ESPE Abstracts

Background: Resistance to thyroid hormones (RTH) is a rare disease, characterized by reduced sensitivity of target tissues to thyroid hormones. Pathognomonically, thyroid hormones are moderately elevated, whereas TSH levels are inappropriately normal or elevated. Over 100 thyroid hormone receptor beta (THRB) mutations have been reported, which are found in around 85% of RTH cases. Hypercholesterolemia was not previously reported in RTH patients....

Background: PATRO children is an international, open, longitudinal, noninterventional study of the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).Objective and hypotheses: The primary objective is to assess the long-term safety of Omnitrope®, particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies, and potential risks of...

Background: PATRO Children is an international, open, longitudinal, non-interventional study designed to evaluate the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).Objective and hypotheses: Long-term safety of Omnitrope® is the primary objective of PATRO Children (particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies,...

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...

Background: Decreasing age at the onset of puberty is observed worldwide, several countries report also increasing incidence trends of central precocious puberty (CPP), and some observed an importantly higher number of CPP cases during the COVID19 pandemic. Scarce data report also rising trends in isolated precocious adrenarche (IPA) and isolated precocious telarche (IPT).Objectives: To determine the national incidence a...

Background: Central precocious puberty (CPP) is a premature activation of the hypothalamic-pituitary-gonadal axis. One of its negative effects is lower final adult height (FAH) due to premature epiphyseal closure. GnRH analogues act by desensitizing GnRH receptors and stopping the progression of puberty and prolonging growth period.Objectives: We aimed to evaluate the effect of the GnRH analogue on FAH and identify facto...

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...

Introduction: Recently novel approaches, through implementation of next-generation sequencing (NGS) in clinical practice for genetic evaluation of idiopathic short stature, has permitted to identify new variants of genes which modulate function of growth plate, including heterozygous mutations of the aggrecan gene. Aggrecan, a large chondroitin sulfated proteoglycan, is a major structural component of the extracellular matrix of cartilage, including growth pla...

Objectives: PATRO Children is an observational, international, longitudinal study of the long-term safety of a recombinant human growth hormone (rhGH; Omnitrope®, Sandoz). In particular, the study will assess the impact of rhGH on glucose metabolism and risk of malignancies. Long-term effectiveness is a secondary objective.Methods: The study population includes infants, children and adolescents receiving ...

Background: GH administration restores normal growth in children with GH deficiency (GHD). However, current daily s.c. injection treatment regimens may be inconvenient leading to impaired adherence and subsequently suboptimal treatment outcomes. NNC0195-0092 is a novel, reversible albumin-binding GH developed for once-weekly administration.Objective and hypotheses: This was a randomised, open-label, active-controlled, dose-escalation trial (NCT01973244) ...