The Efficacy and Safety of Different Growth Hormone Treatment Regimens in Children with Idiopathic Short Stature

DOI: 10.12201/bmr.202511.00012

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  Abstract: Abstract Objective To evaluate the efficacy, safety, and adherence of different growth hormone treatment regimens in children with idiopathic short stature (ISS). Method A total of 67 children diagnosed with ISS at Haining People’s Hospital were retrospectively analyzed and categorized into three groups according to treatment regimen: the long-acting group (n = 16), the switch group (n = 14), and the short-acting group (n = 37). The long-acting group received polyethylene glycol recombinant human growth hormone (PEG-rhGH) throughout treatment, the switch group initially received PEG-rhGH and was later switched to recombinant human growth hormone (rhGH), and the short-acting group received rhGH for the entire treatment period. Growth and safety indicators were compared before treatment and at 3, 6, and 9 months of therapy. Result No significant differences were observed among the three groups in height standard deviation score (Ht SDS), change in Ht SDS (ΔHt SDS), glucose metabolism, liver and kidney function, or thyroid function at any time point (all P > 0.05). Serum insulin-like growth factor-1 (IGF-1) levels in the switch group were significantly higher than those in the other two groups at 3, 6, and 9 months (all P < 0.05). The missed-injection rates were 0%, 7.1%, and 34.3% in the long-acting, switch, and short-acting group, respectively, with the short-acting group showing a significantly higher rate than the long-acting group (Z＝4.47, P＜0.001). No serious adverse events were observed in any group. Conclusion The three growth hormone regimens demonstrated comparable efficacy and safety in children with ISS. The switch group showed better treatment adherence, reducing the missed-injection rate by approximately 27% compared with the short-acting group. However, serum IGF-1 levels should be closely monitored after switching formulations.

  Key words: polyethylene glycol recombinant human growth hormone; idiopathic short stature; efficacy; safety; adherence

  Submit time: 8 November 2025

  Copyright: The copyright holder for this preprint is the author/funder, who has granted biomedRxiv a license to display the preprint in perpetuity.
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