Comparative Outcomes of Growth Hormone Treatment in Pediatric Idiopathic Short Stature and Growth Hormone Deficiency

Context Growth hormone (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 (SAS) had birthdate and GH exposure information. The effectiveness analysis set (EAS) was GH-naïve with valid baseline information. GH exposure, effectiveness, and safety outcomes were analyzed annually, for ≤10 years. Results The SAS included 3816 children with ISS and 22,858 with GHD. The EAS comprised 18,405 children (ISS: 2684; GHD: 15721), 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, while 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 5–10 years effectively increased height in children with ISS and children with GHD, including those who reached NAH, with a favorable benefit-risk profile.