ESPE Yearbook of Paediatric Endocrinology

Brief summary: This retrospective study describes a marked increase in the survival of children and youth living with Type 1 diabetes mellitus (T1D) in Tanzania, before (1991–2004), during (2005–2010) and after (2011–2019) implementation of the Life For A Child (LFAC) and Changing Diabetes in Children (CDiC) programs.This article focuses specifically on diabetes-related mortality. It offers both a message of hope and a candid examination o...

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 study used single-cell RNA-seq and murine transgenic models combined with elegant cell lineage tracing to characterise the gene expression profiles and trajectories of pituitary thyrotropes. It identifies a novel population of pituitary thyrotropes that co-express Nr5a1 (Sf1) and Pouf1 (Pit1) and a novel developmental trajectory for a subpopulation of Nr5a1-derived thyrotropes.Classical studies on pituitary cell lineages and cell diffe...

Brief Summary: This single-center, prospective study used brain MRI to assess the prevalence of cerebrovascular abnormalities in a large cohort of adult patients born SGA and treated with GH during childhood, 12 years after rhGH treatment cessation. GH treatment was not associated with a higher incidence of aneurysms, intracerebral hemorrhages, microbleeds or other vascular abnormalities.The French population-based cohort of the Safety and Appropriatenes...

Brief Summary: This large patient cohort of children with endogenous Cushing syndrome describes their anthropometric, clinical, and biochemical characteristics, as well as their associated complications and outcomes. The findings inform diagnosis, treatment, and management.Comment: Endogenous Cushing syndrome is rare in children and adolescents, and has variable manifestations (1-6). This prospective, multicenter cohort of 342 children and adolescents ag...

Brief Summary: This brief and concise Consensus statement by the French Society of Endocrinology (SFE) and French Society of Pediatric Endocrinology & Diabetology (SFEDP), describes a systematic review and provides recommendations for the use of genetic screening in Cushing’s syndrome (CS).Comment: The etiology of CS may involve both germline genetic alterations (detectable in leukocyte DNA) and somatic mutations (present only in tumor DNA). Two...

Brief Summary: This retrospective study evaluated the risk and clinicopathological features of second primary thyroid cancer (SPTC) in cancer survivors. The Surveillance, Epidemiology, and End Results (SEER) Program collected cancer incidence and mortality data from 8 population-based registries. They identified 7066 patients with SPTC and 83,113 patients with primary thyroid cancer (TC). The standardized incidence ratio (SIR) of SPTC in cancer survivors was higher than in the...

Brief Summary: This double-blind, multicenter trial randomized 328 children and adolescents (8-17 years-old) diagnosed with type 1 diabetes (T1D) within the past 6 weeks to receive either teplizumab or placebo for two 12-day courses. Teplizumab increased stimulated C-peptide levels after 1.5 years, indicating better preservation of β-cell function.Teplizumab is a monoclonal antibody that binds to CD3 on the surface of T-cells, thereby reducing the i...

Brief Summary: The Diabetes Virus Detection (DiViD) Intervention phase 2, placebo-controlled, double-blind trial randomized 96 children and adolescents (age 6-15 years) with new-onset type 1 diabetes (T1D) to receive either the antivirals, Pleconaril and Ribavirin, (n=47) or placebo (n=49) for 6 months. The antiviral treatment improved β-cell function at 12 months, and it was well tolerated and safe.Viral infections, particularly enteroviruses, have...

Brief Summary: This experimental study used positron emission tomography (PET) as non-invasive method to monitor stem cells-islet grafts. [18F]exendin and [18F]FDOPA PET showed potential as non-invasive methods to assess stem cells-islet graft size and aspects of graft composition.Stem cell-derived islets (SC-islets) are used as an emerging cell replacement therapy for insulin-dependent diabetes (1). Non-invasive long-term monitorin...