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Growth hormone and neurofibromatosis
S J Howell1, P Wilton, A Lindberg
1Department of Endocrinology, Christie Hospital NHS Trust, Manchester, UK.
Insights
Growth hormone (GH) therapy improves growth rates in children with neurofibromatosis (NF) and GH deficiency (GHD). This treatment is safe and well-tolerated, without worsening NF symptoms or causing excess adverse events.
Area of Science:
- Pediatric Endocrinology
- Genetics and Genetic Diseases
Background:
- Neurofibromatosis (NF) is a genetic disorder that can lead to growth hormone deficiency (GHD).
- Children with NF and GHD often experience impaired growth.
Purpose of the Study:
- To evaluate the efficacy and safety of growth hormone (GH) replacement therapy in children with NF and GHD.
Main Methods:
- Retrospective review of data from 102 children with NF and GHD receiving GH therapy.
- GH administered at a mean dose of 0.18 mg/kg/week.
Main Results:
- Significant increase in median height velocity from 4.2 cm/year to 7.1 cm/year during the first year of treatment.
- Median height standard deviation score improved from -2.4 to -1.9.
- GH therapy did not affect NF progression or cause excess adverse events.
Conclusions:
- GH treatment is beneficial for improving growth rate in children with NF and GHD.
- GH therapy is well-tolerated in this patient population.
Abstract:
Data collected from 102 neurofibromatosis (NF) children with growth hormone (GH) deficiency (GHD) who were receiving GH replacement therapy were reviewed to assess the efficacy and safety of GH therapy in this condition. GH was administered at a mean dose of 0. 18 mg/kg/week. During the 1st year the median height velocity increased significantly from 4.2 cm/year before treatment to 7.1 cm/year, and the median height standard deviation score increased from -2.4 to -1.9. The response to therapy, however, was not as good as that observed in patients with idiopathic GHD. GH therapy did not influence the progression of any of the features of NF, including intracranial tumours, and was not associated with an excess of other adverse events. We conclude that GH treatment of NF patients with GHD is beneficial in terms of improved growth rate and is well tolerated.