year and 8.75 ± 0.86 cm/year in the first and second year, respectively, which were significantly more than those who received daily rhGH (9.80 ± 1.05 cm/year, P = 0.002, and 8.03 ± 0.89 cm/year, P < 0.001). The height standard deviation score improved at the end of the second year for all children (P < 0.001). However, children who received PEG-rhGH showed more excellent improvement than those with daily rhGH (1.65 ± 0.38 vs. 1.50 ± 0.36, P = 0.001). In children who received PEG-rhGH, lower missed doses were observed than those with daily rhGH (0.75 ± 1.06 vs. 4.4 ± 2.0, P < 0.001). No serious adverse events were observed.
conclusionPEG-rhGH demonstrated superior effectiveness and adherence compared to daily rhGH in the treatment of children with ISS. The safety profiles were similar between the two treatments. WHAT IS KNOWN: • Recombinant human growth hormone (rhGH) has been used to increase adult height in children with idiopathic short stature (ISS), and its safety profile is comparable to other indications for growth hormone treatment. • The use of long-acting rhGH in children with ISS is still an area of uncertainty. WHAT IS NEW: • This 2-year real-world study provides new evidence that PEGylated rhGH (PEG-rhGH) is more effective than daily rhGH in promoting catch-up growth in children with ISS. • PEG-rhGH also demonstrated superior treatment adherence compared to daily rhGH in children with ISS. • The safety profiles of PEG-rhGH and daily rhGH were found to be similar.
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