Effectiveness and Safety of Adalimumab in Patients With Very Early-Onset Inflammatory Bowel Disease: A Retrospective Study on Behalf of the Porto Inflammatory Bowel Disease Working Group of European Society for Pediatric Gastroenterology Hepatology and Nutrition.

Background And Aims: Patients with very early-onset inflammatory bowel disease (VEO-IBD), with an age of onset < 6 years, can present with severe manifestations and may require biologic therapy. Infliximab and adalimumab are approved for induction and maintenance in pediatric IBD patients but are licensed only above the age of 6 years. Effectiveness and safety data on adalimumab in this patient population are lacking.

Baseline Drug Clearance Predicts Outcomes in Children With Inflammatory Bowel Disease Treated With Vedolizumab: Results From the VedoKids Prospective Multicentre Study.

Background: The pharmacokinetics of biologic agents can differ between children and adults with inflammatory bowel disease (IBD), often necessitating modified paediatric dosing strategies.

Aims: To define the exposure-response relationship of vedolizumab in the paediatric IBD VedoKids cohort including the effect of baseline clearance on deep biochemical remission (normal C-reactive protein [CRP]/erythrocyte sedimentation rate [ESR] and steroid-free remission) at 30 weeks, and to use population pharmacokinetic models to find the best matches between adult and paediatric pharmacokinetic profiles.

Methods: We sought a pharmacokinetic model on 312 serum vedolizumab concentrations from 129 children, assisted by a published adult model as a Bayesian prior.

Variability in disease severity among cystic fibrosis patients carrying residual-function variants: data from the European Cystic Fibrosis Society Patient Registry.

Background: People with cystic fibrosis (CF) variants that exhibit residual function (RF) of the CF transmembrane conductance regulator are considered to have a milder disease; however, the spectrum of CF phenotype within the different RF variants has not been extensively investigated. The aim of the present study was to characterise the spectrum of CF disease severity in people with CF (pwCF) carrying different RF variants, using the European Cystic Fibrosis Society Patient Registry (ECFSPR) data.

Methods: A retrospective cross-sectional and longitudinal cohort study included data from the ECFSPR during 2008-2016.

Anorectal malformation with rectoperineal fistula in males treated with a posterior rectal advancement anoplasty (PRAA) - report of early outcomes.

A novel modification of the cutback procedure, a posterior rectal advancement anoplasty (PRAA) for select male patients with an anorectal malformation and a rectoperineal fistula was recently described which incised only within the limits of the sphincteric ellipse and eliminated an anterior rectal dissection, thus avoiding any possibility of a urethral injury. This report provides longer term postoperative outcomes after PRAA. Method A retrospective, single institution study was performed examining male patients with a rectoperineal fistula between 1/2020-12/2023.

Chronobiological Factors Influencing Glycemic Control and Birth Outcomes in Gestational Diabetes Mellitus.

Background/objectives: Studies have shown that chronobiological factors may adversely affect glycemic control in patients with type 2 diabetes mellitus. We assessed the association of chronobiological factors with glycemic control and neonatal birth weight in women with GDM.

Methods: A prospective cohort study included 208 women aged 18-45 years with a singleton pregnancy who were randomly selected from among women undergoing follow-up for GDM at the Maternal-Fetal Medicine Unit of a tertiary medical center.

Efficacy and safety of ibuprofen and naproxen in the treatment of oligoarticular juvenile idiopathic arthritis: bi-national cohort study.

Objectives: Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in children. Nonsteroidal anti-inflammatory drugs (NSAIDs) and intra-articular corticosteroid injections are first-line therapy for oligoarticular JIA. NSAIDs Adverse events (AEs) include gastrointestinal ulcers/bleeding and impaired renal function.

Modeling GATA2 deficiency in mice: the R396Q mutation disrupts normal hematopoiesis.

GATA2 deficiency is an autosomal dominant germline disorder of immune dysfunction and bone marrow failure with a high propensity for leukemic transformation. While sequencing studies have identified several secondary mutations thought to contribute to malignancy, the mechanisms of disease progression have been difficult to identify due to a lack of disease-specific experimental models. Here, we describe a murine model of one of the most common GATA2 mutations associated with leukemic progression in GATA2 deficiency, Gata2.

Birth weight and head circumference for 22-29 weeks gestation neonates from an international cohort.

Objective: Size at birth is a key indicator of in utero growth. Our objective was to generate sex-specific percentiles for birth weight and head circumference in neonates born between 22 and 29 weeks gestation from pregnancies without hypertension or diabetes and assess differences between vaginal and caesarean births and between singletons and twins.

Methods: We used data from 12 countries participating in the International Network for Evaluating Outcomes in Neonates database from 2007 to 2021.

Small bowel duplication cyst in the pediatric population-when to operate?

Introduction: AIM: The aim of the study is to determine the optimal timing for surgery in patients with small bowel duplications.

Methods: A retrospective cohort study, including all patients younger than 18 years who were diagnosed with small bowel duplications from 2013 until 2024 in a single tertiary medical center, was performed. Patients' demographics, duplication size and location, pathological results, and clinical outcomes were collected.

Development of a reference standard to assign bacterial versus viral infection etiology using an all-inclusive methodology for comparison of novel diagnostic tool performance.

Background: Diagnostic test evaluation requires a reference standard. We describe an approach for creating a reference standard for acute infection using unrestricted adjudication and apply it to compare biomarker tools.

Methods: Adults and children with suspected acute infection enrolled in three prospective studies at emergency departments and urgent cares were included.

The Value of ROH Metrics for Predicting Morbidity: Insights From a Large Cohort Analysis of Chromosomal Microarray.

This retrospective cohort study aimed to define the optimal Regions of Homozygosity (ROH) size cut-offs for prediction of morbidity, based on 13 483 Chromosomal Microarray Analyses (CMA). Receiver operating characteristic (ROC) curves were generated, and area under the curve (AUC) was used to assess the predictive capability of total ROH percentage (TRPS), ROH number and ROH segment size in distinguishing between healthy (n=6,196) and affected (n=6,839) cohorts. The metrics were examined for telomeric and interstitial segments, distinct TRPS categories, and across different ancestral origins.

Implementing digital respiratory technologies for people with respiratory conditions: A protocol for a scoping review.

The value of 'data-enabled', digital healthcare is evolving rapidly, as demonstrated in the COVID-19 pandemic, and its successful implementation remains complex and challenging. Harmonisation (within/between healthcare systems) of infrastructure and implementation strategies has the potential to promote safe, equitable and accessible digital healthcare, but guidance for implementation is lacking. Using respiratory technologies as an example, our scoping review process will capture and review the published research between 12th December 2013 to 12th December 2023.

Long-term outcome of pediatric acute severe colitis: A prospective 5-year follow-up of the PRASCO trial.

Objectives: The PRASCO trial reported the short-term superiority of an antibiotic cocktail plus intravenous corticosteroids (IVCS) over IVCS alone in children with acute severe colitis (ASC). Here, we report the extension of the PRASCO trial and the long-term outcomes of the antibiotic cocktail in ASC.

Methods: This prospective follow-up of the PRASCO trial documented disease outcomes and treatments annually through 5 years.

Potential determinants and predictors of hypoparathyroidism after total thyroidectomy in pediatric patients.

Objective: Few studies have reported risk factors for post-thyroidectomy hypoparathyroidism (HPT) in children. We aimed to identify predicting factors for transient and permanent HPT after total thyroidectomy in pediatric patients.

Study Design: Retrospective review of medical charts.

A Variable Clinical Presentation of Hemoglobin City of Hope.

Hemoglobin City of Hope (Hb-COH), NC_000011.9(NM_000518.5):c.

Towards standardization of person-reported outcomes (PROs) in pediatric diabetes research: A consensus report.

Background: Diabetes ranks among the most common chronic conditions in childhood and adolescence. It is unique among chronic conditions, in that clinical outcomes are intimately tied to how the child or adolescent living with diabetes and their parents or carers react to and implement good clinical practice guidance. It is widely recognized that the individual's perspective about the impact of trying to manage the disease together with the burden of self-management should be addressed to achieve optimal health outcomes.

interspecies dissemination of IncM2-type carrying plasmid.

The gastrointestinal tract is a unique ecological niche with a high abundance of various Enterobacterales in close proximity. This allows the exchange of mobile genetic elements that carry resistance determinants. In the hospital setting, resistant organisms are prevalent and selective antibiotic pressure is high, providing a supportive platform for interspecies dissemination of resistance.

Fever duration enhanced biomarker sensitivity in diagnosing radiographically confirmed community-acquired pneumonia in children.

Aim: Our aim was to examine how fever duration affected the ability of biomarkers to diagnose community-acquired pneumonia (CAP).

Methods: This was a retrospective cohort study of children aged 2-18 years who attended the emergency department at Schneider Children's Medical Centre of Israel with CAP from June 2015 to May 2020. The children underwent biomarker measurements and chest radiographs and optimal biomarker thresholds were identified.

Antibiotic over-administration in hospitalized infants with Adenovirus infection.

Background: Infection with Adenovirus in children may clinically resemble a bacterial infection in several aspects, including high and prolonged fever, and increased inflammation markers. We aimed to estimate the rate of antibiotics administration among hospitalized infants with Adenovirus infection and to evaluate its justification.

Methods: Included were hospitalized patients aged 2 months - 2 years who found positive for Adenovirus in PCR tests (Ct <36).

Cognitive and Neurobiological Correlates for Switching/Inhibition Moderate the Relations Between Word Reading and Reading Comprehension in Hebrew-Speaking Children: An fMRI Study.

The expanded Simple View of Reading model suggests language processing and word reading as contributors to reading comprehension and points at the participation of executive functions as supporting these abilities. Switching and inhibition are both executive functions (EF) contributing to reading, especially in languages with two writing systems-shallow and deep, such as Hebrew. Here, we aimed to determine the specific role of switching/inhibition both cognitively and neurobiologically in the Simple View of Reading model among 49 eight- to 12-year-old Hebrew-speaking children.

The circadian clock in inflammatory bowel diseases.

Inflammatory bowel diseases (IBDs) involve relapsing-remitting immune activation and inflammation. Both immune system activity and digestive tract function exhibit diurnal variations, regulated by the circadian clock. Disruption of this clock is linked to inflammation, suggesting that circadian regulators may play a pivotal role in the inflammatory process of IBD.