ISSN 1662-4009 (online)

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Yearbook of Paediatric Endocrinology 2021

3. Thyroid

Clinical studies

ey0018.3-12 | Clinical studies | ESPEYB18

3.12. Randomised trial of block and replace vs. dose titration thionamide in young people with thyrotoxicosis

CL Wood , M Cole , M Donaldson , DB Dunger , R Wood , N Morrison , JNS Matthews , SHS Pearce , TD Cheetham

Eur J Endocrinol. 2020;183:637–645. doi: 10.1530/EJE-20-0617.This study provides for the first time clear evidence that the block and replace (BR) strategy (combination of carbimazole plus levothyroxine) is not superior to the carbimazole dose titration (DT) strategy for treatment of paediatric Graves’ disease.Wood et al. present the first randomized c...

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...

ey0018.3-14 | Clinical studies | ESPEYB18

3.14. Identification of resistance to exogenous thyroxine in humans

N Lacamara , B Lecumberri , B Barquiel , A Escribano , I Gonzalez-Casado , C Alvarez-Escola , F Aleixandre-Blanquer , F Morales , R Alfayate , MC Bernal-Soriano , R Miralles , I Yildirim Simsir , AG Ozgen , J Bernal , P Berbel , JC Moreno

Thyroid. 2020;30:1732–1744. doi: 10.1089/thy.2019.0825.This study is of importance, revealing a relevant clinical problem in patients under long-term levothyroxine (LT4) substitution and challenging the concept that all hypothyroid patients can be well controlled with LT4 monotherapy.Lacámara et al. present a small but elegant clinical study describi...