hrp0089rfc15.5 | Growth and syndromes | ESPE2018

Effect of Adjusting for Tanner Stage Age on Short and Tall Stature Prevalence in US Youths

Addo O Yaw , Sarafoglou Kyriakie , Miller Bradley

Background: Although differences in pubertal timing alters frequency of indicators of attained stature at the extremes, its magnitude is unknown across ethnic groups of US youths.Methods: We performed analyses of anthropometry and Tanner staging data of 3206 cross-sectional national sample of youths ages 8–18y (53% male (n=1606), 72% Non-Hispanic White (NHW), 9% Mexican American (MA) and 19% Non-Hispanic Black (NHB). Specialized Tanner-stag...

hrp0086p1-p623 | Growth P1 | ESPE2016

Autosomal Dominant Growth Hormone Deficiency due to a Novel Mutation in the gh1 Gene

Ternand Christine , Gao Harry , Miller Bradley

Background: Familial growth hormone deficiency (GHD) with an autosomal dominant inheritance pattern (isolated GHD type II) due to multiple different mutations in the GH1 gene have been described.Objective and hypotheses: Describe the clinical characteristics and mutation analysis of affected individuals in a family with growth hormone deficiency inherited in an autosomal dominant pattern.Method: Medical record review.<p class="...

hrp0092p1-226 | Growth and Syndromes (to include Turner Syndrome) (1) | ESPE2019

Response to Growth Hormone in Very Young Children (® International Outcome Study and ANSWER Program

Rohrer Tilman R , Miller Bradley , Ostrow Vlady , Pietropoli Alberto , Polak Michel , Ross Judith

Objectives: Limited information is available on how very young children with growth hormone deficiency (GHD) respond to growth hormone (GH) replacement. We compared response to 1 year of GH therapy in children aged <2 years and prepubertal children aged ≥2 years.Methods: The two non-interventional, multicentre studies, NordiNet® International Outcome Study (IOS) (NCT00960128) and the ANSWER Prog...

hrp0089p1-p141 | GH &amp; IGFs P1 | ESPE2018

Autosomal Dominant Growth Hormone Deficiency due to a Novel c.178G>A Mutation in the GH1 Gene Causing Instability of the Mutant GH Protein (p.Ala34Thr)

Miller Bradley , Tan Jimmy , Parween Shaheena , Eble Andree , Ternand Christine , Gregory Louise , Dattani Mehul , Pandey Amit

Background: The most frequent cause of familial growth hormone deficiency (GHD) is Type II autosomal dominant GHD (isolated GHD type II) due to several heterozygous GH1 mutations.Method: Genomic DNA from patients with familial GHD was enriched for the coding exons using hybrid capture technology and GH1 was sequenced using Next Generation Sequencing technology. Plasmids containing genes for WT and A34T GH1 were transfected into NCI HEK2...

hrp0094p2-369 | Pituitary, neuroendocrinology and puberty | ESPE2021

6-Month GnRHa Formulations Are a Good Choice During the COVID-19 Pandemic and Beyond

R. Benson Matthew , N. Atkinson Stuart , M. Boldt-Houle Deborah , Miller Bradley S. ,

Introduction: Achieving/maintaining effective hormone suppression is fundamental in treating Central Precocious Puberty(CPP). CPP patients are vulnerable to late dosing as they cannot self-administer and require clinic/hospital visits for injections, currently exacerbated by COVID-19. In children, the hypothalamic-pituitary-gonadal axis may rebound faster than elderly oncology patients, so hormone escapes are possible with late dosing. Additionally, the stimul...

hrp0095fc5.4 | Adrenals and HPA Axis | ESPE2022

Reference Growth Charts in Children with Congenital Adrenal Hyperplasia

Sarafoglou Kyriakie , Miller Bradley , Munoz Yesica , Jaber Mu'taz , Yaw Addo O.

Introduction: Adult height in individuals with Congenital Adrenal Hyperplasia (CAH) is reduced compared to the general population as their growth during childhood can be negatively impacted by both the disease and its treatment. Excess production of androgens through aromatization to estrogens can accelerate height velocity and skeletal maturity, and lead to short stature if not adequately suppressed. Over suppression of adrenal steroids through excess glucoco...

hrp0086rfc8.1 | Growth: Clinical | ESPE2016

Somavaratan (VRS-317) Treatment of Children with Growth Hormone Deficiency (GHD): Results at 2 Years (NCT02068521)

Bright George , Moore Wayne V. , Nguyen Huong Jil , Kletter Gad B. , Miller Bradley S. , Fechner Patricia Y. , Ng David , Humphriss Eric , Cleland Jeffrey L.

Background: Somavaratan, a novel long-acting rhGH fusion protein with t1/2>100 h, previously demonstrated clinically meaningful improvements in height velocity (HV) and IGF-I in prepubertal GHD children (Moore JCEM 2016).Objective and hypotheses: To evaluate maintenance of somavaratan treatment effects in the 2nd treatment year.Method: After subcutaneous pediatric doses were evaluated in a single dose PK/PD study (<e...

hrp0082p1-d3-171 | Growth (2) | ESPE2014

Safety and Efficacy Results of a 6 Month, Randomized, Multi-Center Trial of a Novel Long-Acting rhGH (VRS-317) in Naïve to Treatment, Pre-Pubertal Children with GH Deficiency

Bright George M , Moore Wayne V , Nguyen H Q , Kletter Gad B , Miller Bradley S , Rogers Douglas G , Humphriss Eric , Cleland Jeffrey

Background: VRS-317, a novel fusion protein of rhGH, was safe and well tolerated in single dose studies of adults and children with growth harmone deficiency (GHD).Objectives: Conduct a 6-month study to determine the safety, tolerability, height velocity and IGF1 response in GHD children.Methods: 64 subjects were randomized into three arms to evaluate monthly, semi-monthly and weekly dosing.Results: At VRS-31...

hrp0084lbp-1267 | Late Breaking Posters | ESPE2015

Safety and Efficacy of Long-Acting GH (VRS-317) in Children with GHD: Effects of Dose Change in the Second Treatment Year

Bright George , Moore Wayne V , Nguyen Huong Jil , Kletter Gad B , Miller Bradley S , Fechner Patricia Y , Ng David , Humphriss Eric , Cleland Jeffrey L

Background: VRS-317, a novel fusion protein of rhGH exhibiting delayed clearance, serum half-life generally >100 h, and potential for once monthly dosing, was previously evaluated in a 6-month phase 1b/2a study of weekly, twice monthly or monthly dosing (5.0 mg/kg per month) in prepubertal GHD children (n=64).Objective and hypotheses: We evaluated whether increased VRS-317 dose from 12 to 18 months can offset the decrease in height velocitie...

hrp0094p2-300 | Growth and syndromes (to include Turner syndrome) | ESPE2021

Association of Daily Growth Hormone Injection Adherence and Height among Children with Growth Hormone Deficiency

Loftus Jane , Miller Bradley S , Parzynski Craig , Alvir Jose , Chen Yong , Jhingran Priti , Gupta Anu , DeKoven Mitch , Divino Victoria , Tse Jenny , He Jing , Wajnrajch Michael ,

Background: Use of recombinant human growth hormone (somatropin) is recommended for children with growth hormone deficiency (GHD) to normalize adult height and avoid extreme short stature. Prior research conducted among small study samples, across different study populations and health care systems, has suggested an association between adherence to somatropin and clinical outcomes, including height velocity. There is a need for further research, using real-wor...