hrp0084p2-461 | Growth | ESPE2015

French Growth Reference Charts should be Updated

Stoupa Athanasia , Goischke Alexandra , Garcin Camille , Elie Caroline , Viaud Magali , Thery Anne , Richard Genevieve , Polak Michel

Background: Growth charts constitute an important tool to monitor a child’s growth and development, and thus detect growth anomalies. Growth assessment allows early referral and management of treatable disorders. In France, the currently used growth reference charts were derived at the end of 1970s, based on children born on 1950s in Paris area. Questions are raised about their use for growth monitoring of more recently born children.Objectives: To ...

hrp0084p2-479 | Growth | ESPE2015

Moya Moya Syndrome in a Patient with Growth Hormone Deficiency and Hypergonadotropic Hypogonadism: to Treat or not to Treat with Growth Hormone Therapy?

Briceno Laura Gabriela Gonzalez , Stoupa Athanasia , Pinto Graziella , Touraine Philippe , Polak Michel

Background: Moya Moya disease is a chronic cerebrovascular angiopathy characterized by progressive stenosis of terminal part of internal carotid vessels and the compensatory development of collateral vessels. We present here the case of a young boy with growth hormone deficiency (GHD) and testicular insufficiency that was diagnosed with Moya Moya syndrome.Case presentation: A 12.9 year-old boy of Serbian origin was referred to the Endocrinology Departmen...

hrp0084p3-1107 | Pituitary | ESPE2015

Between 3 to 4 Years after Severe Traumatism Brain Injury 22% at Least of Children and Adolescents do have Persistent Pituitary Dysfunction

Dassa Yamina , Personnier Claire , Crosnier Helene , Chevignard Mathilde , Bourgeois Marie , Viaud Magali , Polak Michel

Background: Traumatic brain injury (TBI) is common in childhood but long-term endocrine consequences are yet to be documented by prospective data.Objective and hypotheses: We have previously demonstrated in prospective study that, 1 year after severe accidental TBI (ATBI) or inflicted TBI (ITBI), children and adolescents may present pituitary and growth hormone (GH) dysfunction. We present here the follow-up of this population to determine whether or not...

hrp0094p1-169 | Growth B | ESPE2021

Safety of growth hormone and cardiovascular outcomes in patients with Noonan syndrome enrolled in NordiNet® International Outcome Study (IOS) and the ANSWER Program

Romano Alicia , Kaski Juan Pablo , Dahlgren Jovanna , Kelepouris Nicky , Pietropoli Alberto , Rohrer Tilman R. , Polak Michel ,

Introduction: Growth hormone (GH) treatment has been shown to increase height velocity and adult height in patients with Noonan syndrome (NS). NS is also associated with cardiovascular (CV) anomalies, namely pulmonary stenosis and hypertrophic cardiomyopathy. Concerns persist about the role of GH in progression of CV conditions despite data, albeit limited, showing low rates of CV events and left ventricular wall thickness remaining normal. This pooled analysi...

hrp0094p2-283 | Growth and syndromes (to include Turner syndrome) | ESPE2021

Assessment of genetic defects, baseline characteristics and adverse events reported in the Increlex® registry

Polak Michel , Bang Peter , Perrot Valerie , Sert Caroline , Storr Helen L. , Woelfle Joachim ,

Background: Certain genetic defects in the growth hormone (GH)/insulin-like growth factor 1 (IGF-1) axis are associated with severe primary IGF-1 deficiency (SPIGFD) and short stature. Detection of genetic defects may confirm short stature aetiology alongside clinical/biochemical features. Increlex® is a recombinant human IGF-1 (rhIGF-1) approved for children/adolescents with SPIGFD.Methods: The Increlex<...

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...

hrp0092p3-238 | Sex Differentiation, Gonads and Gynaecology or Sex Endocrinology | ESPE2019

Bilateral Testicular Atrophy and Normal Inhibin B level: A Paradoxal Clinical Finding For A Rare Biochemical Cause !

ESSADDAM Leïla , PIKETTY Marie , KALLALI Wafa , GUEDRI Rahma , GONZALEZ Laura , MATTOUSSI Nadia , POLAK Michel , BEN BECHER Saayda

Background: Testicular atrophy is a rare complication following inguinal hernia repair particularly in children<2 years and those with an undescended testis at highest risk> with an undescended testis. Inhibin B is secreted from the testis as a product of Sertoli cells, and has been suggested as a good marker for spermatogenesis. Its value is expected to be very low in children with bilateral testicular atrophyCase Report...

hrp0089rfc12.6 | Diabetes and Insulin 2 | ESPE2018

AMGLIDIA, a Suspension of Glibenclamide for Patients with Neonatal Diabetes, Long Term Data on Efficiency and Tolerance

Beltrand Jacques , Meyzer Candice , Colas Sandra , Semeraro Michaela , Godot Cecile , Treluyer Jean-Marc , Elie Caroline , Polak Michel

Background: Glibenclamide has proven to be efficient for patients with neonatal diabetes owing to potassium channel mutations. We developed a suspension of glibenclamide (EMA CHMP Authorization February 2018) fitting recommendations of drug administration to allow a precise dosage. We reported it to be practical, efficient and well tolerated after 3 months of use.Objective: To determine long term efficiency and tolerance of a new suspension of glibenclam...

hrp0089p2-p099 | Diabetes &amp; Insulin P2 | ESPE2018

Metabolic Improvement Offered by Medtronic Minimed 640 G Associated to Transient Insulin Perfusion Suspension before Hypoglycemia in Young Patients with Type 1 Diabetes

Al Hage Chehade Ghada , Godot Cecile , Jourdon Isabelle , Lepage Nadine , Eve Schmidt Marie , Polak Michel , Beltrand Jacques

Objective: Fear of hypoglycemia interferes frequently with metabolic control of type 1 diabetes especially in patients under 5 years of age who are at high risk of hypoglycemia and low metabolic control. Medtronic Minimed 640 G insulin pump with Smart Guard technology (suspension of insulin perfusion in predictive hypoglycemia situations) appears to be an adequate system for these patients by reducing the risk of hypoglycemia.Research design and methods:...

hrp0095rfc7.2 | Growth and Syndromes | ESPE2022

Once-Weekly Somapacitan vs Daily GH in Children with GH Deficiency: The Randomized Phase 3 REAL 4 Trial

Miller Bradley , Blair Joanne , Højby Michael , Böttcher Volker , Juul Kildemoes Rasmus , Maniatis Aristides , Beck Bang Rikke , Mori Jun , Polak Michel , Stagi Stefano , Horikawa Reiko

Background: Growth hormone (GH) replacement therapy usually requires daily subcutaneous (s.c.) injections that can be burdensome for patients and their caregivers. Long-acting GH formulations aim to establish a less burdensome dosing regimen that is as safe and efficacious as daily GH to potentially improve adherence and clinical outcomes. Somapacitan, a long-acting reversible albumin-binding GH derivative, is in development for once-weekly s.c. administration...