Evaluation of Phoenix Sepsis Score Criteria: Exploratory Analysis of Characteristics and Outcomes in an Emergency Transport PICU Cohort From the United Kingdom, 2014-2016.

Objectives: To assess characteristics and outcomes of children with suspected or confirmed infection requiring emergency transport and PICU admission and to explore the association between the 2024 Phoenix Sepsis Score (PSS) criteria and mortality.

Design: Retrospective analysis of curated data from a 2014-2016 multicenter cohort study.

Setting: PICU admission following emergency transport in South East England, United Kingdom, from April 2014 to December 2016.

National survey found that paediatricians frequently used messaging apps to hold informal consultations with parents and patients.

Aim: Paediatricians using instant messaging phone apps for informal medical discussions poses ethical and legal risks. We filled a gap in the research, by assessing the use of apps and the possible risks.

Methods: A national, cross-sectional, questionnaire study was conducted in Israel from 11 August to 20 November 2019.

Novel Staged Device Occlusion of Portosystemic Shunt With Atrial Flow Regulator in IVC Stent Platform.

We present the cases of 2 children diagnosed with extrahepatic portosystemic shunts, a very rare vascular anomaly, on investigation of cardiac symptomatology. Poorly developed portal venous systems necessitated staged shunt occlusion. This was achieved using atrial flow regulator devices positioned in an inferior vena cava stent platform performed in the cardiac catheterization laboratory.

Cell-based BSEP trans-inhibition: A novel, non-invasive test for diagnosis of antibody-induced BSEP deficiency.

Background & Aims: Antibody-induced bile salt export pump deficiency (AIBD) is an acquired form of intrahepatic cholestasis, which may develop following orthotopic liver transplantation (OLT) for progressive familial intrahepatic cholestasis type 2 (PFIC-2). Approximately 8-33% of patients with PFIC-2 who underwent a transplant develop bile salt export pump (BSEP) antibodies, which trans-inhibit this bile salt transporter from the extracellular, biliary side. AIBD is diagnosed by demonstration of BSEP-reactive and BSEP-inhibitory antibodies in patient serum.

Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort.

Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts AIM: To explore these models in a prospective multicentre inception cohort METHODS: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC).

Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively.

Polygenic risk score-based phenome-wide association study identifies novel associations for Tourette syndrome.

Tourette Syndrome (TS) is a complex neurodevelopmental disorder characterized by vocal and motor tics lasting more than a year. It is highly polygenic in nature with both rare and common previously associated variants. Epidemiological studies have shown TS to be correlated with other phenotypes, but large-scale phenome wide analyses in biobank level data have not been performed to date.

Early-life liver cirrhosis and variable clinical presentation in telomere disease.

Aim: Telomeres are DNA sequences of tandem TTAGGG repeats that protect chromosome ends from degradation and instability. Constitutional loss-of-function telomerase mutations result in rapid telomere shortening, premature senescence and cell death. Liver cirrhosis is rare and has only been reported in adults.

Disease severity of people with cystic fibrosis carrying residual function mutations: Data from the ECFS Patient Registry.

Rational: People with cystic fibrosis carrying residual function (RF) mutations are considered to have a mild disease course. This may influence caregivers and patients on how intensive the treatments should be.

Objectives: Characterize disease severity of patients carrying RF mutations, using the European CF Society Patient Registry (ECFSPR) data.

Lack of Association of Group A Streptococcal Infections and Onset of Tics: European Multicenter Tics in Children Study.

Background And Objectives: The goal of this work was to investigate the association between group A streptococcal (GAS) infections and tic incidence among unaffected children with a family history of chronic tic disorders (CTDs).

Methods: In a prospective cohort study, children with no history for tics who were 3 to 10 years of age with a first-degree relative with a CTD were recruited from the European Multicentre Tics in Children Study (EMTICS) across 16 European centers. Presence of GAS infection was assessed with throat swabs, serum anti-streptolysin O titers, and anti-DNAse titers blinded to clinical status.

Gonadectomy in conditions affecting sex development: a registry-based cohort study.

Objectives: To determine trends in clinical practice for individuals with DSD requiring gonadectomy.

Design: Retrospective cohort study.

Methods: Information regarding age at gonadectomy according to diagnosis; reported sex; time of presentation to specialist centre; and location of centre from cases reported to the International DSD Registry and who were over 16 years old in January 2019.

Folinic acid over rescue of high dose methotrexate: How problematic citations conserve discredited concepts.

Background: The outdated axiom that the dose of Folinic acid (FA) rescue used after high dose Methotrexate (HDMTX) should be kept to a minimum in order to prevent a reduction of prognosis ("over rescue") continues to be expressed even though the concept has been seriously challenged. Study aim The ways "problematic citations" are used to support an old theory, such as this, was examined.

Results: Ten patterns of "problematic citation" use were identified.

Add-on therapy with dapagliflozin under full closed loop control improves time in range in adolescents and young adults with type 1 diabetes: The DAPADream study.

Aim: To investigate the effect of the sodium-glucose co-transporter-2 inhibitor dapagliflozin on glucose levels overnight and during the following day after two unannounced meals under full closed loop (FCL) conditions.

Materials And Methods: For this single-centre, double-blind, randomized, placebo-controlled, cross-over trial, non-obese persons with type 1 diabetes (T1D) were studied twice (10 mg dapagliflozin bid vs. placebo) for 24 hours with two unannounced mixed meal tests 6 hours apart under FCL conditions.

Pediatric Preparedness of European Emergency Departments: A Multicenter International Survey.

Introduction: Children and adolescents often lack optimal emergency care. The objective of the study was to assess the level of preparedness of European emergency departments (EDs) for pediatric patients.

Methods: This was an international multicenter Internet-based survey of EDs with attending children and adolescents younger than 18 years in 101 EDs from 21 countries.

Faster Compared With Standard Insulin Aspart During Day-and-Night Fully Closed-Loop Insulin Therapy in Type 1 Diabetes: A Double-Blind Randomized Crossover Trial.

Objective: We evaluated the safety and efficacy of day-and-night fully closed-loop insulin therapy using faster (Faster-CL) compared with standard insulin aspart (Standard-CL) in young adults with type 1 diabetes.

Research Design And Methods: In a double-blind, randomized, crossover trial, 20 participants with type 1 diabetes on insulin pump therapy (11 females, aged 21.3 ± 2.

Long-term Outcomes of Paediatric Patients Admitted With Acute Severe Colitis- A Multicentre Study From the Paediatric IBD Porto Group of ESPGHAN.

Background And Aim: Acute severe colitis [ASC] is associated with significant morbidity in paediatric patients with ulcerative colitis [UC]. Most outcome studies in ASC since tumour necrosis factor alpha [TNFα] antagonists became available have focused on the first year after admission. The aim of this study was to characterise the longer-term outcomes of paediatric patients admitted with ASC.

Validation and impact of paediatric malnutrition screening tool in hospitalised children on awareness of medical staff and health-related outcomes.

Aims: This study aimed to evaluate the use of the Screening Tool for the Assessment of Malnutrition in Paediatrics (STAMP) among children admitted in a paediatric hospital, and assess its impact on the nutritional status awareness among the medical staff and on health outcomes at discharge.

Methods: STAMP performed by nurses on admission was compared with full nutritional assessment performed by a dietitian. Area under the receiving operating characteristic (AUROC) curve was used to evaluate validity of the tool.

The Brussels Infant and Toddler Stool Scale: A Study on Interobserver Reliability.

Objectives: The Bristol Stool Form Scale (BSFS) is inadequate for non-toilet trained children. The Brussels Infant and Toddler Stool Scale (BITSS) was developed, consisting of 7 photographs of diapers containing stools of infants and toddlers. We aimed to evaluate interobserver reliability of stool consistency assessment among parents, nurses, and medical doctors (MDs) using the BITSS.

Thrombophilia screening and thromboprophylaxis may benefit specific ethnic subgroups with paediatric acute lymphoblastic leukaemia.

This study investigated the prevalence of inherited thrombophilia, risk of venous thromboembolism (VTE) and benefit of low molecular weight heparin prophylaxis in 476 Israeli children with acute lymphoblastic leukaemia (ALL) treated between 2004 and 2016. Thrombophilia was found in 15·5%. Arab children had a higher prevalence of F5 R506Q (factor V Leiden) than Jewish children (19·4% vs.