Key Takeaways & Executive Findings
- •• Recombinant human growth hormone (rhGH) significantly improves height SDS and growth velocity in children with growth hormone deficiency. • A higher dose of rhGH (0.2 IU/kg/day) results in greater growth outcomes compared to a lower dose (0.1 IU/kg/day). • rhGH treatment is generally safe, with mild and transient adverse events such as injection site reactions and elevated liver enzymes. • The study supports the use of rhGH as an effective therapy for pediatric GHD, with dose optimization important for maximizing growth benefits.
Abstract
Objective: To evaluate the efficacy and safety of recombinant human growth hormone (rhGH) in the treatment of growth hormone deficiency (GHD) in children. Methods: A multicenter, randomized, open-label, parallel-controlled clinical trial was conducted. A total of 240 children with GHD were randomly assigned to receive either rhGH at a dose of 0.1 IU/kg/day (low-dose group) or 0.2 IU/kg/day (high-dose group) for 12 months. The primary efficacy endpoint was the change in height standard deviation score (Ht SDS) from baseline. Secondary endpoints included growth velocity (GV), insulin-like growth factor-1 (IGF-1) levels, and safety parameters. Results: After 12 months of treatment, the high-dose group showed a significantly greater increase in Ht SDS (1.2 ± 0.3) compared to the low-dose group (0.8 ± 0.2) (P < 0.05). GV was also significantly higher in the high-dose group (10.5 ± 2.1 cm/year) than in the low-dose group (8.7 ± 1.8 cm/year) (P < 0.05). IGF-1 levels increased significantly in both groups, with no significant difference between groups. Adverse events were reported in 15% of patients in the low-dose group and 20% in the high-dose group, with the most common being injection site reactions and transient increases in liver enzymes. No serious adverse events were observed. Conclusion: rhGH is effective and safe for the treatment of GHD in children, with a dose-dependent effect on growth. The high-dose regimen (0.2 IU/kg/day) provides superior growth outcomes without additional safety concerns.
1. Introduction
Growth hormone deficiency (GHD) is a disorder characterized by inadequate secretion of growth hormone from the anterior pituitary gland, leading to short stature and metabolic abnormalities in children. Recombinant human growth hormone (rhGH) has been the mainstay of treatment for GHD since its introduction, effectively improving linear growth and final adult height. However, optimal dosing strategies remain debated, with varying recommendations for starting doses and titration based on individual response and safety profiles.
This multicenter, randomized, open-label, parallel-controlled clinical trial was designed to evaluate the efficacy and safety of two different doses of rhGH (0.1 IU/kg/day vs. 0.2 IU/kg/day) in prepubertal children with GHD. The primary objective was to compare changes in height standard deviation score (Ht SDS) after 12 months of treatment, with secondary endpoints including growth velocity, IGF-1 levels, and safety assessments. The results of this study aim to provide evidence-based guidance for clinicians in selecting appropriate rhGH doses to optimize growth outcomes while minimizing potential risks.
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ZHANG Wei, LI Ming, WANG Fang, et al. (2025). Efficacy and Safety of Recombinant Human Growth Hormone in the Treatment of Growth Hormone Deficiency: A Multicenter, Randomized, Open-Label, Parallel-Controlled Clinical Trial. Chinese Journal of New Drugs. https://doi.org/cast_zgxyzz_1236731777960694027
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Frequently Asked Questions
What is the recommended dose of recombinant human growth hormone for children with growth hormone deficiency?
The recommended dose varies, but this study suggests that a higher dose of 0.2 IU/kg/day may provide superior growth outcomes compared to 0.1 IU/kg/day, with a similar safety profile. However, dosing should be individualized based on clinical response and tolerability.
How effective is recombinant human growth hormone in improving height in children with GHD?
In this trial, after 12 months of treatment, children receiving rhGH showed significant improvements in height SDS and growth velocity. The high-dose group (0.2 IU/kg/day) achieved a mean increase in height SDS of 1.2, while the low-dose group (0.1 IU/kg/day) achieved 0.8.
What are the common side effects of recombinant human growth hormone therapy?
Common side effects include injection site reactions (such as pain, redness, or swelling) and transient increases in liver enzymes. These are generally mild and resolve without intervention. Serious adverse events are rare.
Is recombinant human growth hormone safe for long-term use in children?
Long-term safety data from this study and others indicate that rhGH is generally safe when used under medical supervision. Regular monitoring of growth, IGF-1 levels, and potential side effects is recommended to ensure optimal outcomes.
What is the mechanism of action of recombinant human growth hormone?
Recombinant human growth hormone binds to growth hormone receptors on target cells, stimulating the production of insulin-like growth factor-1 (IGF-1), which mediates many of its growth-promoting effects, including linear bone growth and protein synthesis.
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