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Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial

OBJECTIVE: Growth hormone (GH) treatment is known to be effective in increasing stature in children with a short stature born small for gestational age (SGA). This multicentre, randomized, open-label, comparative, phase III study aimed to evaluate the efficacy and safety of Growtropin-II (recombinan...

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Autores principales: Kim, Su Jin, Kim, Min-Sun, Cho, Sung Yoon, Suh, Byung-Kyu, Ko, Cheol Woo, Lee, Kee-Hyoung, Yoo, Han-Wook, Shin, Choong Ho, Hwang, Jin Soon, Kim, Ho-Seong, Chung, Woo Yeong, Kim, Chan Jong, Han, Heon-Seok, Jin, Dong-Kyu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8322493/
https://www.ncbi.nlm.nih.gov/pubmed/34397702
http://dx.doi.org/10.1097/MD.0000000000026711
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author Kim, Su Jin
Kim, Min-Sun
Cho, Sung Yoon
Suh, Byung-Kyu
Ko, Cheol Woo
Lee, Kee-Hyoung
Yoo, Han-Wook
Shin, Choong Ho
Hwang, Jin Soon
Kim, Ho-Seong
Chung, Woo Yeong
Kim, Chan Jong
Han, Heon-Seok
Jin, Dong-Kyu
author_facet Kim, Su Jin
Kim, Min-Sun
Cho, Sung Yoon
Suh, Byung-Kyu
Ko, Cheol Woo
Lee, Kee-Hyoung
Yoo, Han-Wook
Shin, Choong Ho
Hwang, Jin Soon
Kim, Ho-Seong
Chung, Woo Yeong
Kim, Chan Jong
Han, Heon-Seok
Jin, Dong-Kyu
author_sort Kim, Su Jin
collection PubMed
description OBJECTIVE: Growth hormone (GH) treatment is known to be effective in increasing stature in children with a short stature born small for gestational age (SGA). This multicentre, randomized, open-label, comparative, phase III study aimed to evaluate the efficacy and safety of Growtropin-II (recombinant human GH) and to demonstrate that the growth-promoting effect of Growtropin-II is not inferior to that of Genotropin in children with SGA (NCT ID: NCT02770157). METHODS: Seventy five children who met the inclusion criteria were randomized into 3 groups in a ratio of 2:2:1 (the study group [Growtropin-II, n = 30], control group [Genotropin, n = 30], and 26-week non-treatment group [n = 15]). The study and control groups received subcutaneous injections of Growtropin-II and Genotropin, respectively for 52 weeks, whereas the non-treatment group underwent a non-treatment observation period during weeks 0 to 26 and a dosing period during weeks 27 to 52 and additional dosing till week 78 only in re-consenting children. RESULTS: No significant differences in demographic and baseline characteristics between the groups were observed. The mean ± standard deviation change difference in annualized height velocity (aHV) (study group - control group) was 0.65 ±2.12 cm/year (95% confidence interval [CI], −0.53 to 1.83), whereas the lower limit for the 2-sided 95% CI was −0.53 cm/year. Regarding safety, treatment-emergent adverse events (TEAEs) occurred in 53.33% children in the study group and 43.33% children in the control group; the difference in the incidence of TEAEs between the 2 treatment groups was not statistically significant (P = .4383). A total of 17 serious adverse events (SAEs) occurred in 13.33% children in the treatment groups, and no significant difference in incidence between groups (P = .7065) was seen. Two cases of adverse drug reaction (ADR) occurred in 2 children (3.33%): 1 ADR (injection site swelling or pain) occurred in 1 child (3.33%) each in the study and control groups. CONCLUSIONS: This study demonstrates that the change in aHV from the baseline till 52 weeks with Growtropin-II treatment is non-inferior to that with Genotropin treatment in children with short stature born SGA. Growtropin-II is well-tolerated, and its safety profile is comparable with that of Genotropin over a 1-year course of treatment.
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spelling pubmed-83224932021-08-02 Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial Kim, Su Jin Kim, Min-Sun Cho, Sung Yoon Suh, Byung-Kyu Ko, Cheol Woo Lee, Kee-Hyoung Yoo, Han-Wook Shin, Choong Ho Hwang, Jin Soon Kim, Ho-Seong Chung, Woo Yeong Kim, Chan Jong Han, Heon-Seok Jin, Dong-Kyu Medicine (Baltimore) 4300 OBJECTIVE: Growth hormone (GH) treatment is known to be effective in increasing stature in children with a short stature born small for gestational age (SGA). This multicentre, randomized, open-label, comparative, phase III study aimed to evaluate the efficacy and safety of Growtropin-II (recombinant human GH) and to demonstrate that the growth-promoting effect of Growtropin-II is not inferior to that of Genotropin in children with SGA (NCT ID: NCT02770157). METHODS: Seventy five children who met the inclusion criteria were randomized into 3 groups in a ratio of 2:2:1 (the study group [Growtropin-II, n = 30], control group [Genotropin, n = 30], and 26-week non-treatment group [n = 15]). The study and control groups received subcutaneous injections of Growtropin-II and Genotropin, respectively for 52 weeks, whereas the non-treatment group underwent a non-treatment observation period during weeks 0 to 26 and a dosing period during weeks 27 to 52 and additional dosing till week 78 only in re-consenting children. RESULTS: No significant differences in demographic and baseline characteristics between the groups were observed. The mean ± standard deviation change difference in annualized height velocity (aHV) (study group - control group) was 0.65 ±2.12 cm/year (95% confidence interval [CI], −0.53 to 1.83), whereas the lower limit for the 2-sided 95% CI was −0.53 cm/year. Regarding safety, treatment-emergent adverse events (TEAEs) occurred in 53.33% children in the study group and 43.33% children in the control group; the difference in the incidence of TEAEs between the 2 treatment groups was not statistically significant (P = .4383). A total of 17 serious adverse events (SAEs) occurred in 13.33% children in the treatment groups, and no significant difference in incidence between groups (P = .7065) was seen. Two cases of adverse drug reaction (ADR) occurred in 2 children (3.33%): 1 ADR (injection site swelling or pain) occurred in 1 child (3.33%) each in the study and control groups. CONCLUSIONS: This study demonstrates that the change in aHV from the baseline till 52 weeks with Growtropin-II treatment is non-inferior to that with Genotropin treatment in children with short stature born SGA. Growtropin-II is well-tolerated, and its safety profile is comparable with that of Genotropin over a 1-year course of treatment. Lippincott Williams & Wilkins 2021-07-30 /pmc/articles/PMC8322493/ /pubmed/34397702 http://dx.doi.org/10.1097/MD.0000000000026711 Text en Copyright © 2021 the Author(s). Published by Wolters Kluwer Health, Inc. https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the Creative Commons Attribution License 4.0 (CCBY), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. http://creativecommons.org/licenses/by/4.0 (https://creativecommons.org/licenses/by/4.0/)
spellingShingle 4300
Kim, Su Jin
Kim, Min-Sun
Cho, Sung Yoon
Suh, Byung-Kyu
Ko, Cheol Woo
Lee, Kee-Hyoung
Yoo, Han-Wook
Shin, Choong Ho
Hwang, Jin Soon
Kim, Ho-Seong
Chung, Woo Yeong
Kim, Chan Jong
Han, Heon-Seok
Jin, Dong-Kyu
Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial
title Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial
title_full Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial
title_fullStr Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial
title_full_unstemmed Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial
title_short Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: A randomized, multicentre, comparative phase III trial
title_sort efficacy and safety of the recombinant human growth hormone in short children born small for gestational age: a randomized, multicentre, comparative phase iii trial
topic 4300
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8322493/
https://www.ncbi.nlm.nih.gov/pubmed/34397702
http://dx.doi.org/10.1097/MD.0000000000026711
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