hrp0095rfc7.2 | Growth and Syndromes | ESPE2022

Once-Weekly Somapacitan vs Daily GH in Children with GH Deficiency: The Randomized Phase 3 REAL 4 Trial

Miller Bradley , Blair Joanne , Højby Michael , Böttcher Volker , Juul Kildemoes Rasmus , Maniatis Aristides , Beck Bang Rikke , Mori Jun , Polak Michel , Stagi Stefano , Horikawa Reiko

Background: Growth hormone (GH) replacement therapy usually requires daily subcutaneous (s.c.) injections that can be burdensome for patients and their caregivers. Long-acting GH formulations aim to establish a less burdensome dosing regimen that is as safe and efficacious as daily GH to potentially improve adherence and clinical outcomes. Somapacitan, a long-acting reversible albumin-binding GH derivative, is in development for once-weekly s.c. administration...

hrp0097fc11.1 | GH and IGFs | ESPE2023

GH replacement therapy with once-weekly somapacitan in children with GH deficiency is effective and well-tolerated: 2-year results from REAL4

Miller Bradley , Blair Joanne , Højby Rasmussen Michael , Maniatis Aristides , Mori Jun , Böttcher Volker , Bang Rikke , Polak Michel , Horikawa Reiko

Daily subcutaneous (s.c.) injections of growth hormone (GH) to treat GH deficiency (GHD) in children is burdensome for both patients and caregivers. Somapacitan (Novo Nordisk) is a long-acting reversible albumin-binding human GH derivative in development for once-weekly s.c. administration in children with GHD, and aims to overcome the treatment burden of daily injections. REAL4 is a multi-national, randomised, open labelled phase 3 trial with a 52-week main phase followed by ...

hrp0098fc15.6 | Late Breaking | ESPE2024

Pharmacodynamic Endpoints After Once-Weekly Somapacitan in Children With GHD: 3-year results from REAL4 phase 3 study

S. Miller Bradley , C. Blair Joanne , Højby Rasmussen Michael , Maniatis Aristides , Mori Jun , Böttcher Volker , Kim Ho-Seong , Polak Michel , Horikawa Reiko

Somapacitan (Novo Nordisk A/S) is a long-acting GH derivative, approved for once-weekly administration in children and adults with GHD. IGF-I is monitored and includes a fraction bound to binding proteins, e.g., IGF binding protein-3 (IGFBP-3). The IGF-I/IGFBP-3 molar ratio can be used as surrogate marker for freely circulating bioactive IGF-I. REAL4 is a randomised, open-label, multi-national phase 3 trial (NCT03811535) with a 52-week main phase and a 3-year extension (week 5...

hrp0095p1-224 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

High Vitamin Deficiency Throughout Childhood. Inma-Asturias Cohort. Spain

Rodríguez-Dehli Cristina , Fernández-Somoano Ana , Riaño-Galán Isolina , Venta Rafael , Tardon Adonina

Background: Vitamin D (25OHD) insufficiency is becoming a public health issue. Adequate 25OHD status during pregnancy may influence the health status of the offspring.Objective: To evaluate the levels of 25OHD from gestation to 12 years, assessing seasonality and intake.Material and methods: Data were obtained from 485 pregnant mothers recruited in Asturias between 2004-2007 and th...

hrp0095p1-60 | Diabetes and Insulin | ESPE2022

Prevalence of Autoimmunity in Relatives of Patients with Type 1 Diabetes: Time to The Clinical Onset of Diabetes and Associated Risk Factors

Marcelo Irene , Urrutia Ines , Calvo Begoña , Martinez Rosa , Saso Laura , Castaño Luis , Rica Itxaso , Group Collaborative

Introduction: Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the presence of pancreatic-autoantibodies, which are diagnostic biomarkers. The detection of autoantibodies in first-degree relatives of patients with T1D is considered a risk factor for developing the disease; however, there is little information on how long it takes from the detection of autoimmunity to the clinical onset of T1D.Objectives:</st...

hrp0095p1-69 | Fat, Metabolism and Obesity | ESPE2022

Clinical Evolution After A 24 Month Nutritional Intervention Based on Carbohydrate Restriction in Adolescents with Obesity

González-Leal Rocío , María Andrés Esteban Eva , Argente Jesús , Ángel Martos-Moreno Gabriel

Introduction: Carbohydrate restriction (CH-R) in adolescents with obesity results in faster BMI reduction and glucose metabolism improvement than diets with normal carbohydrate proportion, without a higher weight regain in the short term. However, data on weight and metabolic trajectories of these patients after a more prolonged follow-up are sparse.Objectives: To evaluate metabolic and body composition evolution during ...

hrp0095p1-314 | Growth and Syndromes | ESPE2022

Klinefelter Syndrome: Clinical Spectrum Based on 21 years experience of a Pediatric Endocrinology Unit

Filipa Pinto Oliveira Joana , Cardoso Sandra , João Oliveira Maria , Freitas Joana , Ribeiro Luís , Mendes Catarina , Borges Teresa

Background and Aims: Klinefelter syndrome (KS) is the most common chromosomal abnormality in men, but because of its highly variable phenotype and clinical unawareness, it remains largely under-diagnosed. Cardinal features include tall stature, gynecomastia, hypergonadotropic hypogonadism, azoospermia and infertility. Learning and behavioral problems are also common. Testosterone replacement therapy (TRT) helps to prevent or ameliorate many of the comorbiditie...

hrp0095lb13 | Late Breaking | ESPE2022

Residual C-peptide secretion and glycemic control in pediatric patients with Diabetes Mellitus type 1

João Gaia Maria , Pedrosa Inês , Adriana Rangel Maria , Arménia Campos Rosa , Luísa Leite Ana

Introduction: Residual C-peptide secretion has been associated with less hypoglycemic events and lower glycemic variability in patients with Diabetes Mellitus type 1 (DM1), primarily in adults. The authors sought to evaluate the association between residual C-peptide secretion in pediatric patients with DM1 and glycemic control variables.Methods: Cross-sectional study conducted in a level II hospital, in DM1 patients you...

hrp0095p2-145 | GH and IGFs | ESPE2022

A user-centred approach to improve digital health solutions in patients requiring growth hormone therapy: Results of a participatory workshop

Alherbish Abdullah , Kaplan Walid , Fernandez-Luque Luis , Rivera-Romero Octavio , Tamer Eladly Farag , Martin O. Savage , Koledova Ekaterina

Background: Digital health is becoming increasingly important worldwide. However, there is a lack of research on barriers to the implementation of digital health solutions for growth disorders in the Gulf Region. Participatory research can help to understand barriers faced by different stakeholders, design effective solutions and evaluate their implementation, whilst taking cultural aspects into consideration. Involving clinicians in understanding barriers to ...

hrp0089p3-p141 | Fat, Metabolism and Obesity P3 | ESPE2018

Serum Hepcidin and Ferritin in Prepubertal Obese Children

Gajewska Joanna , Klemarczyk Witold , Ambroszkiewicz Jadwiga , Gła¸b-Jabłońska Ewa , Chełchowska Magdalena

Introduction: Obesity is the direct cause of a number of immediate problems during childhood. Recently, fat mass was described as a significant negative predictor of serum iron. Hepcidin is a hormone stimulated by an increase in plasma iron levels and iron deposits in tissues, and decreases iron release from macrophages and duodenal enterocytes into the plasma. This protein prevents excessive iron absorption and iron accumulation in tissues. Ferritin is the most commonly deplo...