ESPE Abstracts

Background: `(LAGHs) are under investigation to treat SGA. Results from the REAL5 phase 2 trial (randomised, multinational, open-label; NCT03878446) indicate that the LAGH somapacitan has similar efficacy, safety, and tolerability profile as daily GH1. Predicting GH therapy response is critical to improve clinical management of short stature. Here, we compare the prediction of growth response in SGA children treated with daily GH or somapacitan based on ...

Background: Oral LUM-201 normalizes growth rates in moderate PGHD over 12 months (Phase 2 Trials: OraGrowtH210/OraGrowtH212) [FC-746 ESPE 2024]. In OraGrowtH212, GH profiles, based on 10-minute sampling over 12 hours (8am-8pm), showed significant increases in inter-pulse, pulsatile and total GH secretion in response to 1.6 and 3.2 mg/kg/day doses of LUM-201 over 6 months, with no difference between the doses [FC14.3, ESPE 2023].O...

Introduction: Genetic investigation in patients with short stature allows diagnostic definition and impacts therapeutic decisions, clinical follow-up, and genetic counseling. That said, next-generation sequencing has created a new clinical challenge by allowing the identification of findings unrelated to the complaint that prompted testing. The ACMG published a list of actionable incidental findings that includes genes whose variants should be reported to pati...

Objectives: Achondroplasia (ACH) is characterized by disproportionate short stature and is associated with medical complications and functional constraints. Infigratinib is an oral FGFR3 inhibitor that targets overactive FGFR3 signaling at its origin and is under development as a treatment option for children with ACH. We report Month 18 results from PROPEL 2 (NCT04265651), a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluat...

Aims: To investigate the prevalence of diabetic polyneuropathy (DPN), cardiac autonomic neuropathy (CAN) and sudomotor dysfunction in children and adolescents with type 1 diabetes using bedside modalities. Secondly, to evaluate the co-existence of these types of diabetes neuropathies.Methods: Cross-sectional study including 221 children and adolescents with type 1 diabetes. DPN was assessed by vibration sensation thresho...

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...