Efficacy and Safety Evaluation of Human Growth Hormone Therapy in Patients with Idiopathic Short Stature in Korea - A Randomised Controlled Trial.
Human growth hormone
clinical trial
idiopathic
short stature
Journal
European endocrinology
ISSN: 1758-3780
Titre abrégé: Eur Endocrinol
Pays: England
ID NLM: 101574781
Informations de publication
Date de publication:
Apr 2020
Apr 2020
Historique:
received:
21
03
2019
accepted:
07
07
2019
entrez:
30
6
2020
pubmed:
1
7
2020
medline:
1
7
2020
Statut:
ppublish
Résumé
This trial evaluated the efficacy and safety of growth hormone (GH) therapy (Norditropin®; Novo Nordisk, Bagsværd, Denmark) in paediatric patients with idiopathic short stature (ISS) in Korea. This was an open-label, parallel-group, multicentre, interventional trial (ClinicalTrials.gov identifier: NCT01778023). Pre-pubertal patients (mean age 6.2 years; height, 107.1 cm) were randomised 2:1 to 12 months' GH treatment (0.469 mg/kg/week; group A, n=36) or 6 months untreated followed by 6 months' GH treatment (group B, n=18). Safety analysis was based on adverse events (AEs) in all GH-treated patients. After 6 months, height velocity (Ht-V), change in both height standard deviation score (Ht-SDS) and insulin-like growth factor 1 (mean difference [95% confidence interval {CI}]: 5.15 cm/year [4.09, 6.21]; 0.57 [0.43, 0.71]; 164.56 ng/mL [112.04, 217.08], respectively; all p<0.0001) were greater in group A than in group B. Mean difference in Ht-V for 0-6 months versus 6-12 months was 2.80 cm/year (95% CI 1.55, 4.04) for group A and -4.60 cm/year (95% CI -6.12, -3.09; both p<0.0001) for group B. No unexpected AEs were reported. During the first 6 months, height was significantly increased in GH-treated patients versus untreated patients with ISS. Safety of GH was consistent with the known safety profile.
Sections du résumé
BACKGROUND
BACKGROUND
This trial evaluated the efficacy and safety of growth hormone (GH) therapy (Norditropin®; Novo Nordisk, Bagsværd, Denmark) in paediatric patients with idiopathic short stature (ISS) in Korea.
METHODS
METHODS
This was an open-label, parallel-group, multicentre, interventional trial (ClinicalTrials.gov identifier: NCT01778023). Pre-pubertal patients (mean age 6.2 years; height, 107.1 cm) were randomised 2:1 to 12 months' GH treatment (0.469 mg/kg/week; group A, n=36) or 6 months untreated followed by 6 months' GH treatment (group B, n=18). Safety analysis was based on adverse events (AEs) in all GH-treated patients.
RESULTS
RESULTS
After 6 months, height velocity (Ht-V), change in both height standard deviation score (Ht-SDS) and insulin-like growth factor 1 (mean difference [95% confidence interval {CI}]: 5.15 cm/year [4.09, 6.21]; 0.57 [0.43, 0.71]; 164.56 ng/mL [112.04, 217.08], respectively; all p<0.0001) were greater in group A than in group B. Mean difference in Ht-V for 0-6 months versus 6-12 months was 2.80 cm/year (95% CI 1.55, 4.04) for group A and -4.60 cm/year (95% CI -6.12, -3.09; both p<0.0001) for group B. No unexpected AEs were reported.
CONCLUSIONS
CONCLUSIONS
During the first 6 months, height was significantly increased in GH-treated patients versus untreated patients with ISS. Safety of GH was consistent with the known safety profile.
Identifiants
pubmed: 32595770
doi: 10.17925/EE.2020.16.1.54
pmc: PMC7308103
doi:
Banques de données
ClinicalTrials.gov
['NCT01778023']
Types de publication
Journal Article
Langues
eng
Pagination
54-59Informations de copyright
© Touch Medical Media 2020.
Déclaration de conflit d'intérêts
Disclosures: Heon-Seok Han received clinical research funding from Novo Nordisk. Vinay Prusty was an employee of Novo Nordisk Pharma Korea Ltd at the time of the study. Min Ho Jung, Byung-Kyu Suh, Cheol Woo Ko, Kee-Hyoung Lee, Dong-Kyu Jin, Han-Wook Yoo, Jin Soon Hwang, Woo Yeong Chung and Ho-Seong Kim have no financial or non-financial relationships or activities to declare in relation to this article.
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