ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Front Endocrinol (Lausanne) 2017;8:170Increasing sedentary behaviours and availability of high energy dense processed foods are the obvious culprits to explain the trends towards more overweight and obese populations around the world. However, we should remember that many other hypotheses are proposed, including higher ambient room temperatures due to ce...

Brief summary: Patients with congenital hypothyroidism have a higher rate of comorbidities in adult age (1). This nationwide population-based register study aimed at investigating the incidence of comorbidities present at birth or acquired during childhood in patients with congenital hypothyroidism and prescription of specific drugs (antidepressants, antipsychotics, medication for ADHS).The study analyzed data from n=438 full-term patients with ...

Brief summary: This study reports data from the full Kabi/Pfizer International Growth Database (KIGS), a large international database including 83803 children treated with rhGH for GHD and non-GHD conditions. The data confirm that rhGH is effective in increasing short-term height gain and adult height in various conditions and validate rhGH safety, with no new serious adverse events reported.KIGS (Kabi/Pfizer International Growth Database), first establi...

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...

In Brief: This phase 1 study in healthy adults assessed the safety and feasibility of transcon-CNP, a novel prodrug that releases native C-type natriuretic peptide (CNP). The novel drug was well tolerated and CNP remained in systemic circulation for >7 days following a single dose.Commentary: Achondroplasia is caused by autosomal activating mutation in the fibroblast growth factor receptor 3 gene (FGFR3) resulting in constitutive receptor ac...

Brief summary: This prospective longitudinal study of childhood-onset inflammatory bowel disease (IBD) analysed areal bone mineral density (aBMD) and alterations of bone microstructure in 49 young adult male patients with childhood-onset IBD, compared to 249 controls from the same region and matched for age, sex and height.Dual x-ray-absorptiometry (DXA) is routinely used in clinical practice to evaluate aBMD and the related risk of fracture, but it is u...

Brief summary: This Iranian open label, quasi-experimental and matched controlled clinical trial included 59 girls with stage III–IV chronic kidney disease (CKD), short stature and delayed puberty. Patients were treated with GH (mean dose 0.05 mg/kg/day) and Ethinyl Estradiol (EE). Initial EE dose was 5 μg/day orally, doubled every 3–6 months to a maximum dose before growth plate closure 30 μg/day, and then increased gradually up to 500 μg/day. EE ther...

Brief summary: This single-centre retrospective study evaluated cortisol responses during febrile episodes in children with cancer. Low cortisol responses were common and were unrelated to current steroid therapy.Random serum cortisol and ACTH were measured in 75 children and adolescents with cancer while admitted for fever during chemotherapy; 47/75 patients received glucocorticoids as part of their treatment (steroid group). A low cortisol response (LC...

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: This ‘Letter to the Editor’ provides a critical review on how behavioural and physiological aspects of type 1 diabetes (T1D) and its management can interfere with standard principles of good quality and duration of sleep.Quality of sleep is important for general human functioning, cognitive performance, emotional well-being, as well as immune function and cardiovascular health (1). The benefits of optimal sleep apply to everyone ...