ISSN 1662-4009 (online)

ey0015.8-12 | Clinical Trials – New Treatments | ESPEYB15

8.12 Adjunctive Glucocorticoid Therapy in Patients with Septic Shock

B Venkatesh , S Finfer , J Cohen , D Rajbhandari , Y Arabi , R Bellomo , L Billot , M Correa , P Glass , M Harward , C Joyce , Q Li , C McArthur , A Perner , A Rhodes , K Thompson , S Webb , J Myburgh

To read the full abstract: N Engl J Med. 2018; 378(9): 797-808Sepsis has been identified by the WHO as a global health priority, however, there has been no proven pharmacologic treatment other than the appropriate antibiotic agents, fluids and vasopressors as required. Reported death rates among hospitalized patients with sepsis range between 30-45% (22). Glucocorticoids have been used as ...

ey0015.8-13 | New Genes | ESPEYB15

8.13 Activating PRKACB somatic mutation in cortisol-producing adenomas

S Espiard , MJ Knape , K Bathon , G Assié , M Rizk-Rabin , S Faillot , W Luscap-Rondof , D Abid , L Guignat , D Calebiro , FW Herberg , CA Stratakis , J Bertherat

To read the full abstract: JCI Insight. 2018; 3(8). pii: 98296Adrenocortical cells depend on cAMP/PKA signaling for growth and steroidogenesis. Several adrenal disorders manifesting with Cushing syndrome (CS) are due to activation of the PKA pathway, including Carney complex and primary pigmented nodular adrenal disease (due to germline mutations of PRKA1RA), McCune-Albright syndrome (due to ...

ey0015.10-15 | Comorbidities – short and long-term complications | ESPEYB15

10.15 ACE inhibitors and statins in adolescents with T1DM

ML Marcovecchio , ST Chiesa , S Bond , D Daneman , S Dawson , KC Donaghue , TW Jones , FH Mahmud , SM Marshall , HAW Neil , RN Dalton , J Deanfield , DB Dunger , AdDIT Study Group

To read the full abstract: New Engl J Med 2017; 377:1733-1745We are still unable to reach treatment goals in all children and adolescents with T1DM. The usually earliest microvascular complication developed in adolescents and young adults is microalbuminuria. Therefore this trial focused on the prevention of microalbuminuria in high risk adolescents with an albumin-to-creatinine ratio in the uppe...

ey0015.10-19 | Prevention | ESPEYB15

10.19 Infant Feeding and Risk of T1DM in Two Large Scandinavian Birth Cohorts

NA Lund-Blix , S Dydensborg Sander , K Størdal , AM Nybo Andersen , KS Rønningen , G Joner , T Skrivarhaug , PR Njølstad , S Husby , LC Stene

To read the full abstract: Diabetes Care. 2017;40:920-927Breast feeding has been identified as one albeit weak protective factor protecting from the development of for example obesity, autoimmune disease and in particular T1DM. The mechanisms of this preventive effect are not known. One explanation might still be a confounding effect of social status, overall healthy lifestyles in the families in who...

ey0015.14-17 | Who could have predicted it? | ESPEYB15

14.17 Exenatide once weekly versus placebo in Parkinson’s disease: a randomised, double-blind, placebo-controlled trial

D Athauda , K Maclagan , SS Skene , M Bajwa-Joseph , D Letchford , K Chowdhury , S Hibbert , N Budnik , L Zampedri , J Dickson , Y Li , I Aviles-Olmos , TT Warner , P Limousin , AJ Lees , NH Greig , S Tebbs , T Foltynie

To read the full abstract: Lancet 2017;390:1664-1675Whoever could have imagined that a compound discovered as being secreted by enteroendocrine cells in the gut could have a role in treating Parkinson’s disease? GLP-1 does indeed have diverse actions on several peripheral organs (including tongue, stomach, adipose tissues, muscle, pancreas and liver) and on the brainstem to regulate...

ey0020.6-6 | Clinical Trials – New Treatments | ESPEYB20

6.6. Crinecerfont, a CRF1 receptor antagonist, lowers adrenal androgens in adolescents with congenital adrenal hyperplasia

Newfield Ron S , Sarafoglou Kyriakie , Fechner Patricia Y , Nokoff Natalie J , Auchus Richard J , Vogiatzi Maria G , Jeha George S , Giri Nagdeep , Roberts Eiry , Sturgeon Julia , Chan Jean L , Farber Robert H

Brief summary: This study evaluated the safety, tolerability and efficacy of Crinecerfont, a CRF1R antagonist in adolescents with classic congenital adrenal hyperplasia (CAH).Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD) is characterized by impaired cortisol synthesis and excess adrenal androgen secretion. Sufficient suppression of adrenal andogen production in classic CAH may be difficult with current formulations...

ey0020.10-1 | New Data on the Epidemiology of T2D in Children | ESPEYB20

10.1. Trends in incidence of youth-onset type 1 and type 2 diabetes in the USA, 2002-18: results from the population-based SEARCH for Diabetes in Youth study

LE Wagenknecht , JM Lawrence , S Isom , ET Jensen , D Dabelea , AD Liese , LM Dolan , AS Shah , A Bellatorre , K Sauder , S Marcovina , K Reynolds , C Pihoker , G Imperatore , J Divers , SEARCH for Diabetes in Youth Study Divers

Brief summary: The incidences of T1D and T2D in children and young people increased in the USA over the last 2 decades. For the first time, the incidence of T2D has surpassed that of T1D.Comment: The SEARCH study has served as a national resource to explore the epidemiology and consequences of diabetes in the US. Published over the years, the findings contribute to understanding future trends in other parts of the world. Overall, the current report shows...

ey0018.2-10 | Neonatal diabetes mellitus | ESPEYB18

2.10. NKX2-2 mutation causes congenital diabetes and infantile obesity with paradoxical glucose-induced ghrelin secretion.

A Auerbach , A Cohen , N Ofek Shlomai , A Weinberg-Shukron , S Gulsuner , MC King , R Hemi , E Levy-Lahad , A Abulibdeh , D Zangen

J Clin Endocrinol Metab. 2020 Nov 1;105(11):dgaa563. doi: 10.1210/clinem/dgaa563. PMID: 32818257.This manuscript describes an unusual case of a baby born with a homozygous Nkx2.2 mutation who developed severe neonatal diabetes mellitus and then on follow up went onto develop severe obesity characterized by marked hyperphagia.Nkx2.2 is an important transcription factor...