hrp0097p2-115 | GH and IGFs | ESPE2023

Are there sex differences among patients to whom a growth hormone (GH) test with clonidina stimulus was required during the study of short stature?

Vela Amaya , GarcÍA Maria , Izquierdo Jon , Ugeso Alberto , Grau Gema , Rica Itxaso

Introduction: GH stimulus test is usually performed in pacientes With short stature and/or growth delayed study if GH deficit is suspected. Several articles concluded that male are more frequently diagnosed and treated of GH deficiency than femaleObjectives: To know gender's ratio and the possible anthropometric differences between patients that required a consult for short stature and to whom a stimulus test was pe...

hrp0086s4.3 | New avenues in paediatric autoimmune disease | ESPE2016

Type 1 Diabetes: Lessons from nPOD Pathology and Clinical Trials

Pugliese Alberto

Type 1 diabetes (T1D) is considered chronic autoimmune disease in which autoreactive T-cells and inflammation cause severe loss of pancreatic beta cells. Insulitis, the pathologic hallmark of T1D, is an inflammatory lesion consisting of immune cell infiltrates around and within the islets. New research initiatives and methodologies are advancing our understanding of pancreas pathology. A major impetus to the field has been given by the institution of the JDRF nPOD (Network for...

hrp0095p1-513 | Growth and Syndromes | ESPE2022

Long-term effectiveness and safety of childhood growth hormone treatment in Turner syndrome from two large observational studies

Backeljauw Philippe , Pietropoli Alberto , Rohrer Tilman

Patients with Turner syndrome (TS) have short stature, despite having normal growth hormone (GH) secretion. Treatment with recombinant human GH is recommended. The effectiveness and safety of Norditropin® (somatropin, Novo Nordisk) over ≤10 years of follow-up were investigated in two non-interventional studies: NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905). Of 2,409 children with TS, 2,377 were included in the full analysis s...

hrp0086p2-p306 | Diabetes P2 | ESPE2016

Maturity-Onset Diabetes of the Young (MODY): Tracking and Clinical Follow-up

Bezerra Arthur Pires , Ramos Alberto Jose Santos , Braz Adriana Farrant

Background: Maturity-onset diabetes of the young (MODY) is a monogenic form of diabetes, with an autosomal dominant mode of inheritance and high penetrance. To this date, it is known 13 subtypes of MODY with different genetic etiologies. It is characterized by high incidence in the family, an early onset and primary defect in pancreatic β-cell function.Objective and hypotheses: The primary objective of this study is to identify patients with MODY an...

hrp0094p2-280 | Growth and syndromes (to include Turner syndrome) | ESPE2021

Outcomes in growth hormone-treated Noonan syndrome children: impact of PTPN11 mutation status

AL Jorge Alexander , Pietropoli Alberto , Kelepouris Nicky , Horikawa Reiko ,

Objectives: Mutations in PTPN11 are known to be associated with Noonan syndrome (NS), accounting for approximately 50% of cases. Data from a non-interventional and phase 3 study of Norditropin (somatropin; Novo Nordisk A/S, Denmark) were used to assess the impact of PTPN11 mutation status on growth outcomes in children with NS receiving growth hormone therapy (GHT).Methods: The ANSWER (NCT01009905) prog...

hrp0095lb17 | Late Breaking | ESPE2022

Mapping of the current transition of care practice for patients with pituitary disease at Endo-ERN reference centers

Shishkov Savi , Tuccillo Luigi , Iotova Violeta , Pivonello Rosario , Pelsma Iris , Pereira Alberto , Biermasz Nienke

Rare endocrine conditions present specific diagnostic and management challenges to healthcare providers, especially regarding transition. Despite the need for practical guidance on this subject, consensus on structured approaches to transition are lacking. The aim was to map the current clinical practice, and identify the unmet needs regarding transition of care for patients with pituitary disease in the reference centers (RCs) of the European Reference Network on Rare Endocri...

hrp0095p2-99 | Fat, Metabolism and Obesity | ESPE2022

Gender differences in Italian pediatric obese subjects

Inzaghi Elena , Tarissi Isabella , Deodati Annalisa , Fintini Danilo , Villani Alberto , Cianfarani Stefano

Background: Significant aspects of metabolic homeostasis are regulated differently in males and females and sex differences can influence diagnostic approach and therapeutic responses. The underlying mechanisms accounting for gender difference remain to be established and may involve genetic and hormonal factors.Aim: This study aimed at evaluating gender differences in anthropometric and metabolic parameters in a large c...

hrp0092p1-226 | Growth and Syndromes (to include Turner Syndrome) (1) | ESPE2019

Response to Growth Hormone in Very Young Children (® International Outcome Study and ANSWER Program

Rohrer Tilman R , Miller Bradley , Ostrow Vlady , Pietropoli Alberto , Polak Michel , Ross Judith

Objectives: Limited information is available on how very young children with growth hormone deficiency (GHD) respond to growth hormone (GH) replacement. We compared response to 1 year of GH therapy in children aged <2 years and prepubertal children aged ≥2 years.Methods: The two non-interventional, multicentre studies, NordiNet® International Outcome Study (IOS) (NCT00960128) and the ANSWER Prog...

hrp0089p2-p252 | Growth &amp; Syndromes P2 | ESPE2018

Final Results of NordiNet® International Outcome Study: Key Outcomes in Paediatric Patients

Polak Michel , Blair Jo , Rohrer Tilman R. , Pietropoli Alberto , Tonnes Pedersen Birgitte , Savendahl Lars

Background: NordiNet® International Outcome Study ([IOS]; NCT00960128), a non-interventional study (2006–2016), assessed the effectiveness and safety of real-world treatment with Norditropin®. Outcomes were assessed in children with growth hormone deficiency (GHD), born small for gestational age (SGA), Turner syndrome (TS), chronic renal disease (CRD), idiopathic short stature (ISS), Noonan syndrome (NS) and Prader-Willi syndrome (PWS)....

hrp0086p1-p899 | Thyroid P1 | ESPE2016

Clinical and Histopathologic Features and Follow-up of Paediatric Patients with Papillary Thyroid Cancer: A 10 Years Experience

de Jesus Zuart Ruiz Roberto , Serrano Bello Carlos Alberto , Sauza Jorge Cortes , Bravo Patricia Medina

Background: The incidence of paediatric papillary thyroid cancer (PTC) is increasing.Objective and hypotheses: To describe the clinical and histopathologic features at diagnosis, and follow-up of paediatric patients with PTC at Children’s Hospital of Mexico in a 10 years period.Method: Comparative longitudinal study. We included 22 paediatric patients with histopathologic diagnosis of PTC between 2004–2014, divided into r...