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

作者：Mohamad Maghnie, Michael B. Ranke, Mitchell E. Geffner, Elpis Athina Vlachopapadopoulou, Lourdes Ibáñez, Martin Ove Carlsson, Wayne S. Cutfield, Raoul Rooman, Roy Gomez, Michael Paul Wajnrajch, Agnès Linglart, Renata Stawerska, Peter Clayton, Feyza Darendelıler, Anita Hokken-Koelega, Reiko Horikawa, Toshiaki Tanaka, Helmuth‐Günther Dörr, Kerstin G. Albertsson-Wikland, Michel Polak, Adda Grimberg · 发表于：The Journal of Clinical Endocrinology & Metabolism · 年份：2022 · DOI：10.1210/clinem/dgac517 · 被引用次数：117 · 研究领域：Growth Hormone and Insulin-like Growth Factors、Pituitary Gland Disorders and Treatments、Genetic Syndromes and Imprinting

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),...