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The long-term safety and effectiveness of growth hormone treatment in Japanese children with short stature born small for gestational age

Authors
  • Horikawa, Reiko1
  • Tanaka, Toshiaki2
  • Nishinaga, Hiromi3
  • Nishiba, Yosuke3
  • Yokoya, Susumu4
  • 1 Department of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo, Japan
  • 2 Tanaka Growth Clinic, Tokyo, Japan
  • 3 Novo Nordisk Pharma Ltd., Tokyo, Japan
  • 4 Thyroid and Endocrine Center, Fukushima Medical University, Fukushima, Japan
Type
Published Article
Journal
Clinical Pediatric Endocrinology
Publisher
The Japanese Society for Pediatric Endocrinology
Publication Date
Oct 03, 2020
Volume
29
Issue
4
Pages
159–171
Identifiers
DOI: 10.1297/cpe.29.159
PMID: 33088015
PMCID: PMC7534526
Source
PubMed Central
Keywords
License
Green

Abstract

This study aimed to characterize the safety and effectiveness of GH treatments, in usual clinical practice, in children with short stature born small for gestational age (SGA). This was a multicenter, open-label, non-interventional study ( NCT01110928 ) conducted at 150 sites in Japan (2009–2018). The primary objective was to assess the type and frequency of serious adverse drug reactions (SADRs) associated with long-term GH use. Overall, 452 naïve and 46 non-naïve (previously treated) children were enrolled. GH treatment was well‑tolerated, with SADRs occurring in 1.3% (6/452) and 0% (0/46) of naïve and non-naïve children, respectively. No new safety concerns or notable changes in glucose metabolism were identified during long-term treatment. Altogether, 57 children (32 naïve and 25 non-naïve) reached near adult height (NAH). In naïve and non-naïve children, mean ± standard deviation (SD) height standard deviation score (SDS) at NAH were –2.03 ± 0.77 and –1.53 ± 0.81, respectively, representing a change of +0.85 ± 0.72 and +1.24 ± 0.66 from baseline height SDS, respectively. Mean treatment duration to NAH was 4.29 (naïve) and 7.26 (non-naïve) yr. Thus, long-term GH treatment for short stature in children born SGA was confirmed to have a good safety profile and was effective for improving adult height.

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