Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort

Maghnie M, Ranke MB, Geffner ME, Vlachopapadopoulou E, Ibanez L, Carlsson M, Cutfield W, Rooman R, Gomez R, Wajnrajch MP, Linglart A, Stawerska R, Clayton PE, Darendeliler F, Hokken-Koelega ACS, Horikawa R, Tanaka T, Dorr HG, Albertsson-Wikland K, Polak M, Grimberg A (2022)

Publication Type: Journal article

Publication year: 2022

Journal

DOI: 10.1210/clinem/dgac517

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.

Involved external institutions

Pfizer, Inc. United States (USA) (US) Istituto Giannina Gaslini Italy (IT) Hospital Sant Joan de Déu Barcelona Spain (ES) Bicêtre Hospital France (FR) Istanbul University / İstanbul Üniversitesi Turkey (TR) National Center for Child Health and Development Japan (JP) Instytut Centrum Zdrowia Matki Polki w Łodzi (ICZMP) / Polish Mother’s Memorial Hospital Research Institute Poland (PL) Tanaka Growth Clinic Japan (JP) University of Gothenburg / Göteborgs universitet Sweden (SE) PendoCon B.V. Belgium (BE) Université de Paris France (FR) Athens General Children’s Hospital “Pan. & Aglaia Kyriakou” Greece (GR) Children's Hospital Los Angeles United States (USA) (US) University of Auckland New Zealand (NZ) Universitätsklinikum Tübingen Germany (DE) University of Manchester United Kingdom (GB) Children's Hospital of Philadelphia United States (USA) (US) Erasmus University Medical Center (MC) Netherlands (NL)

How to cite

APA:

Maghnie, M., Ranke, M.B., Geffner, M.E., Vlachopapadopoulou, E., Ibanez, L., Carlsson, M.,... Grimberg, A. (2022). Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort. Journal of Clinical Endocrinology and Metabolism. https://doi.org/10.1210/clinem/dgac517

MLA:

Maghnie, Mohamad, et al. "Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort." Journal of Clinical Endocrinology and Metabolism (2022).

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