Roland Pfäffle1, Christof Land2, Eckhard Schönau3, Paul-Martin Holterhus4, Judith L Ross5, Carolina Piras de Oliveira6, Christopher J Child7, Imane Benabbad8, Nan Jia9, Heike Jung10, Werner F Blum11. 1. Department of Paediatric Endocrinology, Children's Hospital, Leipzig, Germany. 2. Practice for Children's Endocrinology and Diabetology, Gauting, Germany. 3. Department of Paediatrics, University Hospital of Cologne, Cologne, Germany. 4. Division of Paediatric Endocrinology and Diabetes, Christian-Albrechts-University Kiel and University Hospital Schleswig-Holstein, Kiel, Germany. 5. Department of Pediatrics, Thomas Jefferson University, Philadelphia, Pennsylvania, USA. 6. Lilly USA LLC, Corporate Center, Indianapolis, Indiana, USA. 7. Eli Lilly, Windlesham, United Kingdom. 8. Medical Diabetes, Lilly France, Neuilly-sur-Seine, France. 9. Eli Lilly, Indianapolis, Indiana, USA. 10. Medical Department Diabetes, Lilly Deutschland, Bad Homburg, Germanyjung_heike@lilly.com. 11. Children's Hospital, University of Giessen, Giessen, Germany.
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.
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.
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Authors: Lars Sävendahl; Michel Polak; Philippe Backeljauw; Joanne C Blair; Bradley S Miller; Tilman R Rohrer; Anita Hokken-Koelega; Alberto Pietropoli; Nicky Kelepouris; Judith Ross Journal: J Clin Endocrinol Metab Date: 2021-05-13 Impact factor: 5.958