Pharmacological interventions for short stature: Pros and cons

Although growth hormone (GH) therapy is virtually always effective in accelerating growth and restoring height potential to children with GH deficiency (GHD), the expansion of its use to a wide variety of other clinical disorders associated with short stature has resulted in considerable ethical and cost-benefit issues. Logic would demand that therapy should either be restricted to true 'replacement', thereby limiting its use to cases of unequivocal GHD, or treatment should be considered as a legitimate 'enhancement', and be available to all children with significant short stature. Consideration of the latter option requires a careful look at issues surrounding efficacy (both in terms of stature and any perceived disability resulting therefrom), cost and potential adverse effects. Similar concerns involve treatment with insulin-like growth factor-I and any related growth-augmenting therapy. To date, safety issues have been addressed through pharmaceutical-sponsored postmarketing surveillance studies. While of definite use, such investigations also have significant limitations, especially in addressing long-term concerns. The possibility of lifespan cohort studies, with surveillance of all GH recipients throughout life and comparison with data from appropriate controls, should be considered.