ESPE Abstracts

Introduction: Hypopituitarism is the partial or complete insufficiency of a single or multiple pituitary hormones. The clinical manifestation of hypopituitarism varies depending on the number and severity of hormone deficiencies. Familial cases are rare, compared to the sporadic ones. Its estimated incidence is between 1:4000-1:10000 live births.Case report: We report the clinical case of familial hypopituitarism in a fa...

Background: In diabetic patients vitamin D seems to play a role not only on bone metabolism, but also on many other organs and/or systems, such as lipid profile, cardiovascular system, etc. We decided to evaluate whether the celiac disease antibodies in type 1 diabetic patients have a connection with 25-OH vitamin D status.Materials and Methods: 78 young patients with type 1 diabetes were evaluated with mean age of 14&#1...

The objective of the study was to evaluate the Pediatric Quality of Life (PedsQL) of children from 8-12 y/o with T1DM, to compare PedsQL perceived by their parents, to understand gender and other factors influence on PedsQL and disease management.We conducted a prospective non-randomized cross-sectional study. Children with T1DM were identified from pediatric endocrinology department registries. The onset of diabetes had to be more than 6 months. All con...

Introduction: Neonatal diabetes is a rare condition that can present in the first months of life. Neonatal diabetes has more than 20 genetic origins that are currently known. About 40% of these patients carry mutations in KCNJ11 and ABCC8 genes, which impair the pancreatic beta-cell K-ATP channels and can be treated with oral sulfonylureas. The purpose of this case report is to present a patient diagnosed with neonatal diabetes and the subsequent management of...

Background: Growth hormone deficiency (GHD) is a disorder affecting children's linear growth and leading to short stature without initiation of treatment with growth hormone (GH). Administration of GH has been shown to be safe and effective to increase children's final height in GHD. It is important to start the treatment as early as the GHD is diagnosed. The objective of the current study is to evaluate the growth velocity and height standard deviat...

Introduction: Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder associated with congenital errors of steroid biosynthesis in the adrenal glands. The prevalence of CAH is estimated to be 1:10.000-20.000 for classic and 1:200-1.000 for non-classic types in Europe. In the Caucasus & Central Asia (C&CA), the prevalence is unknown. A delayed diagnosis is associated with an increased risk of neonatal morbidity/mortality, whereas early d...

Background: Hypopituitarism is a rare condition characterized by the insufficiency of 2 and more hormones produced by anterior pituitary gland. The major causes of hypopituitarism are brain tumors located near or in the pituitary gland and/or hypothalamus, cranial radiation, chemo- or surgical therapy, cranial traumas, neuroinfections, autoimmune hypophysitis (immune-mediated inflammation of pituitary gland) etc. Brain tumors are the second most frequent type ...

Introduction: Lonapegsomatropin (SKYTROFA, TransCon hGH), a prodrug of somatropin administered once-weekly, is approved for the treatment of pediatric growth hormone deficiency (GHD) by the Food and Drug Administration and European Commission. In the pivotal 52-week phase 3 heiGHt trial and 26-week fliGHt trial (enrolled treatment-naïve and treatment-experienced participants, respectively), lonapegsomatropin demonstrated safety and efficacy in children wi...

Background: TransCon hGH is a sustained-release prodrug in development as a long-acting GH for children with growth hormone deficiency (GHD). TransCon hGH consists of a parent drug, growth hormone (hGH; somatropin), that is transiently bound to a carrier via a TransCon linker. The carrier extends hGH circulation time in the body and fully active hGH is released over one week at physiologic pH and temperature. Unlike other molecules in development, TransCon hGH...

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...