Therapy of growth disorders

We review recent developments in the therapy of growth disorders, focusing mainly on recent developments in the field of growth hormone (GH) therapy.

Review of current practice reveals wide variation in the approach to short stature and the institution of GH therapy. No new indications for GH treatment have been approved by the Food and Drug Administration since 2007, but off-label use has been explored in several chronic illnesses. There is also increasing focus on GH effects beyond increase in stature (e.g. effects on body composition and bone density). As the experience with GH increases, the long-term risks and outcomes are better understood and risks appear to be low. Data regarding beneficial effects of GH in Prader-Willi syndrome are accumulating but questions remain about patient selection, start age and safety. Recombinant insulin-like growth factor 1 has become more widely available and has been aggressively promoted for Food and Drug Administration-approved indications as well as off-label uses in the last few years. The use of aromatase inhibitors has been tempered by concern about side-effects and lack of efficacy.

Growth hormone remains the mainstay of therapy for growth disorders, though other options, including recombinant insulin-like growth factor 1, are being investigated in various settings.