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Comparative Outcomes of GH Treatment in Pediatric Idiopathic Short Stature and GH Deficiency

Year: 2025

Type: article

Abstract: 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; GHD: 15 721), 1856 of whom reached NAH (ISS: 230; GHD: 1626). Average dose of GH was higher for children with ISS vs children with GHD but mean duration of treatment was shorter. At NAH, height SD score (mean [SD]) was -1.21 (1.09) and -0.90 (1.20) for children with ISS and GHD, respectively, whereas change in height SD score (mean [SD]) from baseline to 10 years was 1.21 (0.86) and 1.45 (1.09). Incidence of adverse reactions was similar across indications, with no new safety signals. Conclusion: GH treatment over 5 to 10 years effectively increased height in children with ISS and children with GHD, including those who reached NAH, with a favorable benefit-risk profile.

Source: Journal of the Endocrine Society

DOI: 10.1210/jendso/bvaf133