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Cited by 19Growth Hormone and Insulin-like Growth FactorsPituitary Gland Disorders and TreatmentsHypothalamic control of reproductive hormones

Real-life GH dosing patterns in children with GHD, TS or born SGA: a report from the NordiNet® international outcome study

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Blankenstein, Oliver, Snajderova, Marta, Blair, Jo, Pournara, Effie, Pedersen, Birgitte Tønnes, Petit, Isabelle Oliver·2017·European Journal of Endocrinology
DOI 10.1530/eje-16-1055PMC PMC5488395
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Abstract

Objective To describe real-life dosing patterns in children with growth hormone deficiency (GHD), born small for gestational age (SGA) or with Turner syndrome (TS) receiving growth hormone (GH) and enrolled in the NordiNet International Outcome Study (IOS; Nbib960128) between 2006 and 2016. Design This non-interventional, multicentre study included paediatric patients diagnosed with GHD (isolated (IGHD) or multiple pituitary hormone deficiency (MPHD)), born SGA or with TS and treated according to everyday clinical practice from the Czech Republic (IGHD/MPHD/SGA/TS: n = 425/61/316/119), France ( n = 1404/188/970/206), Germany ( n = 2603/351/1387/411) and the UK ( n = 259/60/87/35). Methods GH dosing was compared descriptively across countries and indications. Proportions of patients by GH dose group (low/medium/high) or GH dose change (decrease/increase/no change) during years 1 and 2 were also evaluated across countries and indications. Results In the Czech Republic, GH dosing was generally within recommended levels. In France, average GH doses were higher for patients with IGHD, MPHD and SGA than in other countries. GH doses in TS tended to be at the lower end of the recommended label range, especially in Germany and the UK; the majority of patients were in the low-dose group. A significant inverse association between baseline height standard deviation score and GH dose was shown ( P < 0.05); shorter patients received higher doses. Changes in GH dose, particularly increases, were more common in the second (40%) than in the first year (25%). Conclusions GH dosing varies considerably across countries and indications. In particular, almost half of girls with TS received GH doses below practice guidelines and label recommendations.

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  1. [29]Bondy CA & Turner Syndrome Study Group. Care of girls and women with Turner syndrome: a guideline of the Turner Syndrome Study Group. Journal of Clinical Endocrinology and Metabolism 2007 92 10– 25. (10.1210/jc.2006-1374) 17047017 · DOI · Paper page

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Authors
Blankenstein, O., Snajderova, M., Blair, J., Pournara, E., Pedersen, B. T., & Petit, I. O.
Affiliations
  • Charité – Universitätsmedizin Berlin (DE)
  • 22nd Faculty of Medicine, Charles University and University Hospital Motol, Prague, Czech Republic
  • 3Alder Hey Children’s NHS Foundation Trust, Liverpool, UK
  • Novo Nordisk (Switzerland) (CH)
  • Novo Nordisk (Denmark) (DK)
  • Hôpital des Enfants (FR)
Corresponding author
Blankenstein, Oliver
ORCID
Venue
European Journal of Endocrinology
Publisher
Oxford University Press (OUP)
Published
2017-08
Volume
177
Issue
2
Pages
145-155
ISSN
  • 0804-4643 (ISSN-L)
  • 1479-683X
DOI
10.1530/eje-16-1055
PMC
PMC5488395
OpenAlex
W2614506492
Cited by
19
References
37
Citations by year
2026: 1 · 2025: 2 · 2024: 6 · 2022: 1 · 2021: 1 · 2019: 2 · 2018: 6
FWCI
1.73
Type
journal article
Language
en
Open access
Licence
Locations
  • European Journal of Endocrinology (publishedVersion, cc-by-nc)
  • PubMed Central (publishedVersion, cc-by)
  • PubMed (publishedVersion)
Funders
Novo Nordisk
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
growth hormone deficiency, Turner syndrome, small for gestational age, isolated growth hormone deficiency, multiple pituitary hormone deficiency, dose adjustment, height standard deviation score, clinical practice variation
MeSH terms
Adolescent, Child, Child, Preschool, Cross-Sectional Studies, Dose-Response Relationship, Drug, Dwarfism, Pituitary, Female, Follow-Up Studies, France, Germany, United Kingdom, Growth Disorders, Humans, Infant, Small for Gestational Age, Male, Prospective Studies, Turner Syndrome, Treatment Outcome, Czech Republic, Human Growth Hormone, Internationality, Research Report

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