ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica

Page 3 of about 54 results

ey0015.12-13 | New treatments | ESPEYB15

12.13 Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children with Heterozygous Familial Hypercholesterolemia: The CHARON Study (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label)

M Braamskamp , G Langslet , BW McCrindle , D Cassiman , GA Francis , C Gagne , D Gaudet , KM Morrison , A Wiegman , T Turner , E Miller , DM Kusters , JS Raichlen , PD Martin , EA Stein , JJP Kastelein , BA Hutten

To read the full abstract: Circulation 2017;136:359-366The National Heart, Lung and Blood Institute Panel on integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents recommended a universal screening approach that would include one-time testing of all children aged 9-11 years for dyslipidemia. This recommendation has raised controversy regarding t...
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ey0020.8-16 | New Hopes | ESPEYB20

8.16. Engineering the lymph node environment promotes antigen-specific efficacy in type 1 diabetes and islet transplantation

JM Gammon , ST Carey , V Saxena , HB Eppler , SJ Tsai , C Paluskievicz , Y Xiong , L Li , M Ackun-Farmmer , LH Tostanoski , EA Gosselin , AA Yanes , X Zeng , RS Oakes , JS Bromberg , CM Jewell

Brief summary: In this experimental study, immunomodulatory microparticles, consisting of encapsulating self-antigens with rapamycin, were injected into mouse lymph nodes to protect against type 1 diabetes (T1D) and islet graft rejection. Antigens and rapamycin were both required for maximal efficacy and they induced durable tolerance, accompanied by expansion of antigen-specific regulatory T cells (Treg) in both treated and untreated lymph nodes.Antigen...
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ey0020.9-8 | Genetic Obesity and Genetic Risk Score | ESPEYB20

9.8. A National Multicenter Study of Leptin (LEP) and Leptin Receptor (LEPR) deficiency and systematic review

O Besci , SN Fırat , S Ozen , S Cetinkaya , L Akın , Y Kor , Z Pekkolay , S Ozalkak , E Ozsu , SS Erdeve , S Poyrazoglu , M Berberglu , M Aydin , T Omma , B Akinici , K Demir , EA Oral

Brief summary: This paper describes 18 patients with bi-allelic leptin deficiency (LEP, n=11) or leptin receptor deficiency (LEPR, n=7), including 10 new cases and two novel variants. In addition, in a review of the literature (until July 2022), the authors identified n=75 patients living with LEP deficiency and n=90 with LEPR deficiency (n=152 included for comparison between groups).<p class=...
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ey0016.14-13 | (1) | ESPEYB16

14.13. Effect of genetic diagnosis on patients with previously undiagnosed disease

K Splinter , DR Adams , CA Bacino , HJ Bellen , JA Bernstein , AM Cheatle-Jarvela , CM Eng , C Esteves , WA Gahl , R Hamid , HJ Jacob , B Kikani , DM Koeller , IS Kohane , BH Lee , J Loscalzo , X Luo , AT McCray , TO Metz , JJ Mulvihill , SF Nelson , CGS Palmer , JA, 3rd Phillips , L Pick , JH Postlethwait , C Reuter , V Shashi , DA Sweetser , CJ Tifft , NM Walley , MF Wangler , M Westerfield , MT Wheeler , AL Wise , EA Worthey , S Yamamoto , EA Ashley

To read the full abstract: N Engl J Med 2018;379:2131–2139.The authors reviewed data on 1519 patients referred to the Undiagnosed Diseases Network (UDN), a US NIH funded network linking seven clinical sites. 53% of patients were female and their symptoms were neurologic (40%), musculoskeletal (10%), immunological (7%), gastrointestinal (7%), or rheumatological (6%). Of the 38...
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ey0019.11-3 | New findings in adipose tissue biology | ESPEYB19

11.3. Isthmin-1 is an adipokine that promotes glucose uptake and improves glucose tolerance and hepatic steatosis

Z Jiang , M Zhao , L Voilquin , Y Jung , MA Aikio , T Sahai , FY Dou , AM Roche , I Carcamo-Orive , JW Knowles , M Wabitsch , EA Appel , CL Maikawa , JP Camporez , GI Shulman , L Tsai , ED Rosen , CD Gardner , BM Spiegelman , KJ Svensson

katrinjs@stanford.edu Cell Metab 2021; 33(9): 1836–1852.e11http://www.ncbi.nlm.nih.gov/pubmed/34348115Brief Summary: This rodent study identified a novel adipokine in mice which triggers a signaling cascade similar to that of insulin. By acting via an unknown tyrosine kinase, isthmin-1 (ISM1) ameliorates metabolic disturbances associated wit...
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ey0016.5-8 | Clinical Advances in Treatment | ESPEYB16

5.8. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial

EA Imel , FH Glorieux , MP Whyte , CF Munns , LM Ward , O Nilsson , JH Simmons , R Padidela , N Namba , HI Cheong , P Pitukcheewanont , E Sochett , W Hogler , K Muroya , H Tanaka , GS Gottesman , A Biggin , F Perwad , M Mao , CY Chen , A Skrinar , J San Martin , AA Portale

Abstract: Lancet. 2019 May 16.In brief: In a randomised, active-controlled, open-label, phase 3 trial, burosumab (an anti-FGF23 antibody) demonstrated significantly greater clinical improvements in rickets severity, growth, and biochemistries among children with X-linked hypophosphataemia compared with continuation of conventional therapy with oral phosphate and active vitamin D ...
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ey0020.10-2 | New Data on the Epidemiology of T2D in Children | ESPEYB20

10.2. The Coronavirus Disease 2019 pandemic is associated with a substantial rise in frequency and severity of presentation of youth-onset type 2 diabetes

SN Magge , RM Wolf , L Pyle , EA Brown , VC Benavides , ME Bianco , LC Chao , A Cymbaluk , PG Balikcioglu , K Halpin , DS Hsia , L Huerta-Saenz , JJ Kim , S Kumar , LE Levitt--Katz , BE Marks , A Neyman , KL O'Sullivan , SS Pillai , AS Shah , AH Shoemaker , JAW Siddiqui , S Srinivasan , IH Thomas , JB Tryggestad , MF Yousif , MM Kelsey , COVID-19 and Type 2 Diabetes Consortium Kelsey

Brief summary: A significant increase in the incidence T2D occurred in the USA during the COVID-19 pandemic, particularly among children with obesity and males.Comment: This is the first multicenter report on the incidence of T2D during the first year of the COVID-19 pandemic in the USA. The average number of new diagnoses per year in the two pre-pandemic years was 825, compared with 1463 during the first pandemic year. This increase of 77% is significan...
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ey0017.14-10 | (1) | ESPEYB17

14.10. Low-dose aspirin for the prevention of preterm delivery

MK Hoffman , SS Goudar , BS Kodkany , M Metgud , M Somannavar , J Okitawutshu , A Lokangaka , A Tshefu , CL Bose , A Mwapule , M Mwenechanya , E Chomba , WA Carlo , J Chicuy , L Figueroa , A Garces , NF Krebs , S Jessani , F Zehra , S Saleem , RL Goldenberg , K Kurhe , P Das , A Patel , PL Hibberd , E Achieng , P Nyongesa , F Esamai , EA Liechty , N Goco , J Hemingway-Foday , J Moore , TL Nolen , EM McClure , M Koso-Thomas , M Miodovnik , R Silver , RJ Derman , ASPIRIN Study Group

To read the full abstract: Lancet. 2020 Jan 25;395(10220):285-293. doi: 10.1016/S0140-6736(19)32973-3.This randomised, placebo-controlled trial (‘ASPIRIN’) in 11,976 nulliparous women in six low or middle-income countries (India, Democratic Republic of the Congo, Guatemala, Kenya, Pakistan, and Zambia) found that low-dose aspirin (81 mg daily from early pregnancy) reduced t...
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ey0017.14-8 | (1) | ESPEYB17

14.8. Patient-customized oligonucleotide therapy for a rare genetic disease

J Kim , C Hu , C Moufawad El Achkar , LE Black , J Douville , A Larson , MK Pendergast , SF Goldkind , EA Lee , A Kuniholm , A Soucy , J Vaze , NR Belur , K Fredriksen , I Stojkovska , A Tsytsykova , M Armant , RL DiDonato , J Choi , L Cornelissen , LM Pereira , EF Augustine , CA Genetti , K Dies , B Barton , L Williams , BD Goodlett , BL Riley , A Pasternak , ER Berry , KA Pflock , S Chu , C Reed , K Tyndall , PB Agrawal , AH Beggs , PE Grant , DK Urion , RO Snyder , SE Waisbren , A Poduri , PJ Park , A Patterson , A Biffi , JR Mazzulli , O Bodamer , CB Berde , TW. Yu

To read the full abstract: N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279.This remarkable case report - by authors from Boston Children’s Hospital and funded by Mila’s Miracle Foundation - describes the development and use of a patient-customised antisense oligonucleotide drug that was tailored specifically against the unique DNA sequence mutation in a 6-ye...
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ey0020.5-1 | Clinical Guidance and Studies | ESPEYB20

5.1. Leuprolide and triptorelin treatment in children with idiopathic central precocious puberty: an efficacy/tolerability comparison study

M Valenzise , C Nasso , A Scarfone , M Rottura , G Cafarella , G Pallio , G Visalli , E Di Prima , E Nasso , V Squadrito , M Wasniewska , P Irrera , V Arcoraci , F Squadrito

Brief summary: This retrospective study compared the efficacy and tolerability profiles of leuprolide and triptorelin in patients with central precocious puberty and did not find any significant difference between the two drugs.Treatment with GnRH analogues represents the standard of care for central precocious puberty (CPP) in order to preserve adult height potential1. Leuprolide and Triptorelin are the most prescribed drugs (1,2). They are g...
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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