hrp0094fc9.3 | Growth Hormone and IGFs | ESPE2021

Once-weekly somapacitan versus daily growth hormone in growth hormone deficiency: 3 year efficacy and safety results from REAL 3, a randomised controlled phase 2 trial

Savendahl Lars , Battelino Tadej , Rasmussen Michael Hojby , Saenger Paul , Horikawa Reiko ,

Growth hormone (GH) replacement therapy currently requires daily subcutaneous injections. Somapacitan is a long-acting GH-derivative in development for once-weekly use in children with GH deficiency (GHD). A phase 2, multinational, randomised, open-label, controlled trial (NCT02616562) investigated the efficacy and safety of somapacitan compared with daily GH (Norditropin®). GH-treatment-naïve prepubertal children with GHD received 0.04 (n = 16), 0.08 (n...

hrp0089fc10.2 | Late Breaking | ESPE2018

Efficacy and Safety of Once-Weekly Somapacitan in Childhood Growth Hormone Deficiency: Results of a Randomised Open-Label, Controlled Phase 2 Trial

Savendal Lars , Rasmussen Michael , Horikawa Reiko , Khadilkar Vaman , Battelino Tadej , Saenger Paul

Background: Growth hormone deficiency (GHD) requires long-term daily injections with GH replacement therapy and is associated with considerable treatment burden by patients and caregivers. Somapacitan is a long-acting GH derivative that is being developed for once-weekly dosing in adults and children with GHD. A well-established protraction method, which is successfully in use to extend the half-life of insulin and glucagon-like peptide (GLP)-1, has been applied in developing ...

hrp0092fc14.5 | GH and IGF5 | ESPE2019

OOnce-weekly Somapacitan vs Daily Growth Hormone (Norditropin®) in Childhood Growth Hormone Deficiency: One-year Results from a Randomised Phase 2 Trial

Sävendahl Lars , Battelino Tadej , Horikawa Reiko , Juul Rasmus Vestergaard , Rasmussen Michael Højby , Saenger Paul

Daily injections are required for human growth hormone (GH) replacement therapy. Somapacitan is a long-acting GH derivative being developed for once-weekly dosing in adults and children using a proven protraction method, currently successfully in use to extend the half-life of insulin and glucagon-like peptide 1.To evaluate the efficacy and safety of three different once-weekly somapacitan doses compared with Norditropin®, a daily GH, ove...

hrp0094p1-161 | Growth B | ESPE2021

Continued Safety and Efficacy of Weekly Lonapegsomatropin (TransCon hGH) for up to Two Years in Children with Growth Hormone Deficiency (GHD)

Aghajanova Elena M. , Casella Samuel J. , Nadgir Ulhas , Hofman Paul , Saenger Paul , Song Wenjie , Mao Meng , Chessler Steven , Komirenko Allison S. , Beckert Michael , Shu Aimee D. , Thornton Paul S. , Maniatis Aristides K. ,

Lonapegsomatropin (TransCon hGH) is an investigational once-weekly prodrug of somatropin for the treatment of GHD. Previous trials in treatment-naïve (52-week heiGHt Trial) and treatment-experienced children (26-week fliGHt Trial) have reported the efficacy and safety of lonapegsomatropin. Subjects were eligible to enter the open-label extension enliGHten Trial, which continues to evaluate weekly lonapegsomatropin in pediatric GHD. In heiGHt, treatment-naïve subjects...

hrp0086s6.1 | Prevention of childhood obesity | ESPE2016

Long-term Consequences of Childhood Obesity: The Impact of Genes and Lifestyle

Franks Paul

In most complex traits, susceptibility to certain risk exposures and response to clinical interventions in is under genetic control, a concept broadly termed “gene-environment interaction”. Although in animals and in plants there is evidence supporting this notion, in humans most evidence is confined to rare monogenic disorders. In complex diseases like type 2 diabetes and obesity, interactions between genetic and environmental risk factors are likely to begin very e...

hrp0094wg7.3 | ESPE Working Group for Paediatric Endocrine Nurses (ESPEN) Symposium | ESPE2021

Nursing care for the child with gender dysphoria

Carruthers Paul ,

This presentation will provide an insight into the Gender identity Development service (GIDS) endocrine pathway. The service has experienced a difficult year with well documented legal challenges; these will be briefly presented highlighting the impact this has had on patient care. It is important when working with gender incongruent young people to be aware of the use of appropriate pronouns, co-occurring difficulties and the importance of advocacy in our nursing practice. Th...

hrp0094p1-26 | Diabetes A | ESPE2021

Efficacy and Tolerability of GLP-1 Receptor Agonists in Children and Adolescents with Obesity

Ryan Paul ,

Background: Rates of pediatric obesity are on a concerning upward trajectory globally, with the prevalence approaching 1 in 5 children and adolescents aged 2-19 years in the United States. As a direct consequence of this obesity surge, we will continue to experience a substantial adult cardiometabolic burden. Although public health measures to optimize lifestyle interventions and reduce culpable environmental exposures are the primary target for governments an...

hrp0089s8.2 | Thyroid disorders | ESPE2018

Central Hypothyroidism – an Update

van Trotsenburg Paul

Central hypothyroidism can be best defined as lower than desirable thyroid hormone production and secretion because of insufficient stimulation of a normal thyroid gland by a defective pituitary or hypothalamus, resulting in a too low plasma or serum (free) thyroxine (FT4) concentration accompanied by a more or less normal thyrotropin (TSH) concentration. Central hypothyroidism can occur isolated or as part of multiple pituitary hormone deficiency and can be a congenital or ac...

hrp0082p2-d3-479 | Hypoglycaemia | ESPE2014

The Majority of Late Presenting Congenital Hypoglycaemia Disorders are Really Missed Diagnosis: What Can we do to Improve Diagnosis in the New-Born Period?

Troung Lisa , Thornton Paul

Background: Patients with hyperinsulinism (HI) and anterior hypopituitarism often present in the new-born period (NBP). However up to 30% patients with HI and an unknown number with hypo-pit will present in the first year of life. Despite advances in care the long-term neurological outcome for patients with HI is poor in 20-40% cases.Aim: To retrospectively evaluate the care given during the NBP in a series of patients diagnosed with late presenting pote...

hrp0084p3-933 | GH & IGF | ESPE2015

Adult Height in Children Born Small for Gestational Age and Treated with GH: Data from the French KIGS Database

Polak Michel , Czernichow Paul

Background: Treatment with GH in children born small for gestational age (SGA) increases height velocity (HV) but data on adult height (AH) are scarce.Objective and hypotheses: To report AH in a group of SGA children treated with GH.Method: This is a post-marketing longitudinal analysis of SGA children treated with GH and included in France in KIGS. Selection criteria were children followed at least one year and having reached AH b...