hrp0082fc2.6 | Bone & Mineral | ESPE2014

Efficacy and Safety Following 4 Monthly s.c. Doses of a Human Anti-Fibroblast Growth Factor 23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia

Peacock Munro , Imel Erik , Zhang Xiaoping , Ruppe Mary , Weber Thomas , Klausner Mark , Ito Takahiro , Vergeire Maria , Humphrey Jeffrey , Glorieux Francis , Portale Anthony , Insogna Karl , Carpenter Thomas

Background: In X-linked Hypophosphatemia (XLH), abnormally elevated serum Fibroblast Growth Factor 23 (FGF23) results in low renal maximum threshold for phosphate reabsorption (TmP/GFR), low serum phosphorus (Pi), inappropriately normal 1,25-dihydroxyvitamin D (1,25(OH)2D) and development of rachitic deformities.Methods: Up to four SC KRN23 doses were given every 28 days to 28 adults with XLH (26 completed) according to a dose-escalation algorithm (0.05&...

hrp0082p1-d2-35 | Bone | ESPE2014

Assessment of Quality of Life Data After 4 Monthly S.C. Doses of a Human Monoclonal Anti-Fibroblast Growth Factor 23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia

Ruppe Mary , Zhang Xiaoping , Imel Erik , Weber Thomas , Klausner Mark , Ito Takahiro , Vergeire Maria , Humphrey Jeffrey , Glorieux Francis , Portale Anthony , Insogna Karl , Peacock Munro , Carpenter Thomas

Objectives: In X-linked hypophosphatemia (XLH), abnormally elevated serum Fibroblast growth Factor 23 (FGF23) results in low renal maximum threshold for phosphate reabsorption, low serum phosphorus, inappropriately normal 1,25-dihydroxyvitamin D, and development of rachitic deformities. The effect of KRN23 on health-related quality of life (HRQL) was assessed.Methods: Open-label KRN23 was given s.c. every 28 days up to four doses to 28 adults with XLH (2...

hrp0086fc2.6 | Bone & Mineral Metabolism | ESPE2016

Effect of KRN23, a Fully Human Anti-FGF23 Monoclonal Antibody, on Rickets in Children with X-linked Hypophosphatemia (XLH): 40-week Interim Results from a Randomized, Open-label Phase 2 Study

Linglart Agnes , Carpenter Thomas , Imel Erik , Boot Annemieke , Hogler Wolfgang , Padidela Raja , van't Hoff William , Whyte Michael , Chen Chao-Yin , Skrinar Alison , Agarwal Sunil , Martin Javier San , Portale Anthony

Background: In XLH, high circulating FGF23 causes hypophosphatemia, rickets, and short stature.Objective and hypotheses: To evaluate KRN23 effects on serum phosphate (Pi) level and rickets severity in XLH children in a Phase 2 study.Method: 52 XLH children (ages 5–12 years, ≤Tanner 2) received KRN23 subcutaneously biweekly (Q2W) or monthly (Q4W). Serum Pi was measured at 2-week intervals. KRN23 dose was titrated (maximum...

hrp0082p1-d1-65 | Diabetes | ESPE2014

Glycaemic Control and Acute Complications in European Children, Adolescents, and Young Adults With Type 1 Diabetes in the Teens Study

Phillip Moshe , Laffel Lori , Domenger Catherine , Dain Marie-Paule , Pilorget Valerie , Candelas Christophe , Danne Thomas , Mazza Carmen , Anderson Barbara , Hanas Ragnar , Waldron Sheridan , Beck Roy , Mathieu Chantal

Aims: The TEENs study is an international, cross-sectional observational study aiming to assess type 1 diabetes (T1D) management and psychosocial parameters in children, adolescents, and young adults, to identify approaches to optimise glycaemic control and outcomes. Results from 11 European countries are presented.Methods: 111 centres providing diabetes care to young T1D patients collected data by participant interview, medical record review and partici...

hrp0084p1-138 | Turner & Puberty | ESPE2015

Weight Gain in Turner Syndrome: Association to Puberty Induction?

Reinehr Thomas , Lindberg Anders , Toschke Christina , Cara Jose , Chrysis Dionisios , Camacho-Hubner Cecila

Background: We have recently reported a BMI-SDS increase in girls with Turner syndrome (TS) treated with growth hormone (GH) (1).Objective and hypothesis: We hypothesise that puberty induction in TS is associated with weight gain.Method: We analysed the weight changes (BMI-SDS) of 888 girls with TS in the Pfizer International Growth Database (KIGS). Overweight was defined by a BMI >90th percentile and obesity by a BMI >97th...

hrp0097p1-278 | Fetal, Neonatal Endocrinology and Metabolism | ESPE2023

Exploration of O-link protein biomarkers in children born after IUGR and early impaired developmental changes in heart function

Änghagen Olov , Rudholm Feldreich Tobias , Ärnlöv Johan , Bang Peter

We recently assessed systolic heart function in children from IUGR and normal control pregnancies and reported early developmental impairment of left ventricular longitudinal strain – a sensitive echocardiographic measure – during the first 3 months of life in IUGR children. In accordance with previous studies, this suggest that the increased cardiovascular risk later in life imposed by IUGR/SGA may, at least to some extent, be primary and not entirely secondary to...

hrp0086s3.2 | Optimizing nocturnal diabetes control | ESPE2016

Optimising Nocturnal Glucose Control in Children with T1D: Therapeutic Implications

Danne Thomas

Background: Nocturnal hypoglycemia is not regularly predictable on the basis of a bedtime BG level and can only be confirmed by BG tests at regular intervals during the night or continuous glucose monitoring (CGM).Objective and hypotheses: A bedtime snack containing carbohydrate as well as fat and protein may be useful in preventing nocturnal hypoglycemia, but this should not be at the expense of high overnight BG levels. In many individuals, a lowering ...

hrp0086wg5.2 | ESPE Diabetes Technology and Therapeutics Working Group | ESPE2016

SWEET: Developing Centers of Excellence (CoR)

Danne Thomas

Background: SWEET (www.sweet-project.eu) is a non-profit entity endorsed by ISPAD aiming to create an extensive network of certified CoRs for childhood diabetes in order to ensure high quality care.Objective and hypotheses: SWEET aims at an improved and more uniform care for people with diabetes through comparing processes and outcomes among participating members. The results of data analysis are conveyed to memb...

hrp0082wg4.2 | Obesity | ESPE2014

Natural Course of Impaired Glucose Tolerance in Obese Children

Reinehr Thomas

Obesity in childhood is associated to several cardiovascular risk factors summarized in the definition of metabolic syndrome such as hypertension, dyslipidemia and impaired glucose tolerance. Besides others, the pathogenetic background is insulin resistance, which deteriorates in mid puberty and normalizes at end of puberty. Accordingly, blood pressure, lipids, fasting glucose and 2 h glucose in oGTT increased from prepubertal stage to pubertal stage and decreased from puberta...

hrp0084wg2.6 | Diabetes Technology and Therapeutics Thursday, 1 October | ESPE2015

Type 2 Diabetes Mellitus in Adolescence

Reinehr Thomas

Type 2 diabetes mellitus (T2DM) is emerging as a new clinical problem within pediatric practice. Recent reports indicate an increasing prevalence of T2DM in children and adolescents around the world in all ethnicities, even if the prevalence of obesity is not increasing any more. There are great differences in T2DM prevalence worldwide. The majority of young people diagnosed with T2DM was found in specific ethnic subgroups such as African-American, Hispanic, Asian/Pacific Isla...