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- Title
Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort.
- Authors
Maghnie, Mohamad; Ranke, Michael B.; Geffner, Mitchell E.; Vlachopapadopoulou, Elpis; Ibáñez, Lourdes; Carlsson, Martin; Cutfield, Wayne; Rooman, Raoul; Gomez, Roy; Wajnrajch, Michael P.; Linglart, Agnès; Stawerska, Renata; Clayton, Peter E.; Darendeliler, Feyza; Hokken-Koelega, Anita C. S.; Reiko Horikawa; Toshiaki Tanaka; Dörr, Helmuth-Günther; Albertsson-Wikland, Kerstin; Polak, Michel
- Abstract
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.
- Publication
Journal of Clinical Endocrinology & Metabolism, 2022, Vol 107, Issue 12, p3287
- ISSN
0021-972X
- Publication type
Article
- DOI
10.1210/clinem/dgac517