Clinical Indications for Growth Hormone Therapy

Key points

Introduction

Recombinant human growth hormone (GH) first received approval from the United States Food and Drug Administration (FDA) for clinical treatment in 1985 for pediatric GH deficiency, and its indications subsequently have expanded. Short stature is one of the most common chief comlpaints for referral to a pediatric endocrinologist. Short stature may be secondary to an underlying genetic abnormality, malnutrition, or systemic condition, or can present in a healthy child. There are currently 8 FDA-approved indications for pediatric GH therapy in the United States. Of these, short stature in GH deficiency and Prader–Willi syndrome is due to a deficiency of GH. The other 6 indications do not involve GH deficiency; treatment aims to augment height by adding to the body’s own endogenous GH production. We will describe the characteristic features for each of these indications, as well as summarize the use of GH, other endocrine manifestations of the conditions, and relevant diagnostic testing. We also will review patterns of growth that warrant referral to a pediatric endocrinologist as well as safety updates on the use of GH treatment.