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A risk-benefit assessment of growth hormone use in children
S L Blethen1, M H MacGillivray
1Genentech Incorporated, South San Francisco, California, USA. blethen.sandra@gene.com
Insights
Recombinant somatropin (growth hormone) effectively treats short stature in children, improving growth rates and final height. Adverse effects are rare in children, differing from adult profiles, with careful monitoring for specific conditions recommended.
Area of Science:
- Pediatrics
- Endocrinology
- Pharmacology
Background:
- Recombinant DNA technology has provided somatropin (growth hormone) for over 11 years.
- Over 38,000 children have been treated with growth hormone products.
- Growth hormone therapy increases statural growth rate in children with short stature from various causes.
Purpose of the Study:
- To review the efficacy and safety of growth hormone treatment in children.
- To assess the impact on final adult height and psychosocial functioning.
- To identify and analyze adverse events in pediatric patients.
Main Methods:
- Analysis of postmarketing safety data from pharmaceutical companies.
- Review of clinical outcomes in over 38,000 treated children.
- Comparison of adverse event profiles between children and adults.
Main Results:
- Growth hormone therapy is most effective in severe growth hormone deficiency but beneficial for various short stature etiologies.
- Benefits include improved growth rate, potential for increased final adult height, enhanced psychosocial functioning, and improved bone mineral density.
- Adverse events in children are uncommon and differ from adults; peripheral edema and carpal tunnel syndrome are rare. Intracranial hypertension and slipped capital femoral epiphyses are rare but potential risks requiring monitoring.
Conclusions:
- Growth hormone treatment is generally safe and effective for pediatric short stature.
- Adverse event profiles in children differ significantly from adults, with specific risks like intracranial hypertension and slipped capital femoral epiphyses needing vigilance.
- Long-term data on final adult height is still limited, especially for idiopathic short stature.
Abstract:
Growth hormone prepared by recombinant DNA technology (somatropin) has been commercially available for over 11 years. More than 38,000 children have been treated with different growth hormone products. While the best response to treatment occurs in children with severe growth hormone deficiency, therapy with growth hormone will increase the rate of statural growth in children with short stature of many different aetiologies. There are few studies of the effect of growth hormone treatment of final adult height, and the magnitude of this effect is harder to gauge, particularly in children with idiopathic short stature. Other benefits of growth hormone treatment in children include improvement in psychosocial functioning and physiological parameters, such as bone mineral density. Adverse effects associated with growth hormone treatment have been relatively uncommon. Most of the safety data on growth hormone have come from large postmarketing databases maintained by 2 pharmaceutical companies. The adverse event profile reported in children treated with growth hormone is different from that found in adults. Peripheral oedema and carpal tunnel syndrome, which are common in adults treated with growth hormone and frequently result in treatment discontinuation, are rare in children. Intracranial hypertension is rare, but can occur in children with growth hormone deficiency, Ullrich-Turner syndrome or renal insufficiency during the first 8 to 12 weeks after the start of growth hormone treatment; it has seldom been reported in adults with growth hormone deficiency. Children with growth hormone deficiency, Ullrich-Turner syndrome or renal insufficiency are prone to develop slipped capital femoral epiphyses both before and during growth hormone treatment. Therefore, limping and complaints of hip or knee pain should be carefully investigated.