Recombinant GH Therapy: Expected Results, Modalities and Monitoring in Children

The past century witnessed the evolution of growth hormone (GH) treatment from pituitary-derived GH, available for treatment of selected cases of GH deficiency (GHD), to recombinant human GH (rhGH), currently approved for several clinical disorders besides GHD. Treatment with rhGH has a proven efficacy in improving short stature in patients affected by GHD, alongside beneficial effects on metabolic and bone health. Long-term safety of rhGH treatment has been intensively investigated, providing reassuring results, especially in patients treated for isolated GHD. The use of rhGH in supraphysiological doses in conditions other than GHD and the intrinsic risk factors associated with certain clinical conditions prompt the need to prolong surveillance studies on rhGH safety. Finally, long-acting rhGH formulations are being introduced into the market, with the goal of increasing adherence and reducing treatment burden for patients on rhGH treatment. In this review, we will discuss indications, efficacy, and safety profile of treatment with rhGH in pediatric patients affected by GHD. We will also briefly discuss the newer formulation of rhGH.