hrp0095t7 | Section | ESPE2022

Efficacy and safety of once-weekly somatrogon in pediatric subjects with growth hormone deficiency: lack of impact of anti-drug antibodies

Deal Cheri , Silverman Lawrence , Korth-Bradley Joan , Roland Carl , Taylor Carrie , Cara Jose , Wajnrajch Michael

Objectives: Somatrogon, a long-acting recombinant human growth hormone (GH) consisting of the amino acid sequence of human GH (hGH) and three copies of the carboxy-terminal peptide (CTP) of human chorionic gonadotropin, is approved by the EMA for treatment of children with GH deficiency (GHD). In this phase 3 study, children with GHD received either somatrogon or Genotropin. The impact of testing positive for anti-drug antibodies to somatrogon (ADA+) on the ef...

hrp0095p1-220 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Hypocalcemia as the Initial Presentation of Type2 Bartter Syndrome: A Family Report

London Shira , A. Levine Michael , Li Dong , Spiegel Ronen , Lebel Asaf , Tenenbaum-Rakover Yardena

Context: Bartter syndrome (BS) is a group of rare autosomal-recessive tubulopathies characterized by hypokalemic, hypochloremic metabolic alkalosis in which the primary defect is a deficiency of transporters involved in sodium chloride reabsorption. Type 2 BS results from a defect in the renal outer medullary potassium channel encoded by the KCNJ1 gene. Type 2 BS presents with polyhydramnios, intrauterine growth retardation, prematurity, failure to thrive, pol...

hrp0095p1-64 | Fat, Metabolism and Obesity | ESPE2022

microRNA-27a - a strand specific regulator of adipogenesis and adipocyte function

Roos Julian , Wu Hang , Pula Taner , Tews Daniel , Wabitsch Martin , Debatin Klaus-Michael , Fischer-Posovszky Pamela

MicroRNAs (miRNAs) are small non-coding RNA molecules and play an important role in the post-transcriptional regulation of gene expression and thereby influence important cellular functions. In adipocytes, miRNAs regulate key processes such as differentiation, inflammation, metabolism, and adipokine secretion. Recently, we found that miR-27a is upregulated under proinflammatory conditions in human adipocytes. Thus, our goal was to better understand the function of miR-27a in a...

hrp0095p1-462 | Fat, Metabolism and Obesity | ESPE2022

Energy Trade-Off Score - a novel anthropometric polygenic trait, and related body types

Hochberg Ze'ev , Albertsson-Wikland Kerstin , Privé Florian , Holmgren Anton , Rubin Lisa , German Alina , Shmoish Michael

Background/Aim: The trade-off theory claims that a resource (calorie) stored in adipose tissue cannot be used for longitudinal growth, and a calorie used for growth will not be stored as fat.Methods: Out of 2339 children with longitudinal heights and weights (birth to adulthood) from the GrowUp1990 Gothenburg cohort, 1993 (996 girls) were analyzed. For everyone we calculated Energy Trade-Off Score: ETOS = at early adulth...

hrp0095p1-119 | Growth and Syndromes | ESPE2022

Post-hoc subgroup analysis of Asian subjects from the pivotal phase 3 study of once-weekly somatrogon vs once-daily Genotropin

Gomez Roy , Khadilkar Vaman , Shembalkar Jayashri , Chu Der-Ming , Woo Ko Cheol , Wajnrajch Michael , Wang Ronnie

Objectives: Somatrogon is a long-acting recombinant human growth hormone (GH) approved by the EMA as a once weekly treatment for children with pediatric GH deficiency (GHD). A global phase 3 study compared the efficacy and safety of once-weekly somatrogon with once-daily Genotropin in pediatric subjects with GHD. The objective of this subgroup analysis was to evaluate the efficacy and safety of once-weekly somatrogon vs once-daily Genotropin in the subset of A...

hrp0095p1-328 | Growth and Syndromes | ESPE2022

A real-world study of persistence with daily growth hormone therapy among children with growth hormone deficiency in Japan

Loftus Jane , Wogen Jenifer , Benjumea Darrin , Jhingran Priti , Chen Yong , Alvir Jose , Wajnrajch Michael

Background: Paediatric growth hormone deficiency (PGHD) is treated with daily somatropin (recombinant human growth hormone) injections. High rates of discontinuation and poor adherence to treatment, which are associated with worse growth outcomes, have been documented previously, for example in the US and EU. Discontinuation of somatropin has not yet been evaluated using real-world data in Japan.Objectives: To describe d...

hrp0095p1-108 | Growth and Syndromes | ESPE2022

Efficacy, Observer-Reported Outcomes, and Safety of Once-Weekly Somapacitan in Children with Growth Hormone Deficiency (GHD): 4-Year Results from the REAL 3 Trial

Sävendahl Lars , Battelino Tadej , Højby Rasmussen Michael , Brod Meryl , Wai Lee Kai , Saenger Paul , Horikawa Reiko

Children with GHD are currently treated with daily subcutaneous growth hormone (GH) injections, which can be burdensome. Somapacitan is a long-acting GH derivative in development for once-weekly use in children with GHD. REAL 3 (NCT02616562) is a phase 2, multinational, randomised, open label, controlled trial assessing efficacy and safety of somapacitan vs daily GH (Norditropin®). Prepubertal, GH-naïve children with GHD received 0.04 (n=16), 0.08 (n</e...

hrp0095lb16 | Late Breaking | ESPE2022

Glucose variability in 6–12-month-old healthy infants

Hauschild Michael , Monnard Cathriona , L. Eldridge Alison , Hansen Erik , A. Dwyer Andrew , Rytz Andreas , Darimont Christian

Background: Continuous glucose monitoring (CGM) devices are novel tools to measure the impact of dietary intake on glucose rhythms/metabolism in children. There is a significant gap in the literature on glycemic response in healthy, term infants and young children up to 2 years of age.Objective: To investigate glucose excursions in response to infant feeding, using CGM in healthy 6–12-month-old infants.<p class...

hrp0095p2-28 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Longitudinal assessment of bone health index (BHI) as a measure of bone health in short-statured children before and during treatment with recombinant growth hormone

Holzapfel Lukas , Bettendorf Markus , Rabe Michael , Mittnacht Janna , Choukair Daniela , Schenk Jens-Peter

Introduction: Bone Health Index (BHI) determined by measurement of cortical thickness of metacarpalia II-IV in x-rays of the left hand represents a method to estimate bone health in children. Aim of this study was to investigate changes of BHI SDS in the course of growth hormone (GH) treatment.Method: 256 consecutive children with short stature (isolated GH deficiency (IGHD) n = 121, multiple pituitary hormone deficiency...

hrp0092fc5.1 | Thyroid | ESPE2019

Randomised Trial of Block and Replace Versus Dose Titration Antithyroid Drug Treatment in Children and Adolescents with Thyrotoxicosis

Wood Claire , Cole Michael , Donaldson Malcolm , Dunger David , Matthews John , Pearce Simon , Cheetham Tim

Background: First line treatment for thyrotoxicosis is thionamide (TA) antithyroid drug therapy. The TA used is usually Carbimazole in the UK. TA can be administered in a relatively large 'blocking' dose that prevents endogenous thyroid hormone synthesis, requiring thyroid hormone replacement (block and replace or BR), or in a smaller dose that renders the patient euthyroid (dose titration or DT). The American Thyroid Association (ATA) recommends DT be...