ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica

Page 1 of about 8 results

ey0017.13-3 | Advocacy, History and Society | ESPEYB17

13.3. Effective coverage measurement in maternal, newborn, child, and adolescent health and nutrition: Progress, future prospects, and implications for quality health systems

AD Marsh , M Muzigaba , T Diaz , J Requejo , D Jackson , D Chou , J Cresswell A , R Guthold , A Moran C , K Strong L , A Banerjee , A Soucat

To read the full abstract: Lancet Glob Health 2020; 8: e730–36. doi: 10.1016/S2214-109X(20)30104-2• Sustainable Development Goals (SDG) were adopted by United Nations Member States in 2015. Universal health coverage is at the centre of SDG #3 but lacks metrics that make it possible to assess how effective the provided healthcare is.• WHO and UNICEF convened a group of ...
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ey0017.10-8 | (1) | ESPEYB17

10.8. An anti-CD3 antibody, teplizumab, in relatives at risk for type 1 diabetes

KC Herold , BN Bundy , SA Long , JA Bluestone , LA DiMeglio , MJ Dufort , SE Gitelman , PA Gottlieb , JP Krischer , PS Linsley , JB Marks , W Moore , A Moran , H Rodriguez , WE Russell , D Schatz , JS Skyler , E Tsalikian , DK Wherrett , AG Ziegler , CJ Greenbaum , Type 1 Diabetes TrialNet Study Group

To read the full abstract: N Engl J Med. 2019 Aug 15;381(7):603–613. doi: 10.1056/NEJMoa 1902226.Type 1 diabetes (T1DM) is a chronic autoimmune disease that leads to destruction of insulin producing beta cells, which leaves the patient dependent on exogenous insulin for survival. Some interventions have delayed the loss of insulin production in T1DM, but interventions to prevent clinic...
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ey0020.8-1 | Clinical Trials – New Treatments | ESPEYB20

8.1. Effect of Verapamil on pancreatic beta cell function in newly diagnosed pediatric type 1 diabetes: A randomized clinical trial

GP Forlenza , J McVean , RW Beck , C Bauza , R Bailey , B Buckingham , LA DiMeglio , JL Sherr , M Clements , A Neyman , C Evans-Molina , EK Sims , LH Messer , L Ekhlaspour , R McDonough , M Van Name , D Rojas , S Beasley , S DuBose , C Kollman , A Moran , CLVer Study Group

Brief summary: In this double-blind, randomized clinical trial conducted in 6 US centres, 88 children and adolescents (aged 7–17 years) with newly diagnosed type 1 diabetes (T1D) were randomized to either once-daily oral verapamil or placebo, within 1 month from diagnosis. Treatment with verapamil led to better stimulated C-peptide secretion at 52 weeks post-diagnosis, with levels 30% higher compared to placebo.Verapamil is a calcium channel blocker...
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ey0021.3-10 | Defects in Thyroid Hormone Transport, Metabolism and Action | ESPEYB21

3.10. Resistance to thyroid hormone induced tachycardia in RTH[alpha] syndrome

Dore Riccardo , Watson Laura , Hollidge Stefanie , Krause Christin , Sentis Sarah Christine , Oelkrug Rebecca , Geiszler Cathleen , Johann Kornelia , Pedaran Mehdi , Lyons Greta , Lopez-Alcantara Nuria , Resch Julia , Sayk Friedhelm , Iwen Karl Alexander , Franke Andre , Boysen Teide Jens , Dalley Jeffrey W , Lorenz Kristina , Moran Carla , Rennie Kirsten L , Arner Anders , Kirchner Henriette , Chatterjee Krishna , Mittag Jens

Brief Summary: This study examined resistance to thyroid hormone (TH)-induced tachycardia in patients with Resistance to THα (RTHα). This rare condition is caused by mutations in the TH receptor alpha (TRα1), and leads to hypothyroidism in TRα1-expressing tissues, including the heart. While high doses of thyroxine (T4) are typically used to treat this condition, patients with RTHα do not exhibit the expected increase in heart rate despite overcoming ti...
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ey0018.14-11 | (1) | ESPEYB18

14.11. Ageing hallmarks exhibit organ-specific temporal signatures

Schaum Nicholas , Lehallier Benoit , Hahn Oliver , Palovics Robert , Hosseinzadeh Shayan , Lee Song E , Sit Rene , Lee Davis P , Losada Patricia Moran , Zardeneta Macy E , Fehlmann Tobias , Webber James T , McGeever Aaron , Calcuttawala Kruti , Zhang Hui , Berdnik Daniela , Mathur Vidhu , Tan Weilun , Zee Alexander , Tan Michelle , The Tabula Muris Consortium , Pisco Angela Oliveira , Karkanias Jim , Neff Norma F , Keller Andreas , Darmanis Spyros , Quake Stephen R , Wyss-Coray Tony

Nature 2020; 583: 596–602https://www.nature.com/articles/s41586-020-2499-yIn order to understand the cellular processes that underlie ageing, the authors performed plasma proteomics at 10 different ages across the lifespan of the mouse. They integrated these data with a parallel large study published alongside this paper in the same edition (1), which describes the ‘Mouse ...
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ey0017.3-12 | Clinical trials for thyroid disease | ESPEYB17

3.12. Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial

S Groeneweg , RP Peeters , C Moran , A Stoupa , F Auriol , D Tonduti , A Dica , L Paone , K Rozenkova , J Malikova , A van der Walt , IFM de Coo , A McGowan , G Lyons , FK Aarsen , D Barca , IM van Beynum , MM van der Knoop , J Jansen , M Manshande , RJ Lunsing , S Nowak , CA den Uil , MC Zillikens , FE Visser , P Vrijmoeth , MCY de Wit , NI Wolf , A Zandstra , G Ambegaonkar , Y Singh , YB de Rijke , M Medici , ES Bertini , S Depoorter , J Lebl , M Cappa , L De Meirleir , H Krude , D Craiu , F Zibordi , I Oliver Petit , M Polak , K Chatterjee , TJ Visser , WE Visser

To read the full abstract: Lancet Diabetes Endocrinol. 2019;7:695–706.The hallmarks of MCT8 deficiency are severe psychomotor retardation due to intracellular hypothyroidism in neuronal tissues and peripheral T3 thyrotoxicosis associated low weight, muscle wasting, high normal or increased heart rate and systolic blood pressure. TRIAC – the T3 analogue 3,3’,5-tri-iodothyroacetic acid – enters target cells in an MCT8 independe...
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ey0018.5-12 | Advances in skeletal biology | ESPEYB18

5.12. Osteoclasts recycle via osteomorphs during RANKL-stimulated bone resorption

McDonald Michelle M , Khoo Weng Hua , Ng Pei Ying , Xiao Ya , Zamerli Jad , Thatcher Peter , Kyaw Wunna , Pathmanandavel Karrnan , Grootveld Abigail K , Moran Imogen , Butt Danyal , Nguyen Akira , Corr Alexander , Warren Sean , Biro Mate , Butterfield Natalie C , Guilfoyle Siobhan E , Komla-Ebri Davide , Dack Michael R G , Dewhurst Hannah F , Logan John G , Li Yongxiao , Mohanty Sindhu T , Byrne Niall , Terry Rachael L , Simic Marija K , Chai Ryan , Quinn Julian M W , Youlten Scott E , Pettitt Jessica A , Abi-Hanna David , Jain Rohit , Weninger Wolfgang , Lundberg Mischa , Sun Shuting , Ebetino Frank H , Timpson Paul , Lee Woei Ming , Baldock Paul A , Rogers Michael J , Brink Robert , Williams Graham R , Bassett J H Duncan , Kemp John P , Pavlos Nathan J , Croucher Peter I , Phan Tri Giang

Cell. 2021 Mar 4;184(5):1330–1347.e13 Abstract: https://pubmed.ncbi.nlm.nih.gov/33636130/In brief: This paper reports an alternative cell fate for multinucleated, bone-resorbing osteoclasts and shows that they may undergo fission into smaller osteomorphs. The findings challenge the current dogma that osteoclasts primarily differentiate from hematopoietic progenitor cells and a...
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ey0018.3-13 | Clinical studies | ESPEYB18

3.13. Disease characteristics of MCT8 deficiency: an international, retrospective, multicentre cohort study

S Groeneweg , FS van Geest , A Abacı , A Alcantud , GP Ambegaonkar , CM Armour , P Bakhtiani , D Barca , ES Bertini , IM van Beynum , N Brunetti-Pierri , M Bugiani , M Cappa , G Cappuccio , B Castellotti , C Castiglioni , K Chatterjee , IFM de Coo , R Coutant , D Craiu , P Crock , C DeGoede , K Demir , A Dica , P Dimitri , A Dolcetta-Capuzzo , MHG Dremmen , R Dubey , A Enderli , J Fairchild , J Gallichan , B George , EF Gevers , A Hackenberg , Z Halasz , B Heinrich , T Huynh , A Kłosowska , MS van der Knaap , MM van der Knoop , D Konrad , DA Koolen , H Krude , A Lawson-Yuen , J Lebl , M Linder-Lucht , CF Lorea , CM Lourenco , RJ Lunsing , G Lyons , J Malikova , EE Mancilla , A McGowan , V Mericq , FM Lora , C Moran , KE Muller , I Oliver-Petit , L Paone , PG Paul , M Polak , F Porta , FO Poswar , C Reinauer , K Rozenkova , TS Menevse , P Simm , A Simon , Y Singh , M Spada , J van der Spek , MAM Stals , A Stoupa , GM Subramanian , D Tonduti , S Turan , CA den Uil , J Vanderniet , A van der Walt , JL Wemeau , J Wierzba , MY de Wit , NI Wolf , M Wurm , F Zibordi , A Zung , N Zwaveling-Soonawala , WE Visser

Lancet Diabetes Endocrinol. 2020;8:594–605. doi: 10.1016/S2213-8587(20)30153-4.Groenenweg et al. present the so far largest retrospective multicentre cohort study on 151 patients with 73 different MCT8 (SLC16A2) mutations to compare and describe in detail the phenotypic spectrum and the disease course of MCT8 deficiency. The careful description of presenting cl...
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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