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Human growth hormone treatment in prepubertal children with achondroplasia
G Weber1, C Prinster, M Meneghel
1Department of Pediatrics, University of Milan, Italy.
Insights
Recombinant human growth hormone (GH) therapy showed potential for increasing growth velocity in some children with achondroplasia. However, responses varied individually, with no significant changes in others.
Area of Science:
- Pediatrics
- Endocrinology
- Genetics
Background:
- Achondroplasia is a common genetic disorder affecting bone growth.
- Growth hormone (GH) therapy is used for various growth deficiencies.
- The efficacy of GH in achondroplasia requires further investigation.
Purpose of the Study:
- To evaluate the effects of recombinant human growth hormone (r-hGH) on growth velocity in prepubertal children with achondroplasia.
- To assess the safety and impact of r-hGH on bone age and craniofacial dimensions.
Main Methods:
- A cohort of 6 prepubertal children with achondroplasia received r-hGH (0.1 IU/kg/day).
- Auxological assessments and bone age were monitored for 12 months.
- MRI and CT scans evaluated craniofacial structures.
Main Results:
- Three patients experienced a growth velocity increase of 1.1–2.6 cm/year.
- No significant growth improvement was observed in the other three patients.
- Two patients showed slight bone age advancement; no changes in foramen magnum dimensions were noted.
Conclusions:
- r-hGH can safely enhance short-term growth velocity in a subset of prepubertal children with achondroplasia.
- Individual variability in response to GH treatment is confirmed.
- Further research is needed to identify predictors of response.
Abstract:
We studied the effects of recombinant human growth hormone (GH) treatment in 6 prepubertal children with achondroplasia. The patients' age ranged from 2 11/12 to 8 5/12 years and the GH dose was of 0.1 IU/kg/day subcutaneously. Auxological assessments and bone age determinations were performed 6 months before, at the beginning, and after 6 and 12 months of therapy. The growth velocity increase during the whole year of treatment ranged from 1.1 to 2.6 cm/year in 3 patients while in the others no variation was detected. No side effects were observed during the trial apart from a slight advancement of bone age in two patients. MRI at the cervicomedullary junction and CT scan of the base of the skull did not show any variation of the dimensions of the foramen magnum at the end of the trial compared to baseline. Our study shows that r-hGH can safely increase short-term growth velocity in some but not all prepubertal children with achondroplasia. Our data confirm the individual variability in the response to the GH treatment.