ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Int J Obes 2018; 42: 115-118Intragastric balloons have now been used for more than 30 years in the treatment of extreme obesity. There are more than 30 studies published with almost 5,000 patients on the efficacy and safety of gastric balloons. However, due to several, sometimes severe side effects and the lack of convincing long-term outcomes, this treatment approach ha...

To read the full abstract: Proceedings of the National Academy of Sciences 2017:201711889Changing your diet can improve both your health and the environment. This study shows that the national dietary recommendations to reduce intakes of animal products can reduce environmental impacts in most high-income nations. We all recommended diets to our patients, and nations recommend diets to their po...

To read the full abstract: Lancet 2017;389:2473-2481We are all familiar with the ‘placebo’ effect, when the psychological anticipation of a ‘benefit’ of treatment is so strong that it adds to or even outweighs the actual physical benefits. Many doctors have even admitted to prescribing placebo tablets, or “sugar pills” to their patients, for exam...

Brief summary: This is the first multi-national phase 2 dose finding trial aimed at investigating efficacy and safety of the long acting GH (Somapacitan) with daily GH in short children born SGA. 62 GH treatment-naïve short children born SGA were enrolled in the study. Efficacy was valuated with estimated mean annualized height velocity (HV) after 26 weeks of follow-up. Somapacitan showed similar efficacy of daily rhGH. The study will be extended for further 4 years in or...

Brief summary: This Danish single-center retrospective study evaluated the occurrence of pathological findings on neuroimaging among children diagnosed with central precocious puberty (CPP).CPP incidence has been increasing over the last four decades (1) and is more prevalent in females than in males. Most cases are idiopathic (2) but brain magnetic resonance imaging (MRI) is routinely performed in order to exclude rare pathological causes.<p class="...

Brief summary: This commentary discusses the implications of the recent licensing of Teplizumab by the Food and Drug Administration (FDA) as the first immunosuppressant for individuals at risk for type 1 diabetes (T1D).The approval of Teplizumab by the FDA in November 2022, as an intervention to delay the onset of stage 3 T1D (clinical T1D) in adults and children aged 8 or older who have stage 2 T1D (two or more islets autoantibodies and dysglycemia but ...

Brief summary: In this double-blind, randomized, placebo-controlled trial, bempedoic acid (Nexletol) reduced the incidence of a composite cardiovascular.Comment: Bempedoic acid is an ATP citrate lyase inhibitor. ATP citrate lyase catalyses the conversion of citrate to acetyl-CoA, the fundamental substrate required for the synthesis of cholesterol and fatty acids. By inhibiting the cholesterol synthesis pathway, bempedoic acid reduces intra-hepatic LDL-C....

In Brief: The authors analysed data from the nationwide Child and Adolescent Twin Study in Sweden (CATSS) to estimate the heritability of avoidant restrictive food intake disorder (ARFID) as defined by DSM-5 criteria. Of the 16 951 twin pairs born between 1992 and 2010, 682 (2.0%) children were identified to have ARFID. By modelling shared risk in monozygous compared to dizygous twin pairs, they estimated the heritability of ARFID as 79% (95%CI, 70–85).<...

Brief Summary: This case series describes the effect of recombinant human IGF-1 (rhIGF-1) administration on growth of three siblings with STAT5B homozygous recessive mutations.The peripheral effects of GH are primarily mediated by IGF-I through the activation of the GH receptor (GHR)-signal transducer and activator of transcription (STAT)-5B signaling. Patients carrying STAT5B mutations have severe postnatal growth failure and IGF-I deficiency a...

Brief Summary: This study examined the potential benefits of Verapamil, a calcium channel blocker traditionally used to treat hypertension, in Type 1 Diabetes (T1D). It shows that Verapamil can prevent the decline of Insulin-like Growth Factor I (IGF-I) levels, which are crucial for β-cell survival and insulin production. Moreover, Verapamil reduced the expression of IGF-binding protein 3 (IGFBP3), enhancing IGF-I signaling and protecting β-cells from apoptosis. Henc...