hrp0089p1-p158 | GH & IGFs P1 | ESPE2018

Patients and Caregivers Perspectives on a Mobile App that Tracks Adherence and Outcomes in Children with Growth Disorders Treated with Recombinant Human Growth Hormone (r-hGH)

McNally Mark , Long Frank , Poskitt Henry , Cancela Jorge , Koledova Ekaterina , Castro Javier Sanchez

Healthcare professionals (HCPs) receive adherence information on patient Saizen® recombinant human growth hormone (r-hGH) treatment via data wirelessly transferred from the easypodTM electromechanical delivery device to the web-based eHealth platform easypodTM connect. In order to empower patients and caregivers with this information and to provide educational tools, the growlinkTM mobile app is being ...

hrp0086rfc14.5 | Growth : Mechanisms | ESPE2016

Gene Expression Profiling of Children with GH Deficiency (GHD) Prior to Treatment with Recombinant Human Growth Hormone (r-hGH) is Associated with Growth Response Over Five Years of Therapy

Stevens Adam , Murray Philip , Koledova Ekaterina , Chatelain Pierre , Clayton Peter

Background: The relationship of pre-treatment gene expression (GE) to long-term growth response in GHD is unknown. Prediction of long-term response to r-hGH therapy would allow better decision making about start and maintenance doses and hence cost:benefit.Objective and hypotheses: To assess the relationship of baseline GE to response to r-hGH over 5 years of therapy in GHD children.Method: Pre-pubertal children with GHD (n</em...

hrp0097p1-99 | GH and IGFs | ESPE2023

Clinicians’ perceptions on ease of use and usefulness of Aluetta® Smartdot™ and comprehensive digital health ecosystem in Italy to support patients receiving r-hGH treatment

Felicia Faienza Maria , Vergani Edoardo , Di Mase Raffaella , Frasca Francesco , Giavoli Claudia , Klain Antonella , Laura Iezzi Maria , Aversa Antonio , Koledova Ekaterina , Rivera Romero Octavio

Background: Aluetta® Smartdot™ (Merck Healthcare KGaA, Darmstadt, Germany) is first of its kind digitally connected smart injection pen device for recombinant-human growth hormone (r-hGH) administration. Aluetta® pen with Smartdot™ knob attachment integrated with Growzen™ digital ecosystem enables healthcare professionals (HCPs) to remotely monitor adherence and achieve optimal clinical outcomes for patients....

hrp0095p1-223 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Real-world data in children with achondroplasia after licensing of Vosoritide

Palm Katja , Bechthold-Dalla Pozza Susanne , Gausche Ruth , Högler Wolfgang , Hoyer- Kuhn Heike , Hübner Angela , Keller Alexandra , Mirante Alice , Mohnike Klaus , Muschol Nicole , Nader Sean , Pfäffle Roland , Quitter Friederike , Rohrer Tilmann , Rutsch Frank , Schnabel Dirk , Semler Oliver , Silva Isabel , B. Sousa Sérgio , M.K. Voelkl Thomas , Wechsung Katja , Weigel Johannes , Woelffle Joachim , Lausch Ekkehart

Background: Achondroplasia (ACH), caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene (FGFR3), is characterized by severe growth failure and may be associated with multisystemic complications. The clinical phenotype is variable and relates to deformity of rhizomelic shortened legs, and myelon compression at cranial base and spine. Recent guidelines are published for diagnostic workflow, neurosurgical, orthopaedic and otorhinolaryngol...

hrp0097p1-293 | GH and IGFs | ESPE2023

Healthcare professionals’ perceptions on the quality and evolution of digital health devices to support paediatric growth hormone therapy: Results of a UK participatory study

Rivera-Romero Octavio , R Kapoor Ritika , Mathew Verghese , Tollerfield Sally , Shah Pratik , Banerjee Indraneel , Koledova Ekaterina

Background: Long-term treatment with recombinant human growth hormone (r-hGH) is often challenging for children and adolescents, their caregivers and healthcare professionals (HCPs), as treatment requires daily injections over several years. Connected digital health devices facilitate this by automating the injection process to improve comfort, reduce anxiety and collect GH treatment data so that accurate adherence information is available to HCPs in real-time...

hrp0082np2.1 | Regenerative Endocrinology | ESPE2014

Regenerative Medicine for β Cell Replacement

Scharfmann R

Pancreatic β cells develop from endodermal pancreatic progenitors that first proliferate and next differentiate into functional insulin-producing cells. This developmental process is complex, each step being controlled by yet unknown signals. Theoretically, the development of a functional β cell mass can be enhanced by: i) activating the proliferation of pancreatic progenitors; ii) activating their differentiation into β cells; iii) activating the proliferation ...

hrp0082p2-d1-460 | Growth | ESPE2014

Skeletal Disproportion and Final Adult Height in Childhood-Onset Crohn’s Disease

Mason Avril , Iljuhhina Jelena , Laird Susan , Ross David , Ahmed Syed Faisal , Gaya Dan R , Gerasamidis Konstantinos

Background: Short stature is a recognized complication of Crohn’s disease (CD) in children. Less is known on the impact of disease on final adult height and body proportions.Objective and hypotheses: To assess height and body proportions in 48 adults (F: 30; M: 18) with childhood (ACO) or adult (AAO) onset CD and 44 children (C) (F:22; M:22) with CD.Method: Standing, sitting height (SH) and leg length (SILL) were measured and ...

hrp0089p1-p134 | Fat, Metabolism and Obesity P1 | ESPE2018

Evaluation of Intraocular Pressure and Retinal Nerve Fiber Layer, Retinal Ganglion Cell, Central Macular Thickness and Choroidal Thickness Using Optical Coherence Tomography in Obese Children and Healthy Controls

Taner Baran Rıza , Baran Serpil , Fusun Toraman Naciye , Bilgilisoy Filiz Meral , Filiz Serkan , Demirbilek Huseyin

Objective: Obesity and its complications affecting many organ systems have been documented. Nevertheless, study conducted on the ophthalmological effects of obesity are scarce. The aim of the present study was to evaluate the changes in the ophthalmological parameters in obese children in comparison to their healthy counterparts.Material and methods: Study included 61 obese and 35 age-sex matched controls. Obesity was defined as body mass index- standard...

hrp0094p1-38 | Fat, Metabolism and Obesity A | ESPE2021

Are Serum Spexin Levels Associated With Metabolic Syndrome Antecedents In Obese Adolescents?

Gulderen Kalay Şenturk Nida , Cakır Aydilek Dağdeviren , Yıldırmak Zeynep Yıldız , Ucar Ahmet ,

Objective: Spexin is a novel peptide implicated in food intake and satiety. Spexin levels are reduced in obese patients.Aim: To evaluate the associations of metabolic syndrome (metS) antecedents with serum spexin levels in obese adolescents.Setting: A university- based tertiary care centre.Patients and methods: Eighty consecutive obese adolescents aged 10-18 y and 80 healthy ...