scholarly journals Recombinant Growth Hormone Therapy in Children With Turner’s Syndrome in Korea: A Phase III Randomized Trial

2021 ◽  
Author(s):  
Jinsup Kim ◽  
Min-Sun Kim ◽  
Byung-Kyu Suh ◽  
Cheol Woo Ko ◽  
Kee-Hyoung Lee ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Serum Insulin ◽  
Standard Deviation Score ◽  
Phase Iii ◽  
Height Velocity ◽  
Insulin Like Growth Factor ◽  
Efficacy And Safety ◽  
Korean Children ◽  
Comparator Group

Abstract Background Short stature is the most consistent characteristic feature of Turner syndrome (TS). To improve final heights of children with TS effectively, it is important to provide them with early and appropriate treatment using growth hormone (GH). The objective of this study was to assess the efficacy and safety of a new recombinant human GH, Growtropin®-II (DA-3002, Dong-A ST Co., Ltd) in comparison with a comparator (Genotropin®, Pfizer Inc.) for Korean children with TS. Methods This open-label, active-controlled, parallel-group, randomized controlled phase III trial was conducted at 11 hospitals in Korea. Eligible patients (n = 58) were randomized to two groups: 1) DA-3002 group (administrated with DA-3002 at 0.14 IU /kg/day); and 2) comparator group (administrated with the comparator at 0.14 IU /kg/day). Results The change from baseline in annualized height velocity (HV) after a 52-week treatment was 4.15 ± 0.30 cm/year in the DA-3002 group and 4.34 ± 0.29 cm/year in the comparator group. The lower bound of 95% two-sided confidence interval for group difference in the change of annualized HV (-1.02) satisfied the non-inferiority margin (-1.5). The change in height standard deviation score (HtSDS) at 52-week was 0.70 ± 0.23 for the DA-3002 group and 0.66 ± 0.39 for the comparator group, showing no significant (p = 0.685) difference between the two groups. Skeletal maturity defined as a change in bone age or a change in chronological age between the two groups was not significantly different (1.25 ± 0.58 for the DA-3002 group and 1.47 ± 0.45 for the comparator group, p = 0.134). Changes of serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3) after 52 weeks of treatment did not differ significantly between the two groups (p = 0.565 and p = 0.388, respectively) either. Adverse events were not significant in either group. Conclusions This study demonstrates that the efficacy and safety of GH treatment with DA-3002 in children with TS are comparable with those of the comparator. Thus, treatment with DA-3002 can be used to improve their growth. DA-3002 is also well tolerated in children with TS. Trial registration: The study was registered at ClinicalTrials.gov. ClinicalTrials.gov identifier: NCT01813630 (19/03/2013)

scholarly journals Recombinant growth hormone therapy in children with Turner Syndrome in Korea: a phase III Randomized Trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Jinsup Kim ◽  
Min-Sun Kim ◽  
Byung-Kyu Suh ◽  
Cheol Woo Ko ◽  
Kee-Hyoung Lee ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Turner Syndrome ◽  
Standard Deviation Score ◽  
Phase Iii ◽  
Height Velocity ◽  
Insulin Like Growth Factor ◽  
Efficacy And Safety ◽  
Korean Children ◽  
Comparator Group

Abstract Background Short stature is the most consistent characteristic feature of Turner syndrome (TS). To improve final heights of children with TS effectively, it is important to provide them with early and appropriate treatment using growth hormone (GH). The objective of this study was to assess the efficacy and safety of a new recombinant human GH, Growtropin®-II (DA-3002, Dong-A ST Co., Ltd) versus a comparator (Genotropin®, Pfizer Inc.) for Korean children with TS. Methods This open-label, active-controlled, parallel-group, randomized controlled phase III trial was conducted at 11 hospitals in Korea. Eligible patients (n = 58) were randomized to two groups: 1) DA-3002 group (administrated with DA-3002 at 0.14 IU [0.0450–0.050 mg] /kg/day); and 2) comparator group (administrated with the comparator at 0.14 IU [0.0450–0.050 mg] /kg/day). Results The change from baseline in annualized height velocity (HV) after a 52-week treatment period was 4.15 ± 0.30 cm/year in the DA-3002 group and 4.34 ± 0.29 cm/year in the comparator group. The lower bound of 95% two-sided confidence interval for group difference in the change of annualized HV (− 1.02) satisfied the non-inferiority margin (− 1.5). The change in height standard deviation score (HtSDS) at 52-week was 0.70 ± 0.23 for the DA-3002 group and 0.66 ± 0.39 for the comparator group, showing no significant (p = 0.685) difference between the two groups. The change of skeletal maturity defined as change in bone age/change in chronological age between the two groups was not significantly different (1.25 ± 0.58 for the DA-3002 group and 1.47 ± 0.45 for the comparator group, p = 0.134). Changes from baseline in serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3) after 52 weeks of treatment did not differ significantly between the two groups (p = 0.565 and p = 0.388, respectively) either. The occurrence of adverse events was not statistically different between groups. Conclusions This study demonstrates that the efficacy and safety of GH treatment with DA-3002 in children with TS are comparable with those of the comparator. It is expected to analysis the long-term effect of DA-3002 on the increase of final adult height in children with TS and possible late-onset complications in the future. Trial registration The study was registered at ClinicalTrials.gov. ClinicalTrials.gov identifier: NCT01813630 (19/03/2013).

scholarly journals Insulin-like growth factor-1 level is a poor diagnostic indicator of growth hormone deficiency

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Hideyuki Iwayama ◽  
Sachiko Kitagawa ◽  
Jyun Sada ◽  
Ryosuke Miyamoto ◽  
Tomohito Hayakawa ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Diagnostic Accuracy ◽  
Growth Hormone Deficiency ◽  
Screening Test ◽  
Characteristic Curve ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Insulin Like Growth Factor ◽  
Predictive Values

AbstractWe evaluated the diagnostic accuracy of insulin-like growth factor-1 (IGF-1) for screening growth hormone deficiency (GHD) to determine the usefulness of IGF-1 as a screening test. Among 298 consecutive children who had short stature or decreased height velocity, we measured IGF-1 levels and performed growth hormone (GH) secretion test using clonidine, arginine, and, in cases with different results of the two tests, L-dopa. Patients with congenital abnormalities were excluded. GHD was defined as peak GH ≤ 6.0 ng/mL in the two tests. We identified 60 and 238 patients with and without GHD, respectively. The mean IGF-1 standard deviation (SD) was not significantly different between the GHD and non-GHD groups (p = 0.23). Receiver operating characteristic curve analysis demonstrated the best diagnostic accuracy at an IGF-1 cutoff of − 1.493 SD, with 0.685 sensitivity, 0.417 specificity, 0.25 positive and 0.823 negative predictive values, and 0.517 area under the curve. Correlation analysis revealed that none of the items of patients’ characteristics increased the diagnostic power of IGF-1. IGF-1 level had poor diagnostic accuracy as a screening test for GHD. Therefore, IGF-1 should not be used alone for GHD screening. A predictive biomarker for GHD should be developed in the future.

Peak and trough growth hormone (GH) concentrations influence growth and serum insulin like growth factor-1 (IGF-1) concentrations in short children

1999 ◽  
Vol 50 (3) ◽  
pp. 301-308 ◽  
Author(s):  
J. C. Achermann ◽  
C. G. D. Brook ◽  
I. C. A. F. Robinson ◽  
D. R. Matthews ◽  
P. C. Hindmarsh
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Serum Insulin ◽  
Insulin Like Growth Factor ◽  
Short Children

Determinants of serum insulin-like growth factor I in growth hormone deficient adults as compared to healthy subjects

1998 ◽  
Vol 48 (4) ◽  
pp. 479-486 ◽  
Author(s):  
Jens O. L. Jørgensen ◽  
Nina Vahl ◽  
Tony B. Hansen ◽  
Christian Skjaerbaek ◽  
Sanne Fisker ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Healthy Subjects ◽  
Serum Insulin ◽  
Insulin Like Growth Factor ◽  
Factor I ◽  
Growth Factor I

Serum Insulin-Like Growth Factor 1 and Growth Hormone Levels of Hypoxic-Ischemic Newborns

Neonatology ◽  
2004 ◽  
Vol 85 (1) ◽  
pp. 15-20 ◽  
Author(s):  
Mehmet Satar ◽  
Kenan Özcan ◽  
Hacer Yapıcıoğlu ◽  
Nejat Narlı
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Serum Insulin ◽  
Insulin Like Growth Factor ◽  
Hormone Levels

Long-term treatment of Laron type dwarfs with insulin-like growth factor-1 increases serum insulin-like growth factor-binding protein-3 in the absence of growth hormone activity

1993 ◽  
Vol 128 (2) ◽  
pp. 144-149 ◽  
Author(s):  
Hannah Kanety ◽  
Avraham Karasik ◽  
Beatrice Klinger ◽  
Aviva Silbergeld ◽  
Zvi Laron
Keyword(s):  
Growth Hormone ◽  
Growth Factor ◽  
Binding Protein ◽  
Serum Insulin ◽  
Continuous Treatment ◽  
Insulin Like Growth Factor ◽  
Factor Binding ◽  
Long Term Treatment ◽  
Igf I ◽  
Igfbp 3

Insulin-like growth factor binding protein-3 (IGFBP-3) is the major carrier of insulin-like growth factor I (IGF-1) in serum, and its production is growth hormone (GH) dependent. It is unclear whether in humans IGFBP-3 production is directly regulated by GH or mediated via IGF-I. We addressed this question in six patients with Laron-type dwarfism, a syndrome characterized by the absence of GH receptor activity (LTD), who were chronically treated with recombinant IGF-I. Analysis of the electrophoretic profiles of serum IGFBPs in these patients by Western ligand blotting revealed an extremely low IGFBP-3 level. A striking progressive increase in serum IGFBP-3 was observed with continuous treatment, despite the absence of GH action. In LTD children, serum IGFBP-3 increased up to 19-fold after six months of therapy and equalled levels observed in controls, whereas in adult LTD patients the increase was smaller. A rise in serum levels of 34, 30 and 24 kDa BPs (presumably IGFBP-2, -1 and -4, respectively was also noted with chronic IGF-I therapy. This proof of GH-independent induction of IGFBP-3 by IGF-1 may be a major advantage in the therapeutic use of biosynthetic IGF-I in several types of short stature children.

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