Growth hormone therapy in children and adolescents: pharmacokinetic/pharmacodynamic considerations and emerging indications

Abstract

Human growth hormone (hGH) has been used for GH deficiency (GHD) for more than 40 years. The availability of hGH produced using recombinant DNA technology beginning in 1985 both eliminated the infectious risk of pituitary-derived hGH and provided a greatly increased supply, allowing for an expanded range of indications. Approved pediatric indications include GHD, idiopathic short stature, children who were small for gestational age with short stature, Turner syndrome, Prader–Willi syndrome, short stature homeobox-containing gene deficiency, Noonan syndrome and chronic renal insufficiency. This review will summarize our current understanding of the pharmacokinetics and pharmacodynamics of hGH, with an emphasis on aspects relevant to recent investigational uses in pediatrics. Promising areas of investigation include cystic fibrosis, juvenile idiopathic arthritis and inflammatory bowel disease. However, given the high cost and potential for rare side effects, controversy remains about the optimal indications, dose and duration of hGH therapy for both the approved and investigational conditions.