Safety and efficacy of pediatric growth hormone therapy: results from the full KIGS cohort
Open accessAbstract
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.
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Data from GH registries provide no evidence of an increase in risk of neoplasia with treatment.
“data from GH registries provide no evidence of an increase in risk of neoplasia with treatment.173,208,222-224”
From Clinical practice guidelines for the care of girls and women with Turner syndrome. (2024) · cites this paper as [173]
No overall increase in mortality due to GH relative to other GH-treated pediatric populations has been reported in TS individuals in GH registries.
“No overall increase in mortality due to GH relative to other GH-treated pediatric populations has been reported in individuals with TS followed in the GH registries.173,197,208,225”
From Clinical practice guidelines for the care of girls and women with Turner syndrome. (2024) · cites this paper as [173]
Individuals with TS appear to be at increased risk of intracranial hypertension, slipped capital femoral epiphysis, and scoliosis during GH treatment compared with children with idiopathic GH deficiency or idiopathic short stature.
“Individuals with TS appear to be at increased risk of intracranial hypertension and slipped capital femoral epiphysis208 as well as scoliosis173,198,208 during GH treatment compared with children with idiopathic GH deficiency or idiopathic short stature.”
From Clinical practice guidelines for the care of girls and women with Turner syndrome. (2024) · cites this paper as [173]
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References 53 · Cited by 120
- Authors
- 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.
- Affiliations
- Istituto Giannina Gaslini (IT)
- University Children's Hospital Tübingen (DE)
- Children's Hospital Los Angeles (US)
- Panagiotis & Aglaia Kyriakou Children's Hospital (GR)
- Hospital Sant Joan de Déu Barcelona (ES)
- Pfizer (United States) (US)
- University of Auckland (NZ)
- PendoCon , Putte 2580 , Belgium
- Pfizer (Belgium) (BE)
- Assistance Publique – Hôpitaux de Paris (FR)
- Medical University of Lodz (PL)
- Manchester Academic Health Science Centre (GB)
- İstanbul University, Istanbul Faculty of Medicine, Pediatric Endocrinology Unit , İstanbul 34452 , Turkey
- Erasmus MC (NL)
- National Center For Child Health and Development (JP)
- Tanaka Growth Clinic (JP)
- Friedrich-Alexander-Universität Erlangen-Nürnberg (DE)
- University of Gothenburg (SE)
- Hôpital Necker-Enfants Malades (FR)
- Children's Hospital of Philadelphia (US)
- Corresponding author
- Grimberg, Adda
- ORCID
- 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
- Horikawa, Reiko
- Tanaka, Toshiaki
- Dörr, Helmuth-Günther
- Albertsson-Wikland, Kerstin
- Polak, Michel
- Grimberg, Adda
- Venue
- The Journal of Clinical Endocrinology & Metabolism
- Publisher
- The Endocrine Society
- Published
- 2022-09-14
- Volume
- 107
- Issue
- 12
- Pages
- 3287-3301
- ISSN
- 0021-972X (ISSN-L)
- 1945-7197
- PMC
- PMC9693805
- OpenAlex
- W4295755914
- Cited by
- 120
- References
- 53
- Citations by year
- 2026: 26 · 2025: 43 · 2024: 36 · 2023: 13 · 2022: 2
- FWCI
- 17.40
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- The Journal of Clinical Endocrinology & Metabolism (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (publishedVersion, cc-by-nc-nd)
- Research Explorer (The University of Manchester) (submittedVersion, cc-by-nc-nd)
- EUR Research Repository (Erasmus University Rotterdam) (submittedVersion, cc-by-nc-nd)
- CINECA IRIS Institutial Research Information System (University of Genoa) (submittedVersion)
- Funders
- Pfizer
- Topics
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Growth Hormone and Insulin-like Growth Factors
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Pituitary Gland Disorders and Treatments
- Health Sciences › Medicine › Reproductive Medicine › Hypothalamic control of reproductive hormones
- Keywords
- recombinant human growth hormone, pediatric growth hormone therapy, growth disorders, idiopathic growth hormone deficiency, idiopathic short stature, Turner syndrome, small for gestational age, near-adult height, height gain, adverse events, scoliosis, real-world evidence, treatment efficacy and safety
- MeSH terms
- Adult, Body Height, Child, Dwarfism, Pituitary, Female, Growth Disorders, Humans, Male, Recombinant Proteins, Growth Hormone, Human Growth Hormone
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