hrp0086p1-p907 | Thyroid P1 | ESPE2016

Dysregulation of the Immune System in Children with Graves Disease – the Role of NK and NKT-Like Cells

Klatka Maria , Polak Agnieszka , Grywalska Ewelina , Kollataj Witold , Rolinski Jacek

Background: Almost all cases of hyperthyroidism in children result from Graves’ disease (GD). Recent studies have confirmed a significant role of T cells in the development of autoimmune diseases. However, the interactions between NKT-like cells and NK cells in GD are still poorly understood.Objective and hypotheses: The aim of the study was to assess the frequencies of peripheral blood T, NK and NKT-like cells in children with GD.<p class="abst...

hrp0082p1-d3-161 | Growth (2) | ESPE2014

Safety and Effectiveness of Increlex® Therapy in Children Enrolled in the Increlex® Growth Forum Database in Europe: 4 Years Interim Results

Polak Michel , Woelfle Joachim , Bang Peter , Sicsic Aude , Fiorentino Bruno

Background: The post-authorization registry, European Increlex® (Mecasermin (rDNA Origin) injection) growth forum database (EU-IGFD) was initiated in Dec 2008 to collect data on children with growth failure treated with Increlex®.Objective: To report 4-year safety and effectiveness data.Methods: Multicenter, open-label observational study, eCRF data collection.Results: As of 30...

hrp0084p2-420 | GH &amp; IGF | ESPE2015

Hypoglycaemic Adverse Events Reported in Children Enrolled in the European Increlex® Growth Forum Database in Europe (5-Year Interim Data)

Woelfle Joachim , Polak Michel , Bang Peter , Houchard Aude , Sert Caroline

Background: The post-authorisation registry, European Increlex® (mecasermin (rDNA origin) injection) Growth European Increlex Growth Forum Database (EU-IGFD), initiated in December 2008, collects safety and efficacy data in children receiving Increlex® for growth failure. Hypoglycaemia has been reported as a common adverse event (AE) during any IGF1 replacement therapy in randomised clinical trials, and is therefore of interest in real-life sett...

hrp0084p3-995 | Gonads | ESPE2015

Puberty and Gonadal Function in Adolescents Girls after Renal Transplantation

Hecquet Blandine , Charbit Marina , Polak Michel , Bidet Maud

Background: Renal transplantation (RTx) is the most common solid organ transplant procedure. Several studies have reported on puberty and gonadal function in female RTx recipients with controversial results.Objective: We sought to describe puberty and gonadal function in adolescents after RTx before 16 years.Methods: We reported retrospectively the clinical signs of puberty, growth, medication and graft function of 20 girls aged 19...

hrp0094p2-186 | Fat, metabolism and obesity | ESPE2021

Diagnostic accuracy of Tri-Ponderal mass index (kg/m3) for identifying glucose intolerance in obese children and adolescents.

Arciniegas Larry , Mogas Eduard , Tomasini Rosangela , Fabregas Ana , Clemente Maria , Yeste Diego ,

Introduction: The identification of obese patients with increased susceptibility and risk for glucose intolerance and type 2 diabetes requires an oral glucose tolerance test (OGTT). Reference values for Body mass index (BMI) and Tri-Ponderal mass index (TMI) according to age and sex of healthy children in Spain without malnutrition or obesity have recently been published (*). TMI values remain very uniform in both boys and girls from the age of 8 to 18 years, ...

hrp0095p1-313 | Growth and Syndromes | ESPE2022

Pathway to assess severe primary IGF-1 deficiency diagnosis in a real-life setting: data from the Global Increlex® Registry

Bang Peter , Polak Michel , Bossowski Artur , De Schepper Jean , Sert Caroline , Perrot Valérie , Woelfle Joachim

Background: Severe primary insulin-like growth factor-1 deficiency (SPIGFD) is a rare condition for which replacement therapy with recombinant human insulin-like growth factor-1 (rhIGF 1; mecasermin [Increlex®]) is approved for treatment in Europe and the USA. SPIGFD is defined as a height standard deviation score (HtSDS) ≤-3, and baseline IGF-1 <2.5th percentile (European indication) or ≤-3 SDS (USA indication) for age and gender, desp...

hrp0092p1-212 | GH and IGFs (1) | ESPE2019

Hypoglycaemia Adverse Events in SPIGFD: Association with Patient Diagnosis, Age, Time-Course and Dosage of Mecasermin: 10-year Data from the European Increlex® Growth Forum Database in Europe (EU-IGFD)

Woelfle Joachim , Polak Michel , Bang Peter , Perrot Valérie , Sert Caroline

Background: In Europe, Increlex® (mecasermin) is approved for treatment of growth failure in children with severe primary insulin-like growth factor-1 deficiency (SPIGFD). We present 10-year data (up to October 2018) from the European Increlex® Growth Forum Database (EU-IGFD) registry (NCT00903110) on the frequency, predictive factors, and the potential impact of hypoglycaemia on efficacy outcomes....

hrp0092p1-214 | GH and IGFs (1) | ESPE2019

The European Increlex® Growth Forum Database (EU-IGFD) Registry: Do Treatment Practices Differ Between European Countries?

Bang Peter , Polak Michel , Woelfle Joachim , Perrot Valérie , Sert Caroline

Background: In the European Union, Increlex® (mecasermin) is approved for the treatment of growth failure in children with severe primary insulin-like growth factor-1 deficiency (SPIGFD).Methods: The European Increlex® Growth Forum Database (EU-IGFD) registry (NCT00903110) is an ongoing, multicentre, open-label, observational study monitoring the safety and efficacy of mecasermin in childr...

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...

hrp0089fc3.1 | Diabetes and Insulin 1 | ESPE2018

Neonatal Diabetes Owned to Potassium Channel Mutation: Response to Sulfonylureas According to the Genotype

Garcin Laure , Fauret Anne-Laure , Cave Helene , Polak Michel , Beltrand Jacques

Introduction / aim: Neonatal diabetes owned to potassium channel mutation can be successfully treated by sulfonylureas (SU). No study has reported SU efficiency according to the genotype.Method: Review of literature conducted in accordance with the control criteria of Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA). Search engine used: PubMed and the Cochrane Library database. Selection of clinical report, case reviews and met...