1. 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).
- Author
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Pfäffle R, Land C, Schönau E, Holterhus PM, Ross JL, Piras de Oliveira C, Child CJ, Benabbad I, Jia N, Jung H, and Blum WF
- Subjects
- Adolescent, Body Height genetics, Child, Child, Preschool, Cohort Studies, Dwarfism, Pituitary drug therapy, Dwarfism, Pituitary epidemiology, Dwarfism, Pituitary genetics, Female, France epidemiology, Genetics, Population, Germany epidemiology, Growth Disorders genetics, Humans, Internationality, Male, Neuroendocrinology, Population Surveillance methods, Treatment Outcome, United States epidemiology, Growth Disorders drug therapy, Growth Disorders epidemiology, Human Growth Hormone therapeutic use
- Abstract
Background/aims: 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., Methods: 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., Results: Children in the USA (n = 9,810), Germany (n = 2,682) and France (n = 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 > 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 >-2). No new or unexpected safety concerns were noted., Conclusion: 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 > 60% of patients to GeNeSIS, indicated no new safety signals and the benefit-risk profile of GH remains unchanged., (© 2018 S. Karger AG, Basel.) more...
- Published
- 2018
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