Comparative outcomes of GH treatment in pediatric idiopathic short stature and GH deficiency

Context: GH treatment in children with idiopathic short stature (ISS) can be controversial, and analyses comparing responses to children with GH deficiency (GHD) are limited. Objective: To compare the effectiveness and safety of GH treatment in children with ISS and GHD, including those reaching near adult height (NAH). Methods: This post hoc analysis of the NordiNet International Outcome Study (2006-2016) and the American Norditropin Studies: Web-Enabled Research Program (2002-2016) included children with ISS or GHD who initiated treatment aged <18 years. The safety analysis set had birthdate and GH exposure information. The effectiveness analysis set was GH-naïve with valid baseline information. GH exposure, effectiveness, and safety outcomes were analyzed annually for ≤10 years. Results: The safety analysis set included 3816 children with ISS and 22 858 with GHD. The effectiveness analysis set comprised 18 405 children (ISS: 2684