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

Oliver Blankenstein1, Marta Snajderova2, Jo Blair3

  • 1Center for Chronic Sick Children, Institute for Experimental Paediatric Endocrinology, Charité - Universitätsmedizin Berlin, Berlin, Germany.

European Journal of Endocrinology
|May 20, 2017
PubMed

Insights

Growth hormone (GH) dosing for children with growth hormone deficiency (GHD), born small for gestational age (SGA), or Turner syndrome (TS) varied significantly by country and indication. Many girls with TS received GH doses below recommended guidelines.

Area of Science:

  • Pediatric Endocrinology
  • Pharmacotherapy
  • Growth Disorders

Background:

  • Growth hormone (GH) therapy is crucial for children with growth hormone deficiency (GHD), those born small for gestational age (SGA), and individuals with Turner syndrome (TS).
  • Understanding real-world GH dosing patterns is essential for optimizing treatment outcomes and adhering to clinical guidelines.

Purpose of the Study:

  • To analyze and compare real-life GH dosing practices in pediatric patients with GHD, SGA, or TS across different European countries.
  • To identify variations in GH dosage based on indication, country, and patient characteristics.

Main Methods:

  • A non-interventional, multicenter study (NordiNet International Outcome Study) involving patients from the Czech Republic, France, Germany, and the UK between 2006 and 2016.
  • Descriptive analysis of GH dosing patterns, including dose groups (low/medium/high) and dose changes over the first two years of treatment, stratified by country and indication.

Main Results:

  • Significant international variations in GH dosing were observed, with France showing higher average doses for GHD and SGA patients.
  • Patients with TS, particularly in Germany and the UK, often received lower GH doses, falling below recommended ranges.
  • A notable inverse relationship was found between baseline height standard deviation score and GH dose, indicating shorter children received higher doses.

Conclusions:

  • Real-world GH dosing for pediatric indications is inconsistent across countries and patient groups.
  • A substantial proportion of girls with Turner syndrome were treated with GH doses below established guidelines and label recommendations, highlighting a need for standardized treatment protocols.
Abstract

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