ISSN 1662-4009 (online)

ey0016.4-5 | New Therapeutic Options | ESPEYB16

4.5. The beneficial effect of combined GH/GnRHa therapy in increasing adult height outcome in children with ISS

L Lazar , S Levy , T Oron , J Meyerovitch , L de Vries , S Shalitin , A Tenenbaum , M Phillip , Y. Lebenthal

To read the full abstract: J Clin Endocrinol Metab. 2019;104:3287-3295.The current definition of idiopathic short stature (ISS) refers to a heterogeneous group of short children, in the absence of any underlying detectable cause, including both normal variants of growth and pathological conditions. ISS subjects have been reported to reach an average final height of −1.5 SDS in boys...

ey0016.10-17 | (1) | ESPEYB16

10.17. Relative prognostic importance and optimal levels of risk factors for mortality and cardiovascular outcomes in type 1 diabetes mellitus

A Rawshani , A Rawshani , N Sattar , S Franzen , DK McGuire , B Eliasson , AM Svensson , B Zethelius , M Miftaraj , A Rosengren , S Gudbjornsdottir

To read the full abstract: Circulation. 2019;139:1900–1912Numerous publications have reported that higher HbA1c levels relate to higher cardiovascular disease (CVD) risk in people with type 1 diabetes (T1D). However, the strength of association and optimal HbA1c levels are not established.This analysis of T1D patients recorded in the Swedish National Diabetes ...

ey0015.2-10 | MEHMO syndrome is a novel X-linked retardation syndrome associated with neonatal hypoglycaemia, hypopituitarism and early onset diabetes mellitus | ESPEYB15

2.10 Two novel EIF2S3 mutations associated with syndromic intellectual disability with severe microcephaly, growth retardation, and epilepsy

S Moortgat , J Désir , V Benoit , S Boulanger , H Pendeville , MC Nassogne , D Lederer , I Maystadt

To read the full abstract: Am J Med Genet A. 2016 Nov;170(11):2927-2933The vast majority of proteins that a cell secretes or displays on its surface first enter the endoplasmic reticulum (ER), where they fold and assemble. Only properly assembled proteins advance from the ER to the cell surface. The ER coordinates protein biosynthetic and secretory activities in the cell. Alterations in ER homeost...

ey0015.3-10 | Graves’ disease | ESPEYB15

3.10 Long-term outcome of thyrotoxicosis in childhood and adolescence in the west of Scotland: the case for long-term antithyroid treatment and the importance of initial counselling

M Kourime , S McGowan , M Al Towati , SF Ahmed , G Stewart , S Williamson , I Hunter , MDC Donaldson

To read the full abstract: Arch Dis Child 2018;103:637-642Management of thyrotoxicosis in children and adolescents remains challenging and treatment varies considerably among institutions. The patient’s age, clinical status and likelihood of remission should be considered when counseling patients and parents. Nevertheless, individual prognosis of antithyroid drug treated Graves’ diseas...

ey0015.10-25 | Therapy and interventions | ESPEYB15

10.25 A phase 3 multicenter, open-label, prospective study designed to evaluate the effectiveness and ease of use of nasal glucagon in the treatment of moderate and severe hypoglycemia in children and adolescents with T1DM in the home or school setting

LC Deeb , H Dulude , CB Guzman , S Zhang , BJ Reiner , CA Piché , S Pradhan , XM Zhang

To read the full abstract: Pediatr Diabetes. 2018 19(5):1007-1013Severe hypoglycemia is one of the most threatening events in management of diabetes for parents and relatives. Although parents and patients are taught about the management in the acute situation, many are unable to use injectable glucagon. For moderate hypoglycemia, injectable glucagon seems to be no alternative for most caregivers. N...

ey0015.13-17 | Advances in the Diagnosis and Management of Congenital Hypothyroidism | ESPEYB15

13.17 Assessment of knowledge, attitudes and practices towards newborn screening for congenital hypothyroidism before and after a health education intervention in pregnant women in a hospital setting in Pakistan

B Tariq , A Ahmed , A Habib , A Turab , N Ali , SB Soofi , S Nooruddin , RJ Kumar , A Tariq , F Shaheen , S Ariff

To read the full abstract: Int Health 2018; 10(2):100-107This article addresses the important issue of acceptance of the congenital screening for congenital hypothyroidism (CH) by the population in general and the families in particular. While it is usually obvious for the health professional that screening for CH is beneficial to the potentially affected neonate, culture, level of education, ...

ey0015.15-13 | The importance of being earnest about diurnal variation | ESPEYB15

15.13 A Transcriptional Circuit Filters Oscillating Circadian Hormonal Inputs to Regulate Fat Cell Differentiation

Z Bahrami-Nejad , ML Zhao , S Tholen , D Hunerdosse , KE Tkach , S van Schie , M Chung , MN Teruel

To read the full abstract: Cell Metabolism 2018;27:854-868. e8People gain weight due to treatment with glucocorticoid (GC) drugs, but also in chronic stress and disrupted circadian rhythms. We now learn that fat-cell maturation increases if the trough in exposure to GC lasts less than 12 hours. A long pulse of glucocorticoids lasting 48 hours led most of the cells to differentiate under the ...

ey0020.10-6 | New Drugs for Children with T2DM | ESPEYB20

10.6. Deterioration of glycemic control in youth-onset type 2 diabetes: What are the early and late predictors?

P Zeitler , L El Ghormli , S Arslanian , S Caprio , E Isganaitis , MK Kelsey , RS Weinstock , NH White , K Drews

Brief summary: In this study of 699 youth 10 to < 18 years old with <2 years duration of type 2 diabetes (T2D) at 15 centers across the USA, baseline HbA1c level, and the change in HbA1c level in the first 6 months are predictors of rapid glycemic deterioration. In addition, subsequent loss of control can be predicted based on both baseline and ongoing clinical characteristics.Comment: The population of individuals with youth-onset T2D is heterog...

ey0020.7-7 | Gonadal Function and Fertility Issues in Childhood Cancer Survivors | ESPEYB20

7.7. The uterine volume is dramatically decreased after hematopoietic stem cell transplantation during childhood regardless of the conditioning regimen

B Courbiere , B Drikes , A Grob , Z Hamidou , P Saultier , Y Bertrand , V Gandemer , D Plantaz , G Plat , M Poiree , S Ducassou , C Pochon , JH Dalle , S Thouvenin , C Paillard , J Kanold , A Sirvent , C Rousset-Jablonski , S Duros , A Gueniffey , C Cohade , S Boukaidi , S Frantz , M Agopiantz , C Poirot , A Genod , O Pirrello , AS Gremeau , S Bringer-Deutsch , P Auquier , G Michel

Brief summary: This French multicenter prospective study analyzed uterine volume by pelvic MRI in 88 women (age range 18–40 years), who were survivors of childhood acute leukemia treated with hematopoietic stem cell transplantation (HSCT). They were compared to 88 healthy women matched for age and parity.Conditioning regimens before HSCT included alkylating agents for 34 women and total body irradiation (TBI) for 54 women. Scans were centralized and...

ey0018.10-14 | (1) | ESPEYB18

10.14. Targeted pharmacological therapy restores [beta]-cell function for diabetes remission

S Sachs , A Bastidas-Ponce , S Tritschler , M Bakhti , A Bottcher , MA Sanchez-Garrido , M Tarquis-Medina , M Kleinert , K Fischer , S Jall , A Harger , E Bader , S Roscioni , S Ussar , A Feuchtinger , B Yesildag , A Neelakandhan , CB Jensen , M Cornu , B Yang , B Finan , RD Di Marchi , MH Tschop , FJ Theis , SM Hofmann , TD Muller , H Lickert

Nature Metabolism. 2020;2(2):192–209. doi: 10.1038/s42255-020-0171-3.This experimental mouse study examined whether or not dedifferentiated β cells could be reversed or targeted by pharmacological intervention for diabetes remission. They identified evidence for β-cell dedifferentiation and dysfunction which could be reversed by single and combined pharmacological approach...