scholarly journals A new and accurate prediction model for growth response to growth hormone treatment in children with growth hormone deficiency

2001 ◽  
pp. 13-20 ◽  
Author(s):  
E Schonau ◽  
F Westermann ◽  
F Rauch ◽  
A Stabrey ◽  
G Wassmer ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Prediction Model ◽  
Growth Response ◽  
Prediction Models ◽  
Bone Age ◽  
Individual Growth ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Therapeutic Success ◽  
Rhgh Therapy

OBJECTIVE: To identify parameters which predict individual growth response to recombinant human GH (rhGH) therapy and to combine these parameters in a prediction model. DESIGN: Fifty-eight prepubertal patients with GH deficiency (17 females) participated in this prospective multicenter trial with 1 year of follow-up. METHODS: Auxological measurements, parameters of GH status and markers of bone metabolism were measured at baseline and at 1, 3 and 6 months after the start of rhGH treatment. Correlations with height velocity during the first 12 months of treatment (HV+12) were calculated. Prediction models were derived by multiple regression analysis. RESULTS: The model which best predicted HV+12 combined the following parameters: pretreatment bone age retardation as a fraction of chronological age, pretreatment serum levels of IGF-I, urinary levels of deoxypyridinoline (a marker of bone resorption) after 1 month of treatment and height velocity after 3 months of treatment. This model explained 89% of the variation in HV+12 with a standard deviation of the residuals of 0.93 cm/year. Defining successful rhGH therapy as a doubling of pretreatment height velocity, the model had a specificity of 90% and a sensitivity of 100% in predicting therapeutic success. CONCLUSIONS: This model is an accurate and practicable tool to predict growth response in GH-deficient children. It may help to optimize rhGH therapy by individual dose adjustment and contribute to improved overall outcomes.

scholarly journals Decreased Thyroxine Levels during rhGH Therapy in Children with Growth Hormone Deficiency

2021 ◽  
Vol 10 (21) ◽  
pp. 5100
Author(s):  
Ewelina Witkowska-Sędek ◽  
Anna Małgorzata Kucharska ◽  
Małgorzata Rumińska ◽  
Monika Paluchowska ◽  
Beata Pyrżak
Keyword(s):  
Growth Hormone ◽  
Thyroid Function ◽  
Growth Response ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Bone Age ◽  
Hormone Deficiency ◽  
Lower Growth ◽  
Rhgh Therapy

Background: Hypothyroidism in children leads to growth retardation. However, there is some evidence that recombinant human growth hormone (rhGH) therapy could suppress thyroid function. The most common observation in rhGH-treated patients is a decrease in thyroxine levels, which is reported as transient, but the studies in the field are inconsistent. We aimed to evaluate thyroid function in initially euthyroid children with idiopathic isolated GH deficiency during long-term rhGH therapy and to determine who is at a higher risk of thyroid function alterations during the therapy. Methods: The study group consisted of 101 children treated with rhGH for at least three years. Serum TSH and fT4 levels were determined at baseline, after the first six months and after each full year of therapy. The associations between changes in thyroid hormone levels during rhGH therapy and GH deficit, insulin-like growth factor-1 levels and growth response were investigated. Results: A significant decrease in fT4 levels (p = 0.01) was found as early as after the first six months of rhGH therapy. This effect persisted in the subsequent years of treatment without any significant changes in TSH values and tended to be rhGH dose related. Children with a greater fT4 decrease after the initiation of rhGH therapy were older, had higher bone age and responded to that therapy worse than children with lower fT4 changes. Conclusions: Our study revealed a long-term decrease in fT4 levels during rhGH therapy in initially euthyroid GHD children. The decrease in fT4 levels was associated with a lower growth response to rhGH therapy.

scholarly journals Use of PEGylated Recombinant Human Growth Hormone in Chinese Children with Growth Hormone Deficiency: A 24-Month Follow-Up Study

2019 ◽  
Vol 2019 ◽  
pp. 1-7
Author(s):  
Yu Qiao ◽  
Zengmin Wang ◽  
Jinyan Han ◽  
Guimei Li
Keyword(s):  
Growth Hormone ◽  
Human Growth Hormone ◽  
Pediatric Patients ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Bone Age ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Rhgh Therapy ◽  
Long Acting

Objective. Once-weekly PEGylated recombinant human growth hormone (rhGH) is the sole long-acting GH formulation available currently for pediatric patients with GH deficiency (GHD). The aim of this study was to evaluate the efficacy and safety of PEGylated rhGH therapy compared to daily rhGH therapy in GHD children treated for two years. Methods. A total of 98 children (49 children for the PEGylated rhGH group and 49 children for the daily rhGH group) with GHD were enrolled in this single-center, prospective, nonrandomized cohort study. PEGylated rhGH or daily rhGH was administered for 2 years. Height, height SDS, height velocity (HV), IGF-1, bone age (BA), and adverse events were determined throughout the treatment. Results. HV significantly increased over the baseline and was similar in both groups. In the PEGylated rhGH cohort, the mean ± SD HV was improved from 3.78 ± 0.78 cm/y at the baseline to 12.44 ± 3.80 cm/y at month 3, to 11.50 ± 3.01 cm/y at month 6, to 11.00 ± 2.32 cm/y at month 12, and finally 10.08 ± 2.12 cm/y at month 24 in the PEGylated rhGH group. In the daily rhGH group, HV was 3.36 ± 1.00 cm/y at baseline, increasing to 12.56 ± 3.71 cm/y at month 3, to 11.82 ± 2.63 cm/y at month 6, to 10.46 ± 1.78 cm/y at month 12, and to 9.28 ± 1.22 cm/y at month 24. No serious adverse event related to PEGylated rhGH or daily rhGH occurred during the 24-month study. Conclusion. PEGylated rhGH replacement therapy is effective and safe in pediatric patients with GHD. The adherence to once-weekly PEGylated rhGH therapy is superior to daily rhGH in children with GHD.

Individualised growth response optimisation (iGRO) tool: an accessible and easy-to-use growth prediction system to enable treatment optimisation for children treated with growth hormone

2017 ◽  
Vol 30 (10) ◽  
Author(s):  
Jane Loftus ◽  
Anders Lindberg ◽  
Ferah Aydin ◽  
Roy Gomez ◽  
Mohamad Maghnie ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Clinical Management ◽  
Growth Response ◽  
Prediction Models ◽  
Hormone Deficiency ◽  
Prediction System ◽  
Growth Prediction ◽  
It Systems ◽  
Short Children ◽  
Predicted Height

AbstractBackground:Growth prediction models (GPMs) exist to support clinical management of children treated with growth hormone (GH) for growth hormone deficiency (GHD), Turner syndrome (TS) and for short children born small for gestational age (SGA). Currently, no prediction system has been widely adopted.Content:The objective was to develop a stand-alone web-based system to enable the widespread use of an ‘individualised growth response optimisation’ (iGRO) tool across European endocrinology clinics. A modern platform was developed to ensure compatibility with IT systems and web browsers. Seventeen GPMs derived from the KIGS database were included and tested for accuracy.Summary:The iGRO system demonstrated prediction accuracy and IT compatibility. The observed discrepancies between actual and predicted height may support clinicians in investigating the reasons for deviations around the expected growth and optimise treatment.Conclusions:This system has the potential for wide access in endocrinology clinics to support the clinical management of children treated with GH for these three indications.

Discordant immune and growth response to pituitary and biosynthetic growth hormone in siblings with isolated growth hormone deficiency type IA

1989 ◽  
Vol 121 (5) ◽  
pp. 609-614 ◽  
Author(s):  
Berthold P. Hauffa ◽  
Ruth Illig ◽  
Toni Torresani ◽  
Herbert Stolecke ◽  
John A. Phillips
Keyword(s):  
Growth Hormone ◽  
Growth Hormone Deficiency ◽  
Growth Response ◽  
Chromosome 17 ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Growth Hormone Gene ◽  
Isolated Growth Hormone Deficiency ◽  
Type Ia ◽  
Deficiency Type

Abstract. Two brothers with familial isolated growth hormone deficiency type IA homozygous for the same 6.7 kb deletion on chromosome 17 including the growth hormone gene were intermittently treated with various forms of hGH for more than 7 years. While the elder brother (Patient 1) showed a good growth response to pituitary hGH, the younger one (Patient 2) developed high titre growth blocking hGH antibodies early in the course of treatment and grew only 2.2–3.9 cm/year on a hGH dose of 12–26 IU/m2 per week. When the younger brother was changed to a higher dose (33 IU/m2 per week) of biosynthetic methionyl hGH he had striking catch-up growth and he has subsequently maintained a height velocity of 10.0 cm/year for the last 2 years. During this time his antibody titres have decreased over 1000-fold. These findings demonstrate that therapy with biosynthetic methionyl hGH may provide an effective form of treatment for subjects with isolated growth hormone deficiency type IA who do not grow in response to native hGH, and imply that biosynthetic methionyl hGH may be less antigenic than pituitary derived hGH.

Recombinant Human Growth Hormone (rhGH) Treatment of Children Undergoing Peritoneal Dialysis

1990 ◽  
Vol 10 (3) ◽  
pp. 209-214 ◽  
Author(s):  
Richard N. Fine ◽  
Vera H. Koch ◽  
M. Ines Boechat ◽  
Barbara H. Lippe ◽  
Pauline A. Nelson ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Peritoneal Dialysis ◽  
Growth Velocity ◽  
Recombinant Human Growth Hormone ◽  
Standard Deviation Score ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Recombinant Growth Hormone ◽  
Rhgh Therapy ◽  
Rhgh Treatment

The authors studied the effect of recombinant growth hormone (rhGH) treatment on 5 growth retarded children, age 21/12 to 178/12 years, who had end-stage renal disease (ESRD) and were undergoing continuous cycling peritoneal dialysis (CCPD). Patients received 0.125 mg/kg of subcutaneous rhGH 3 times weekly. Accelerated height velocity compared to the previous year of CCPD was noted in 2 patients and improvement in the standard deviation score (SDS) as a parameter of improved growth velocity was noted in a third patient. This was associated with an increase in weight and improvement in the midarm muscle circumference (MAMC) suggesting an anabolic effect of rhGH treatment. Bone age advancement was consistent with the period of observation; no advancement greater than that expected for the increase in chronological age was observed. No significant side effects were attributable to rhGH therapy. These preliminary results indicate some growth retarded children without growth hormone deficiency with ESRD undergoing CCPD may respond to exogenous rhGH therapy with an acceleration in growth velocity: However, the failure to achieve uniform acceleration of height velocity indicates the need for controlled studies before rhGH can be recommended for all growth retarded children with ESRD undergoing peritoneal dialysis.

Increased growth rate following transfer to daily sc administration from three weekly im injections of hGH in growth hormone deficient children

1983 ◽  
Vol 104 (2) ◽  
pp. 148-152 ◽  
Author(s):  
K. W. Kastrup ◽  
J. Sandahl Christiansen ◽  
J. Koch Andersen ◽  
H. Ørskov
Keyword(s):  
Growth Hormone ◽  
Growth Rate ◽  
Growth Response ◽  
Pubertal Development ◽  
Human Growth ◽  
Bone Age ◽  
Hormone Deficiency ◽  
First Year ◽  
Weekly Dose ◽  
Second Year

Abstract. The effect of more frequent (daily) injections of human growth hormone (hGH) on growth rate was studied in 16 growth hormone deficient children (12 boys, 4 girls) during 2 years. All had previously been treated with im injection of hGH 2–3 times weekly and in the majority of the patients a waning growth response was observed. For a total weekly dose of 12 IU hGH a daily dose of 2 IU was injected sc at night before sleep. This dosage has been shown by us to imitate the average nocturnal hGH profile in plasma. Growth response on the im treatment was 5.2 ± 1.2 cm/year (sd) in boys and 5.4 ± 0.9 cm/year in girls. A significant increase was seen during the first year of sc treatment to 7.9 ± 2.7 cm in boys and 6.3 ± 2cm in girls. During the second year the growth response was still significantly increased in boys (7.2 ± 1.9 cm). Bone age was more advanced and the period of previous im treatment was longer in girls (6.7 vs 3.6 years) which may be the main cause of the waning second year response (4.7 ±1.3 cm/year). Pubertal development occurred in 9 children during treatment. However, the highest growth rates were not found in these children. Absence of antibodies against hGH and local reactions at the injection site is evidence of the safety of the treatment, which was very well accepted by the children. Daily sc injections thus represent an effective alternative to conventional im injections ensuring high acceptance in children with growth hormone deficiency.

scholarly journals Switch Data From the Open-Label Extension of the Pivotal Phase 3 Study of Once Weekly Somatrogon Compared to Daily Somatropin in Pediatric Patients With Growth Hormone Deficiency (pGHD)

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A686-A687
Author(s):  
Michael Wajnrajch ◽  
Bradley Scott Miller ◽  
Joel Steelman ◽  
Lawrence A Silverman ◽  
Moshe Phillip ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Recombinant Human Growth Hormone ◽  
Bone Age ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Main Study ◽  
Open Label ◽  
Phase 3 ◽  
Pivotal Phase ◽  
Open Label Extension

Abstract Objectives: Somatrogon (hGH-CTP) is a long acting recombinant human growth hormone, consisting of the amino acid sequence of hGH and three copies of the carboxy-terminal peptide (CTP) of human chorionic gonadotropin (hCG) being developed as a once weekly treatment for children with pGHD. This report summarizes data from the first year of the optional open-label extension (OLE) of the pivotal phase 3 global trial (ClinicalTrials. gov: NCT02968004), comparing the efficacy and safety of children switched from Genotropin (rhGH; somatropin) to somatrogon (Geno/Soma) and children maintained on somatrogon (Soma/Soma). Methods: During the main study, 224 children were randomized to receive either once weekly somatrogon (0.66 mg/kg, n=109) or once daily Genotropin (0.24 mg/kg/wk, n=115) for 12 months. Of these, 222 completed the 12-month main study, and 212 chose to enter the OLE study. By Sept 30, 2020, 161 children (including 76 Geno/Soma) had complete auxological data at month 12 of the OLE. Results: At the end of the main study, mean height velocity and gain in height SDS for the somatrogon cohort were 10.10 cm/year and 0.92; for the Genotropin cohort these were 9.78 cm/year and 0.87. Baseline values for the OLE (Soma/Soma group and Geno/Soma group, respectively): height SDS was -1.95 and -1.84, BMI was 17.03 and 15.48 kg/m2 while bone age was 6.54 and 6.40 years. At month 12 (of the OLE), the mean height velocity and the change in height SDS was 8.04 cm/year and 0.41 (Soma/Soma group) and 8.21 cm/year and 0.47 (Geno/Soma group); BMI was 18.07 and 17.49 kg/m2 and bone age was 8.48 and 8.41 years. IGF-1 SDS values were 1.15, and 1.28, while the IGFBP-3 SDS were 0.29 and 0.42, respectively. Dose reductions were required in 16.3% and 20.4% of patients due to IGF-1 SDS >2. Pubertal status changed from Tanner 1 (at OLE baseline) for 13.6% of Soma/Soma patients and 14.6% of Geno/Soma patients. Mean glucose, HbA1c, thyroid function (free T4 and TSH) and cholesterol (total, LDL and HDL) values remained similar to baseline in both groups across the 12 months OLE. The majority of adverse events in both cohorts were mild to moderate (Soma/Soma 94.2%, Geno/Soma 93.5%) and there were no clinically concerning safety observations. During the first 12 months of the OLE six patients discontinued in the Geno/Soma group due to AEs vs zero in the Soma/Soma group. Conclusions: Height velocities and change in height SDS in the OLE were similar between the Geno/Soma and Soma/Soma cohorts. The main phase of the global pivotal phase 3 trial demonstrated that somatrogon (hGH-CTP) given once weekly is non-inferior to Genotropin (hGH) while the OLE demonstrated that catch-up growth continued into the second year of treatment, with ‘switch’ from Genotropin to somatrogon non-inferior to somatrogon given for two years. Metabolic (glycemic, lipid and thyroid) parameters were similar between groups and not meaningfully different from the main study.

scholarly journals GROWTH HORMONE THERAPY;

2018 ◽  
Vol 20 (02) ◽  
pp. 182-187
Author(s):  
COL NAYYAR AHMAD, ◽  
COL. MOHAMMAD TARIQ NADEEM, ◽  
MAJ. ZAMEER AHMAD NAYYAR,
Keyword(s):  
Growth Hormone ◽  
Growth Hormone Deficiency ◽  
Short Stature ◽  
Hormone Replacement ◽  
Bone Age ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Growth Hormone Replacement ◽  
Female Ratio ◽  
The Mean

Objective: To detect growth hormone deficiency in short stature children and to observe the response of growth hormonereplacement therapy in isolated GH deficient. Design: An interventional descriptive study. Place and Duration of Study: The study wascarried out in the Department of Pediatrics at Military Hospital Rawalpindi in collaboration with Armed Forces Institute of PathologyRawalpindi over a period of two years from Jan 2007 to Dec 2008. Patients and Methods: Thirty short children between three to fourteenyears of age having isolated growth hormone deficiency confirmed by laboratory investigation were included in the study prospectivelyand retrospectively. Growth hormone replacement therapy with recombinant GH was given to all children at the dose of 0.14iu/kg, sixdays a week subcutaneously. Each patient was assessed and evaluated after every three months. Results: The mean chronologic agewas 8.05 +/- 2.74 years with a height age of 4.02 years. The male to female ratio was 1.72:1. They were treated with recombinant GH in adose of 0.14iu/kg, six days a week, subcutaneously at evening. Response to GH was excellent and the mean growth speed had gone upfrom 2.53 +/- 0.87 cm per year before the treatment to 8.94 +/- 3.18 cm / year in the first twelve months of treatment and 6.8 +/- 1.6cm / year during the second year of treatment. During the first twenty four months of treatment, height standard deviation score increasedby 1.0 +/- 0.4 SD (p < 0.0001) The height velocity increased, the bone age / chronological age ratio and height SDS for chronologicalage decreased, while height SDS for bone age increased. There were no adverse reactions. Conclusion: Short stature with classic growthhormone deficiency is not uncommon. Early diagnosis and prompt treatment with growth hormone replacement has a very goodoutcome and the child attains a reasonable height.

scholarly journals Weekly Lonapegsomatropin in Treatment-Naïve Children with Growth Hormone Deficiency: The Phase 3 heiGHt Trial

2021 ◽  
Author(s):  
Paul S Thornton ◽  
Aristides K Maniatis ◽  
Elena Aghajanova ◽  
Elena Chertok ◽  
Elpis Vlachopapadopoulou ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Hormone Deficiency ◽  
Bone Age ◽  
Primary Objective ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Weekly Dose ◽  
Open Label ◽  
Phase 3 ◽  
Treatment Naïve

Abstract Context For children with growth hormone deficiency (GHD), treatment burden with daily somatropin injections (hGH) is high, which may lead to poor adherence and suboptimal overall treatment outcomes. Lonapegsomatropin (TransCon hGH) is an investigational long-acting, once-weekly prodrug for the treatment of GHD. Objective The objective of this study was to evaluate the efficacy and safety of once-weekly lonapegsomatropin vs daily somatropin. Design The heiGHt Trial was a randomized, open-label, active-controlled, 52-week phase 3 trial (NCT02781727). Setting This trial took place at 73 sites across 15 countries. Patients This trial enrolled and dosed 161 treatment-naïve, prepubertal patients with GHD. Interventions Patients were randomized 2:1 to receive lonapegsomatropin 0.24 mg hGH/kg/wk or an equivalent weekly dose of somatropin, delivered daily. Main Outcome Measure The primary end point was annualized height velocity (AHV) at Week 52. Secondary efficacy end points included change from baseline in height standard deviation scores (SDS). Results Least squares (LS) mean (SE) AHV at 52 weeks was 11.2 (0.2) cm/year for lonapegsomatropin vs. 10.3 (0.3) cm/year for daily somatropin (P=0.009), with lonapegsomatropin demonstrating both non-inferiority and superiority over daily somatropin. LS mean (SE) height SDS increased from baseline to Week 52 by 1.10 (0.04) vs. 0.96 (0.05) in the weekly lonapegsomatropin vs. daily somatropin groups (P=0.01). Bone age/chronological age ratio, adverse events, tolerability, and immunogenicity were similar between groups. Conclusions The trial met its primary objective of non-inferiority in AHV and further showed superiority of lonapegsomatropin compared to daily somatropin, with similar safety, in treatment-naïve children with GHD.

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.

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