Still too little, too late? Ten years of growth hormone therapy baseline data from the NordiNet ® International Outcome Study

Polak, Michel; Konrad, Daniel; Tønnes Pedersen, Birgitte; Puras, Gediminas; Šnajderová, Marta

doi:10.1515/jpem-2017-0489

Cited by 19Growth Hormone and Insulin-like Growth FactorsHypothalamic control of reproductive hormonesPituitary Gland Disorders and Treatments

Still too little, too late? Ten years of growth hormone therapy baseline data from the NordiNet ® International Outcome Study

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Polak, Michel, Konrad, Daniel, Tønnes Pedersen, Birgitte, Puras, Gediminas, Šnajderová, Marta·2018·Journal of Pediatric Endocrinology and Metabolism

DOI 10.1515/jpem-2017-0489

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Abstract

BACKGROUND: We investigated time trends in age, gender, growth hormone (GH) dose and height standard deviation score (SDS) in children with GH deficiency (GHD), born small for gestational age (SGA) or with Turner syndrome (TS) starting GH treatment. METHODS: Data were obtained from children enrolled in the NordiNet® International Outcome Study (IOS) between 2006 and 2015 in the Czech Republic, France, Germany, Serbia and Montenegro (all indications), and Switzerland and the UK (GHD only). Trends were analyzed by linear regression. Patients were divided by age into early-, medium- or late-start groups in three different time periods. RESULTS: Approximately one-third of children starting treatment for GHD were girls, with no apparent increase in proportion over time. The mean baseline age for starting treatment decreased significantly (p<0.001) for both GHD and SGA in the Czech Republic and Germany. In the other countries studied, over 40% of children started treatment for GHD and SGA late (girls >10, boys >11 years) between 2013 and 2015. The mean baseline GH doses were largely within recommended ranges for GHD and SGA, but below the lowest recommended starting dose for TS in almost every year since 2011 except in France. CONCLUSIONS: Approximately one-third of children starting treatment for GHD were girls. Between 2013 and 2015, more than 40% of children started treatment for GHD and SGA late except in Germany and the Czech Republic. TS patients received below-recommended doses. These results highlight the need for earlier identification of short stature in children, particularly girls, and for dose optimization in TS.

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Observational data show reduced efficacy when prescribed GH doses are lower than recommended doses.
“Observational data185-188 show reduced efficacy when prescribed doses are lower than recommended doses.”
From Clinical practice guidelines for the care of girls and women with Turner syndrome. (2024) · cites this paper as [188]

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[10]10. USA, and Japan: 25 years' KIGS ® experience (1987-2012) and review. Horm Res Paediatr 2017;87:30-41.

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Clinical practice guidelines for the care of girls and women with Turner syndrome.
Gravholt, C. H., Andersen, N. H., Christin-Maitre, S., Davis, S. M., Duijnhouwer, A., Gawlik, A., Maciel-Guerra, A. T., Gutmark-Little, I., Fleischer, K., Hong, D., et al. (2024) · European Journal of Endocrinology · doi:10.1093/ejendo/lvae050 · Cited by 224 · see citing works

Authors: Polak, M., Konrad, D., Tønnes Pedersen, B., Puras, G., & Šnajderová, M.
Affiliations: Hôpital Necker-Enfants Malades (FR)
University Children's Hospital Zurich (CH)
Epidemiology, Novo Nordisk A/S , Søborg , Denmark
Novo Nordisk (Switzerland) (CH)
Charles University (CZ)
Corresponding author: Polak, Michel
ORCID: Polak, Michel
Konrad, Daniel
Šnajderová, Marta
Venue: Journal of Pediatric Endocrinology and Metabolism
Publisher: Walter de Gruyter GmbH
Published: 2018-04-13
Volume: 31
Issue: 5
Pages: 521-532
ISSN: 0334-018X (ISSN-L)
2191-0251
DOI: 10.1515/jpem-2017-0489
OpenAlex: W2798061635
Cited by: 19
References: 42
Citations by year: 2026: 3 · 2024: 4 · 2023: 1 · 2022: 3 · 2021: 4 · 2020: 2 · 2019: 2
FWCI: 1.60
Type: journal article
Language: en
Open access: bronze
doi.org
Locations: Journal of Pediatric Endocrinology and Metabolism (publishedVersion)
PubMed (publishedVersion)
Zurich Open Repository and Archive (University of Zurich) (acceptedVersion, other-oa)
Zurich Open Repository and Archive (University of Zurich) (submittedVersion, other-oa)
Zurich Open Repository and Archive (University of Zurich)
Funders: Ipsen
Topics: Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Growth Hormone and Insulin-like Growth Factors
Health Sciences › Medicine › Reproductive Medicine › Hypothalamic control of reproductive hormones
Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Pituitary Gland Disorders and Treatments
Keywords: growth hormone therapy, growth hormone deficiency, small for gestational age, Turner syndrome, short stature, treatment initiation timing, growth hormone dose, sex differences, height standard deviation score, dose optimization
MeSH terms: Body Height, Child, Dose-Response Relationship, Drug, Female, France, Germany, Growth Disorders, Humans, Male, Outcome Assessment, Health Care, Czech Republic, Human Growth Hormone, Serbia

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Still too little, too late? Ten years of growth hormone therapy baseline data from the NordiNet ® International Outcome Study

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