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Published on: January 7, 2016
Growth hormone treatment of idiopathic short stature: clinical studies
1Stanford University School of Medicine, 300 Pasteur Drive, Stanford, CA 94305, USA. hintz@stanford.edu
Insights
Growth hormone (GH) treatment for idiopathic short stature in children is now FDA-approved, simplifying selection beyond provocative tests. This allows for better treatment decisions based on height deficit and potential for normal adult height.
Area of Science:
- Pediatrics
- Endocrinology
Background:
- Traditional selection for growth hormone (GH) treatment in short-statured children relied on provocative testing.
- Provocative testing is often unphysiologic, difficult to administer, and potentially dangerous.
Purpose of the Study:
- To evaluate the effectiveness and safety of GH treatment for idiopathic short stature in children.
- To inform treatment decisions based on degree of short stature and potential for normal adult height.
Main Methods:
- Analysis of studies involving GH therapy in children with idiopathic short stature.
- Assessment of height outcomes and safety profiles.
Main Results:
- GH therapy can effectively and safely promote height outcomes within the normal adult range.
- Variability in height response to GH treatment was observed, presenting clinical challenges.
Conclusions:
- FDA approval of GH for idiopathic short stature offers a rational treatment option.
- Treatment decisions can now be guided by height deficit and potential for normal adult height, moving beyond problematic provocative tests.
Abstract:
The selection of short-statured children for growth hormone (GH) treatment has long been complicated by the requirement for provocative testing that is unphysiologic, difficult to administer, and potentially dangerous. The recent FDA approval of GH for the treatment of children with idiopathic short stature allows treatment decisions to be based more on the degree of short stature and the potential for attaining a normal adult height. Several studies conducted in children have shown that GH therapy can effectively and safely produce height outcomes within a normal adult range. The observed variability in height response associated with GH use in some of these studies has left the clinician with a difficult decision. Still, the availability of GH for this patient population provides a rational treatment option for patients who fail arbitrary and inaccurate assessments.
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