Growth Hormone Treatment for Short Stature in the USA, Germany and France: 15 Years of Surveillance in the Genetics and Neuroendocrinology of Short-Stature International Study (GeNeSIS)

<b><i>Background/Aims:</i></b> To describe characteristics, auxological outcomes and safety in paediatric patients with growth disorders treated with growth hormone (GH), for cohorts from the USA, Germany and France enrolled in GeNeSIS, a post-authorisation surveillance programme. <b><i>Methods:</i></b> Diagnosis and biochemical measurement data were based on reporting from, and GH treatment was initiated at the discretion of, treating physicians. Auxological outcomes during the first 4 years of GH treatment and at near-adult height (NAH) were analysed. Serious and treatment-emergent adverse events were described. <b><i>Results:</i></b> Children in the USA (<i>n</i> = 9,810), Germany (<i>n</i> = 2,682) and France (<i>n</i> = 1,667) received GH (dose varied between countries), most commonly for GH deficiency. Across diagnostic groups and countries, mean height velocity standard deviation score (SDS) was &#x3e; 0 and height SDS increased from baseline during the first 4 years of treatment, with greatest improvements during year 1. Most children achieved NAH within the normal range (height SDS &#x3e;−2). No new or unexpected safety concerns were noted. <b><i>Conclusion:</i></b> GH treatment improved growth indices to a similar extent for patients in all three countries despite variations in GH doses. Data from these three countries, which together contributed &#x3e; 60% of patients to GeNeSIS, indicated no new safety signals and the benefit-risk profile of GH remains unchanged.