Considerations Related to the Use of Recombinant Human Growth Hormone in Children

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doi:10.1542/peds.99.1.122

Cited by 47 publications

(5 citation statements)

References 53 publications

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“…Short stature [1] and growth failure [2, 3] are some of the most common causes for referral to paediatric endocrinology clinics, general paediatricians, and primary health care physicians [3]. …”

Section: Introductionmentioning

confidence: 99%

Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial

et al. 2018

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Background/Aims: The SYNERGY (Saizen® for Your New Life and Brighter Tomorrow without Growth Deficiency) study is the first randomised multi-centre, open-label study to assess the short-term efficacy and safety of this recombinant human growth hormone (r-hGH) preparation for prepubertal children with idiopathic short stature in South Korea. Methods: The SYNERGY study (ClinicalTrials.gov NCT01746862) was conducted at 9 centres throughout South Korea between December 2012 and March 2015. The primary endpoint was difference in height velocity from baseline to 6 months in the treatment and control arms. Results: 97 children were screened; 90 were randomly assigned: 60 children to 0.067 mg/kg/day r-hGH for 12 months (treatment) and 30 children to 6 months of no treatment followed by 0.067 mg/kg/day r-hGH for 6 months (control). The 6-month mean height velocity in the treatment group increased from 5.63 cm/year (SD 1.62) to 10.08 cm/year (SD 1.92) (p < 0.0001) and from 4.94 cm/year (SD 1.91) to 5.92 cm/year (SD 2.01) (p = 0.0938) in the control group (between-group difference 3.47 cm/year, 95% CI 2.17–4.78; p < 0.0001). Adherence was > 90% throughout the study. The safety profile was consistent with that already known for r-hGH. Conclusion: Treatment with r-hGH in the SYNERGY study demonstrated a statistically significant increase in height velocity at 6 months.

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Section: Introductionmentioning

confidence: 99%

Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial

et al. 2018

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“…Dosing of biologics is generally limited to parenteral routes, which are associated with fear and discomfort, particularly in the pediatric population, thereby potentially affecting adherence ( 1 ). Biologics have transformed our capacity to effectively treat a range of conditions including diabetes mellitus, growth hormone insufficiency, and enzyme deficiencies, which require protein- and nucleic acid–based therapies ( 2 – 4 ). However, biologics are generally degraded in the gastrointestinal tract or poorly absorbed ( 5 , 6 ).…”

Section: Introductionmentioning

confidence: 99%

A microneedle platform for buccal macromolecule delivery

et al. 2021

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Alternative means for drug delivery are needed to facilitate drug adherence and administration. Microneedles (MNs) have been previously investigated transdermally for drug delivery. To date, drug loading into MNs has been limited by drug solubility in the polymeric blend. We designed a highly drug-loaded MN patch to deliver macromolecules and applied it to the buccal area, which allows for faster delivery than the skin. We successfully delivered 1-mg payloads of human insulin and human growth hormone to the buccal cavity of swine within 30 s. In addition, we conducted a trial in 100 healthy volunteers to assess potential discomfort associated with MNs when applied in the oral cavity, identifying the hard palate as the preferred application site. We envisage that MN patches applied on buccal surfaces could increase medication adherence and facilitate the painless delivery of biologics and other drugs to many, especially for the pediatric and elderly populations.

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“…The American Academy of Pediatrics recommends GH treatment for very short children who have limited ability to participate in basic activities of daily living due to their short stature (5). In 2003, the U.S. Food and Drug Administration (FDA) approved the use of GH for children with ISS with a height at or less than -2.25 s.d.…”

Section: Introductionmentioning

confidence: 99%

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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Considerations Related to the Use of Recombinant Human Growth Hormone in Children

Cited by 47 publications

References 53 publications

Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial

Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial

A microneedle platform for buccal macromolecule delivery

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

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