hrp0094fc4.2 | Diabetes | ESPE2021

A Randomised Controlled Cross-Over Trial of the Hybrid Closed-Loop Insulin Delivery System Diabeloop vs. Sensor-Augmented Pump Therapy in Children Aged 6-12 Years

Kariyawasam Dulanjalee , Morin Carole , Casteels Kristina , Tallec Claire Le , Sfez Annie , Godot Cecile , Huneker Erik , Garrec Nathalie , Polak Michel , Charpentier Guillaume , Franc Sylvia , Beltrand Jacques ,

Introduction: HbA1C goals are rarely achieved in children with Type 1 diabetes (T1D) except at the cost of increased hypoglycaemia episodes. The Diabeloop Hybrid Closed-Loop system improves time in range and glycemic control in adults. However, the efficacy and safety of the system had not yet been evaluated in children. Our objective was to evaluate the safety and efficiency of the Diabeloop hybrid closed-loop system in prepubescent children (DBL4K).<p cl...

hrp0086fc5.3 | Management of Disorders of Insulin Secretion | ESPE2016

Glibentek, a New Suspension of Glibenclamide for Patients with Neonatal Diabetes, is as Effective and more Convenient than Crushed Tablets

Beltrand Jacques , Godot Cecile , Busiah Kanetee , Djerada Zoubir , Baron Sabine , Tallec Claire Le , Tessier Raphael , Ribault Virginie , Cartigny Maryse , Bruel Henri , Gozalo Claire , Treluyer Jean-Marc , Elie Caroline , Polak Michel

Background: Glibenclamide has proven to be efficient for patients with neonatal diabetes owing to potassium channel mutations. Anyway its pharmaceutical form is not suitable for young children or infants. The tablets dosage is too high for most infants and must be crushed and diluted before administration. We developed a suspension of glibenclamide (EMA Orphean drug designation january 2016) fitting recommendations of drug administration to allow a precise dosage and designed ...

hrp0094p2-128 | Diabetes and insulin | ESPE2021

Daily Meal Size Variation Does Not Affect Glycemic Control In T1D Adolescent Patients Equipped With The Closed Loop DBLG1 System

Gimenez Paul , Lachal Sylvain , Tourki Yousra , Franc Sylvia , Charpentier Guillaume , Beltrand Jacques , Le Tallec Claire , Benhamou Pierre-Yves ,

The aim of this study was to assess the impact of meal size on glycemic control for T1D adolescent patients equipped with Diabeloop’s Closed Loop, DBLG1 System[1], based on data from the clinical trial NCT04190277. Among this dataset we isolated 37 adolescents. Only days with ≥70% available CGM data and >50% of time in closed-loop were included in the analysis, resulting in an average duration of 18 days per patient for a total of 668 days of treatment. To asses...

hrp0082fc9.5 | Beta cells | ESPE2014

Transient Neonatal Diabetes in Adulthood: Metabolic and Neurodevelopmental Outcomes

Le Bourgeois Fleur , Busiah Kanetee , Abu-Amara-Olivieri Sawsan , Bachere Nadege , Bertrand Anne-Marie , Bourron Olivier , David Paul , De Boisvilliers Fabienne , Deumier Bernard , deVries Liat , Jelliman Stephanie , Le Tallec Claire , Martin-Dessila Amelie , Nimri Paul , Paoli Anne , Perrin Mireille , Stuckens Chantal , Ythier Hubert , Pouvreaux Nathalie , Bellane-Chantelot Christine

Background: Transient neonatal diabetes mellitus (TNDM) is a rare genetic β-cell dysfunction leading to hyperglycaemia that resolves in early childhood. About 80% of patients relapse during adolescence or adulthood. Some of these patients suffer from neurodevelopmental defect. Long-term outcome has been poorly investigated.Objective and Hypotheses: To investigate metabolic and neurologic outcomes in adults affected with TNDM.M...

hrp0086p2-p422 | Gonads &amp; DSD P2 | ESPE2016

Late Clinical Presentation, Biological Assessment and Management of PAIS in a Developing Country

Brindusa Gorduza Daniela , Tambo Mouafo Faustin , Gay Claire-Lise , Plotton Ingrid , Birraux Jacques , Dahoun Sophie , Morel Yves , Mouriquand Pierre , Le Coultre Claude , Mure Pierre-Yves

Background: Partial androgen insensitivity syndromes (PAIS) are rare 46,XY DSD (disorder of sex development).Objective and hypotheses: Three families with PAIS (six patients) are reported, focusing on their phenotype and treatment depending on sex of rearing. Biological investigations and surgical management are described.Method: Between 2009 and 2015 a consultation for uro-genital malformations in pediatric patients was set up in ...

hrp0092rfc15.1 | Late Breaking Abstracts | ESPE2019

Preclinical Studies of Acrodysostosis Gene AAV Therapy in a Knock-In R368X PRKAR1A Mouse Model

Le Stunff Catherine , Gunes Yasemin , Mille Clémence , Bougnères Pierre

The use of recombinant adeno-associated viruses (rAAV) as safe vectors have allowed hundreds of gene therapy attempts to treat monogenic diseases not including bone genetic diseases (Gao G, Nat Rev Drug Dis 2019). To our knowledge, there has been few attempts to apply gene therapy to monogenic bone diseases, largely because most skeletal malformations are being developed during fetal life. Patients affected with acrodysostosis are known to aggravate their skeletal malformation...

hrp0084s7.2 | Hypogonadotropic hypogonadism: diagnostic and therapeutic approach | ESPE2015

Early Therapeutic Approach to the Male Patient with HH

Bouvattier Claire

In male fetuses, during the first 4–6 months of life, the ‘mini-puberty’ represents a period of intense hormonal activity of the hypothalamic–pituitary axis, reflected physically by an increase in testicular volume due to seminiferous tubule elongation and by an increase in penis length. During this period, pituitary LH and FSH levels rise, leading to an increase in circulating levels of testosterone, inhibin B and AMH. Concomitantly, Sertoli cells prolifer...

hrp0084p3-1011 | Growth | ESPE2015

Targeted Birth Length and Parental Height Measurement in Babies with Birthweight≤9th Centile; Improved Uptake During Second Study During 1 Calendar Year in a Single Newborn Unit

Sardar Colette Montgomery , Donnelly Sharon , Siddique Jamila , Gault Emma Jane , Kinmond Sheena , Donaldson Malcolm

Background: The contribution of intrauterine growth restriction (IUGR) and parental height (PH) to childhood short stature is difficult to determine in countries, including the UK, where birth length (BL) is not routinely measured, while accurate PH may become unavailable due to separation/divorce. A previous study (2008–2009) examined the feasibility of BL and PH measurement in the lightest 9% of babies born in a single maternity unit. Uptake was disappointing, with part...

hrp0086p2-p703 | Endocrinology and Multisystemic Diseases P2 | ESPE2016

French National Healthcare Network for Rare Endocrine Diseases (FIRENDO): The First Year of Activity to Monitor Patients with Rare Endocrine Diseases

Givony Maria , Minime Fanny , Lopes Euma Fortes , Varillon Yvonne , Le Verger Delphine , Ghenim Sabine , Provost Marion , Rahabi-Layachi Haifa , Bouvattier Claire , Polak Michel , Brue Thierry , Nunes Marie-Laure , Delemer Brigitte , Netchine Irene , Mouriquand Pierre , Borson-Chazot Francoise , Bony-Trifunovic Helene , Rodien Patrice , Juliane Leger , Bertherat Jerome

Background: Twenty-three national healthcare networks for rare diseases were identified in 2014 as part of the French scheme on rare diseases. The rare endocrine disease national healthcare network FIRENDO (www.firendo.fr) includes six centers of reference with complementary fields of expertise certified between 2005 and 2006, 30 centers of competence covering all French regions, 18 research and 37 diagnostic laboratories, 5 national learned...

hrp0094p2-6 | Adrenals and HPA Axis | ESPE2021

Single centre experience of Hydrocortisone Granules (Alkindi) in children under 6 years of age with Adrenal Insufficiency

Snow Claire , Owen Catherine ,

Introduction: Children with Adrenal insufficiency (AI) are usually managed with glucocorticoid replacement as hydrocortisone. Treatment options in younger patients include administering an aliquot of 10mg hydrocortisone tablets dissolved in water. However, this is time consuming and may be inaccurate. We describe our experience of using Alkindi (www.diurnal.co.uk), the first hydrocortisone preparation licensed for children with AI.<stron...