Efficacy and Safety Evaluation of Human Growth Hormone Therapy in Patients with Idiopathic Short Stature in Korea – A Randomised Controlled Trial

Jung, Min Ho; Suh, Byung‐Kyu; Ko, Cheol Woo; Lee, Kee-Hyoung; Jin, Dong‐Kyu; Yoo, Han‐Wook; Hwang, Jin Soon; Chung, Woo Yeong; Han, Heon-Seok; Prusty, Vinay; Kim, Ho-Seong

doi:10.17925/ee.2020.16.1.54

Cited by 5 publications

(8 citation statements)

References 19 publications

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“…Our study was also in agreement with previous studies in ISS in terms of HT-SDS gain and HV trend (9,10,14,15,16,17). A recent randomized, phase III study in Korea also confirmed the benefit of 1-year GH treatment in prepubertal children with a mean age of 6.2 years, which reported a HV of 10.6 cm/year, height, and ΔHT-SDS at 6 months of 0.57 (95% CI, 0.43-0.71) compared with control (14).…”

Section: Discussionsupporting

confidence: 93%

“…This is the first randomized, controlled study investigating short-term height outcomes of long-acting PEG-rhGH in prepubertal Chinese children with ISS. Several studies have investigated the efficacy and safety of rhGH in children with ISS; however, only a few were randomized and controlled with an untreated control group (10,14,15,16). Although, the optimal age to initiate GH treatment is 5 years to early puberty (7), many other studies recruited children who were slightly older than ours (14,15,16,17).…”

Section: Discussionmentioning

confidence: 99%

“…Several studies have investigated the efficacy and safety of rhGH in children with ISS; however, only a few were randomized and controlled with an untreated control group (10,14,15,16). Although, the optimal age to initiate GH treatment is 5 years to early puberty (7), many other studies recruited children who were slightly older than ours (14,15,16,17). Regardless, our study showed that 52 weeks of PEG-rhGH treatment at either 0.1 or 0.2 mg/kg/week improved HT-SDS to above -2 SDS and showed more significant improvement in HV as early as week 4 than in the control group, suggesting that catch-up growth was achieved with both doses.…”

Section: Discussionmentioning

confidence: 99%

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Long-acting PEGylated growth hormone in children with idiopathic short stature

Luo

Zhao

Yang

et al. 2022

European Journal of Endocrinology

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Objective: To evaluate the safety and efficacy of weekly PEG-rhGH in children with idiopathic short stature (ISS) in China. Design and methods: A multicenter, phase II study randomized subjects 1:1:1 to weekly subcutaneous injections of PEG-rhGH 0.1 (low dose [LD]) or 0.2 mg/kg/week (high dose [HD]) or control for 52 weeks. The primary endpoint was change in HT-SDS from baseline to week 52. Secondary endpoints were height velocity (HV), bone maturity, IGF-1 SDS, and IGF-1/IGFBP-3 ratio. Results: A total of 360 children with ISS were recruited in the study (n=120 in each group). At week 52, the mean change from baseline in HT-SDS was 0.56 ± 0.26, 0.98 ± 0.35, and 0.20 ± 0.26 in the LD, HD, and control groups, respectively (within-group P<0.0001; inter-group P<0.0001). Statistically significant changes in HV, IGF-1, IGF-1/IGFBP-3 ratio, and IGF-1 SDS at week 52 from baseline were observed in both treatment groups (P<0.0001). There were clear dose-dependent responses for all auxological variables. PEG-rhGH was well tolerated throughout the treatment period with treatment-emergent adverse events (TEAEs) reported in 86.5%, 84.6%, and 91.3% of children in the HD, LD, and control groups, respectively. The incidence of TEAEs was similar in all treatment groups despite the difference in doses. A total of 27 (8.7%) children experienced drug-related TEAEs. Conclusion: Fifty-two-week treatment with PEG-rhGH 0.1 or 0.2 mg/kg/week achieved significant improvement in HT-SDS and other growth-related variables, including HV, IGF-1 SDS, and IGF-1/IGFBP-3 ratio, in a dose-dependent manner. Both doses were well tolerated with similar safety profiles.

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Section: Discussionsupporting

confidence: 93%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Long-acting PEGylated growth hormone in children with idiopathic short stature

Luo

Zhao

Yang

et al. 2022

European Journal of Endocrinology

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“…The dosage regimens instituted were extremely similar, so that the authors used doses that varied according to the FDA 2003 [5,8,9,[12][13][14][15][16][17][18] or the Chinese society of pediatric endocrinology [4,6,7,16,19] which recommend doses of 0.30-0.37mg/kg/ week and 0.35-0.46mg/kg/week, respectively. The therapeutic administration in the studies was subcutaneous once a day for 6 to 7 days bedtime.…”

Section: Treatment: Dosage Formulations and Combinationsmentioning

confidence: 99%

Safety of Growth Hormone Therapy in Patients with Idiopathic Short Stature: A Systematic Review

Ribeiro¹

2021

OAJBS

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Introduction: Idiopathic short stature is a common condition in the pediatric population requiring growth hormone therapy. Thus, based on patient safety concepts, we sought to group the main literature data related to this aspect in different countries. Objectives:The aim of this systematic review was to identify what the literature says about the main safety aspects of growth hormone therapy in children with idiopathic short stature.Methods: This is a systematic review using PubMed, BVS portal, Cochrane, LILACS, and CAPES databases, with the descriptors idiopathic short stature, human growth hormone therapy, patient safety, children, and the boolean "AND" between them. Inclusion criteria were children with idiopathic short stature treated at some point during their lifetime with growth hormone and studies dating between 2015 and 2020. Results:The safety of growth hormone therapy in this patient profile remained consistent with data previously described in the literature. There was a difference in the incidence of adverse effects, indicating that there are various degrees of resistance to IGF-1 action in these individuals, and the response to GH may vary in this heterogeneous population. These children need higher doses of GH to get the same benefits compared to other etiologies of short stature. Conclusion:Adverse effects were mostly mild, with rare severe cases, thus, it can be concluded that the use of GH in children with idiopathic short stature has an efficient safety profile, although further long-term studies are required.

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“…8 Patients were followed up for 12 months to obtain information on height and weight changes, insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3) levels before treatment and after 12 months of treatment [(by analyzing 3 ml fasting venous blood samples collected on the day of admission and day of course completion (UniCel DxI800)], adverse events such as lower limb edema, elevated blood glucose, and hypothyroidism, and clinical efficacy-defined as the amount of total height growth. 9,10 An annual growth rate >10cm was considered significant, a rate between 6-10 cm was considered effective, and anything less was considered invalid. The total treatment efficacy was calculated as the combined number of significant and effective clinical efficacies, divided by the total number of patients.…”

Section: Introductionmentioning

confidence: 99%

Effects of high-dose recombinant human growth hormone treatment on IGF-1 and IGFBP-3 levels in idiopathic dwarfism patients

Wu¹,

Lin²,

Gao³

et al. 2022

Pak J Med Sci

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Objective: To investigate the effects of high-dose recombinant human growth hormone(rhGH) treatment on insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3) levels in patients with idiopathic dwarfism. Methods: This study retrospectively investigated records of idiopathic dwarfism patients treated at Anhui Children’s Hospital or the Affiliated Hospital of Xuzhou Medical University between May 2019 and October 2020. The study identified 76 patients, which were divided based on rhGH treatment dosage into high- and low-dose groups. Results: The high-dose group showed a total efficacy of 95.3%, which was significantly higher than the 79.41% observed in the low-dose group (P<0.05). Moreover, height, weight, IGF-1 levels, and IGFBP-3 levels were all significantly elevated in the high-dose group compared to the low-dose group (P<0.05). No significant difference in adverse reaction incidence was observed between high- (9.5%) and low-dose (8.8%) groups (P>0.05). Conclusions: Higher doses of rhGH can provide improved curative effects in patients with idiopathic short stature, likely via elevated levels of IGF-1 and IGFBP-3. doi: https://doi.org/10.12669/pjms.38.4.5502 How to cite this:Wu B, Lin H, Gao J, Sun J, Zhao M. Effects of high-dose recombinant human growth hormone treatment on IGF-1 and IGFBP-3 levels in idiopathic dwarfism patients. Pak J Med Sci. 2022;38(4):---------. doi: https://doi.org/10.12669/pjms.38.4.5502 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Efficacy and Safety Evaluation of Human Growth Hormone Therapy in Patients with Idiopathic Short Stature in Korea – A Randomised Controlled Trial

Cited by 5 publications

References 19 publications

Long-acting PEGylated growth hormone in children with idiopathic short stature

Long-acting PEGylated growth hormone in children with idiopathic short stature

Safety of Growth Hormone Therapy in Patients with Idiopathic Short Stature: A Systematic Review

Effects of high-dose recombinant human growth hormone treatment on IGF-1 and IGFBP-3 levels in idiopathic dwarfism patients

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