Safety and Efficacy of Pediatric Growth Hormone Therapy

Autor: Mohamad Maghnie, Michael B Ranke, Mitchell E Geffner, Elpis Vlachopapadopoulou, Lourdes Ibáñez, Martin Carlsson, Wayne Cutfield, Raoul Rooman, Roy Gomez, Michael P Wajnrajch, Agnès Linglart, Renata Stawerska, Peter E Clayton, Feyza Darendeliler, Anita C S Hokken-Koelega, Reiko Horikawa, Toshiaki Tanaka, Helmuth-Günther Dörr, Kerstin Albertsson-Wikland, Michel Polak, Adda Grimberg
Přispěvatelé: Pediatrics
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: Journal of Clinical Endocrinology and Metabolism, 107(12), 3287-3301. Endocrine Society
Maghnie, M, Ranke, M B, Geffner, M E, Vlachopapadopoulou, E, Ibáñez, L, Carlsson, M, Cutfield, W, Rooman, R, Gomez, R, Wajnrajch, M P, Linglart, A, Stawerska, R, Clayton, P E, Darendeliler, F, Hokken-Koelega, A C S, Horikawa, R, Tanaka, T, Dörr, H G, Albertsson-Wikland, K, Polak, M & Grimberg, A 2022, ' Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort ', The Journal of Clinical Endocrinology & Metabolism . https://doi.org/10.1210/clinem/dgac517
ISSN: 1945-7197
0021-972X
Popis: Context The Kabi/Pfizer International Growth Database (KIGS) is a large, international database (1987-2012) of children treated with recombinant human growth hormone (rhGH) in real-world settings. Objective This work aimed to evaluate the safety and efficacy of rhGH from the full KIGS cohort. Methods Data were collected by investigators from children with growth disorders treated with rhGH (Genotropin [somatropin]; Pfizer). Safety was evaluated in all treated patients, and efficacy in those treated for 1 year or more. A subgroup included patients treated for 5 years or more (≥ 2 years prepubertal) who had reached near-adult height (NAH). Main outcomes included adverse events (AEs), serious AEs (SAEs), and height growth. Results The full KIGS cohort (N = 83 803 [58% male]) was treated for idiopathic GH deficiency (IGHD; 46.9%), organic GHD (10.0%), small for gestational age (SGA; 9.5%), Turner syndrome (TS; 9.2%), idiopathic short stature (ISS; 8.2%), and others (16.2%). Median rhGH treatment duration was 2.7 years and observation 3.1 years. SAEs occurred in 3.7% of patients and death in 0.4%. The most common SAEs were recurrence of craniopharyngioma (n = 151), neoplasm (n = 99), and cancer (n = 91); and scoliosis (n = 91). Median first-year delta height-SD score (SDS) (Prader) in prepubertal patients was 0.66 (IGHD), 0.55 (ISS), 0.58 (TS), and 0.71 (SGA). Median gains in NAH-SDS were 1.79 (IGHD), 1.37 (ISS), and 1.34 (SGA) for boys, and 2.07 (IGHD), 1.62 (ISS), 1.07 (TS), and 1.57 (SGA) for girls. Conclusion Data from KIGS, the largest and longest running international database of rhGH-treated children, show that rhGH is safe and increases short-term height gain and adult height across GHD and non-GHD conditions.
Databáze: OpenAIRE
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