A pilot study of growth hormone administration in boys with predicted adult short stature and near-ending growth

Rothenbühler, Anya; Ormières, Baptiste; Kalifa, G.; Bougnères, Pierre

doi:10.1016/j.ghir.2015.01.002

Cited by 8 publications

(2 citation statements)

References 34 publications

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“…In a variety of prospective/retrospective, randomized/non-randomized, and controlled/ non-controlled trials, some researchers concluded that GH treatment improved ISS while others considered it ineffective. The efficacies in these studies varied dramatically [12][13][14][15][16][17][18]. Some meta-analyses showed that GHtreated ISS patients might gain more FAH than untreated children, but were still shorter than the normal populations.…”

Section: Discussionmentioning

confidence: 99%

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Chen

et al. 2020

BMC Pediatr

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Background: To evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study. Methods: A real-world observation was carried out. Children with ISS in seven hospitals in China were enrolled. The height gains standard deviation score and the height gain over the target height were evaluated. Results: There were 344 ISS patients (217 boys and 127 girls). The baseline average age of boys and girls was 12.7 and 11.7 years, with bone age of 11.7 and 10.1 years, respectively. The baseline height SDS of boys and girls was − 3.07 and − 2.74, and the FAH SDS was − 1.91 and − 1.38, respectively. Compared with the baseline height SDS, the FAH SDS was significantly increased in both boys and girls (both P = 0.0000). The FAH SDS was the highest (gain by 1.54 SD) in the ≥2y treatment course group. Two hundred eighteen patients (218/344, 63.4%) had a FAH SDS > − 2 SD. Among these patients, girls in the 1-2y treatment course group and ≥ 2y group had a FAH SDS higher than TH SDS. Even in the control group, a spontaneous catch-up growth of 1.16 SD was observed. A multivariate linear regression model was used to analyze the results, with FAH SDS as the dependent variable. It was found that the treatment course and baseline height SDS in the boys' model were statistically significant (P < 0.05), whereas the baseline height SDS and baseline bone age significantly affected the girls' FAH SDS (P < 0.05). Conclusions: Both girls and boys of ISS improved FAH by GH therapy even if treatments begin over 10 years old and majority of them reached TH. Some peri-puberty ISS will have a spontaneous height gain. We recommend the course of GH treatment more than 2 years for girls, and longer courses for boys.

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Section: Discussionmentioning

confidence: 99%

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Chen

et al. 2020

BMC Pediatr

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“…Protein therapeutics possess advantages over small-molecule approaches including high target specificity and low off-target effects with normal biological processes. − As a protein drug, human growth hormone (hGH) has a unique role in promoting longitudinal bone growth and is widely used for the clinical treatment of short stature in children caused by growth hormone deficiency or growth failure. − However, the current rhGH standard of care requires frequent subcutaneous injection (i.e., three times a week or daily) as a consequence of its short half-life, which leads to poor patient compliance, higher than desired dose, and increased cost. − Therefore, a sustained-release rhGH formulation would not only provide improved patient compliance but also alleviate the costly burden associated with frequent injections. The best known rhGH-sustained delivery system, Nutropin Depot, which is encapsulation of rhGH in biodegradable poly(lactic acid- co -glycolic acid) (PLGA) microspheres, was approved by the US FDA in 1999 as a monthly drug. − Later in 2004, this product was withdrawn from the market due to several issues, including low loading efficiency, high burst release, protein denaturation during microsphere preparation, and inflammation from acidic PLGA degradation byproducts. , So far, it has remained a challenge to develop an effective rhGH delivery system that lasts longer than a month.…”

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confidence: 99%

Sustained Release of Recombinant Human Growth Hormone from Bioresorbable Poly(ester urea) Nanofibers

Gao

Land

et al. 2017

ACS Macro Lett.

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Recombinant human growth hormone (rhGH) has been used clinically for several decades. However, rhGH has a short half-life in vivo and therefore for efficacy requires frequent subcutaneous injection, leading to pain and poor patient compliance. An effective rhGH delivery system able to continuously release rhGH over a comparatively long period (generally over a month) would be a significant clinical advance. In this study, a novel long-duration release strategy of rhGH was developed by encapsulating sugar glass-stabilized rhGH in electrospun, bioresorbable poly(ester urea) (PEU) nanofibers. The rhGH was found to be randomly dispersed among the fibers, and sustained rhGH release with only a modest burst was observed for at least 6 weeks as studied by a bicinchoninic acid (BCA) protein assay. Significantly, the released rhGH remained bioavailable and bioactive as determined by an Nb2 cell bioassay specific for rhGH. Our results suggest the feasibility of this system as an effective long-term sustained release strategy for rhGH and potentially other therapeutic proteins.

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Recent Trends in Microneedle Development & Applications in Medicine and Cosmetics (2013–2018)

Kochhar¹,

Tan

Kwang

et al. 2019

Microneedles for Transdermal Drug Delivery

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A pilot study of growth hormone administration in boys with predicted adult short stature and near-ending growth

Cited by 8 publications

References 34 publications

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Sustained Release of Recombinant Human Growth Hormone from Bioresorbable Poly(ester urea) Nanofibers

Recent Trends in Microneedle Development & Applications in Medicine and Cosmetics (2013–2018)

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