PMID- 36102184 OWN - NLM STAT- MEDLINE DCOM- 20221128 LR - 20230603 IS - 1945-7197 (Electronic) IS - 0021-972X (Print) IS - 0021-972X (Linking) VI - 107 IP - 12 DP - 2022 Nov 25 TI - Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort. PG - 3287-3301 LID - 10.1210/clinem/dgac517 [doi] AB - 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. CI - (c) The Author(s) 2022. Published by Oxford University Press on behalf of the Endocrine Society. FAU - Maghnie, Mohamad AU - Maghnie M AUID- ORCID: 0000-0002-7183-5238 AD - Department of Pediatrics, IRCCS Giannina Gaslini, Genova 16124, Italy. AD - Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, University of Genova, Genova 16124, Italy. FAU - Ranke, Michael B AU - Ranke MB AUID- ORCID: 0000-0002-3855-6157 AD - Department of Pediatric Endocrinology, University Children s Hospital, Tubingen 72076, Germany. FAU - Geffner, Mitchell E AU - Geffner ME AUID- ORCID: 0000-0002-0384-3287 AD - The Saban Research Institute, Children's Hospital Los Angeles, Los Angeles, California 90027, USA. FAU - Vlachopapadopoulou, Elpis AU - Vlachopapadopoulou E AUID- ORCID: 0000-0001-5641-7081 AD - Department of Endocrinology, Growth and Development, Aglaia Kyriakou Children's Hospital, Athens 11527, Greece. FAU - Ibanez, Lourdes AU - Ibanez L AD - Endocrinology, Pediatric Research Institute Sant Joan de Deu, Barcelona 08950, Spain. AD - Centro de Investigacion Biomedica en Red de Diabetes y Enfermedades Metabolicas Asociadas (CIBERDEM), Instituto de Salud Carlos III, Madrid 28029, Spain. FAU - Carlsson, Martin AU - Carlsson M AUID- ORCID: 0000-0003-3106-0754 AD - Rare Disease, Biopharmaceuticals, Pfizer, New York, NY 10017, USA. FAU - Cutfield, Wayne AU - Cutfield W AUID- ORCID: 0000-0001-6107-3682 AD - Liggins Institute, University of Auckland, Auckland 1142, New Zealand. FAU - Rooman, Raoul AU - Rooman R AD - PendoCon, Putte 2580, Belgium. FAU - Gomez, Roy AU - Gomez R AUID- ORCID: 0000-0001-7029-1542 AD - European Medical Affairs, Pfizer, Brussels 1070, Belgium. FAU - Wajnrajch, Michael P AU - Wajnrajch MP AUID- ORCID: 0000-0002-8211-9237 AD - Rare Disease, Biopharmaceuticals, Pfizer, New York, NY 10017, USA. AD - Department of Pediatrics, New York University Langone Medical Center, New York, NY 10016, USA. FAU - Linglart, Agnes AU - Linglart A AUID- ORCID: 0000-0003-3455-002X AD - Department of Pediatric Endocrinology and Diabetology for Children, AP-HP, Bicetre Paris Saclay, Le Kremlin Bicetre 94270, France. AD - APHP, Reference Center for Rare Disorders of the Calcium and Phosphate Metabolism, Filiere OSCAR and Plateforme d'Expertise Maladies Rares Paris-Sud, Bicetre Paris Saclay Hospital, Le Kremlin Bicetre 94270, France. FAU - Stawerska, Renata AU - Stawerska R AUID- ORCID: 0000-0002-0508-2186 AD - Department of Endocrinology and Metabolic Diseases, Polish Mother's Memorial Hospital-Research Institute, Lodz 93-338, Poland. AD - Department of Pediatric Endocrinology, Medical University of Lodz, Lodz 93-338, Poland. FAU - Clayton, Peter E AU - Clayton PE AD - Developmental Biology and Medicine, Faculty of Biology Medicine and Health, Manchester NIHR Academic Health Science Centre, University of Manchester, Manchester M13 9PL, UK. FAU - Darendeliler, Feyza AU - Darendeliler F AUID- ORCID: 0000-0003-4786-0780 AD - Istanbul University, Istanbul Faculty of Medicine, Pediatric Endocrinology Unit, Istanbul 34452, Turkey. FAU - Hokken-Koelega, Anita C S AU - Hokken-Koelega ACS AUID- ORCID: 0000-0002-1384-7628 AD - Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center, Rotterdam 3015 GD, the Netherlands. FAU - Horikawa, Reiko AU - Horikawa R AUID- ORCID: 0000-0003-3429-3246 AD - Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo 157-8535, Japan. FAU - Tanaka, Toshiaki AU - Tanaka T AD - Tanaka Growth Clinic, Tokyo 158-0097, Japan. FAU - Dorr, Helmuth-Gunther AU - Dorr HG AD - Division of Pediatric Endocrinology, Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander University of Erlangen-Nurnberg, Erlangen 91054, Germany. FAU - Albertsson-Wikland, Kerstin AU - Albertsson-Wikland K AUID- ORCID: 0000-0003-3167-0667 AD - Department of Physiology/Endocrinology, Institute of Neuroscience and Physiology, Sahlgrenska Academy, University of Gothenburg, Gothenburg 405 30, Sweden. FAU - Polak, Michel AU - Polak M AUID- ORCID: 0000-0003-2639-352X AD - Universite de Paris Cite; Hopital Universitaire Necker Enfants Malades, Paris 75015, France. FAU - Grimberg, Adda AU - Grimberg A AUID- ORCID: 0000-0002-1523-1536 AD - Division of Pediatric Endocrinology and Diabetes, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA. LA - eng GR - Pfizer/ PT - Journal Article PT - Research Support, Non-U.S. Gov't PL - United States TA - J Clin Endocrinol Metab JT - The Journal of clinical endocrinology and metabolism JID - 0375362 RN - 12629-01-5 (Human Growth Hormone) RN - 9002-72-6 (Growth Hormone) RN - 0 (Recombinant Proteins) SB - IM CIN - J Clin Endocrinol Metab. 2022 Dec 17;108(1):e1-e2. PMID: 36285732 CIN - J Clin Endocrinol Metab. 2023 May 17;108(6):e354-e355. PMID: 36719012 CIN - J Clin Endocrinol Metab. 2023 May 17;108(6):e356-e357. PMID: 36721915 MH - Adult MH - Female MH - Child MH - Humans MH - Male MH - *Human Growth Hormone/adverse effects MH - Growth Hormone MH - *Dwarfism, Pituitary MH - Growth Disorders/drug therapy MH - Body Height MH - Recombinant Proteins/adverse effects PMC - PMC9693805 OTO - NOTNLM OT - KIGS OT - children OT - efficacy OT - growth hormone OT - safety OT - short stature EDAT- 2022/09/15 06:00 MHDA- 2022/11/29 06:00 PMCR- 2022/09/14 CRDT- 2022/09/14 04:53 PHST- 2022/03/30 00:00 [received] PHST- 2022/09/15 06:00 [pubmed] PHST- 2022/11/29 06:00 [medline] PHST- 2022/09/14 04:53 [entrez] PHST- 2022/09/14 00:00 [pmc-release] AID - 6697043 [pii] AID - dgac517 [pii] AID - 10.1210/clinem/dgac517 [doi] PST - ppublish SO - J Clin Endocrinol Metab. 2022 Nov 25;107(12):3287-3301. doi: 10.1210/clinem/dgac517.