Factors Associated with Pediatric Drowning-Associated Lung Injury.

Objective: To identify risk factors for clinically-important drowning-associated lung injury (ciDALI) in children.

Study Design: This was a cross-sectional study of children (0 through18 years) who presented to 32 pediatric emergency departments (EDs) from 2010 through 2017. We reviewed demographics, comorbidities, prehospital data, chest radiographs reports, and ED course from emergency medical services, medical, and fatality records.

Utility of Aprepitant in the Management of Pediatric Patients with Cyclical Vomiting Syndrome.

: Cyclical vomiting syndrome (CVS) is a recurrent debilitating illness characterized by intense episodes of nausea and emesis with widely varied pharmacological management across the country. Aprepitant is now increasingly used in patients with CVS. The impact of aprepitant as an abortive therapy in the readmission of pediatric patients with CVS is currently unknown.

Insulin clearance at randomisation and in response to treatment in youth with type 2 diabetes: a secondary analysis of the TODAY randomised clinical trial.

Aims/hypothesis: Insulin resistance and compensatory hyperinsulinaemia are core features leading to beta cell failure in youth-onset type 2 diabetes. Insulin clearance (IC) is also a key regulator of insulin concentrations, but few data exist on IC in youth-onset type 2 diabetes. In a secondary analysis of our Treatment Options for Type 2 Diabetes in Adolescents and Youth (TODAY) randomised clinical trial, we investigated potential sex-, race-, ethnicity- and treatment-related differences in IC in youth-onset type 2 diabetes and aimed to identify metabolic phenotypes associated with IC at baseline and in response to metformin, metformin plus a lifestyle intervention, and metformin plus rosiglitazone.

The Psychosocial Burden of Pediatric Food Allergy in the United States: A Population-Based Survey.

Background: Food allergy (FA) affects approximately one in 12 US children, with prevalence increasing. Aside from considerable health care utilization, accumulating research suggests heightened psychosocial burden among this population.

Objective: To characterize FA-related psychosocial burden among a large, nationally representative pediatric sample, and its correlates, including sociodemographic characteristics, comorbid conditions, allergy severity, allergic symptoms, number and type of allergens, and healthcare utilization.

Supporting Children's Mental Health Needs in Disasters.

Public health emergencies, including climate-related and manmade disasters such as active shooter incidents, occur regularly in the United States. A comprehensive approach is needed to ensure that children's mental health needs are adequately addressed following disasters. This article summarizes the latest evidence on how health systems can effectively address children's unique developmental, social, emotional, and behavioral needs in the context of disasters.

Transitioning medically complex infants home: lessons learned from quality improvement efforts.

Objective: Decrease: 1) time from tracheostomy or gastrostomy tube placement to discharge home, and 2) avoidable readmissions within 7 days post-discharge, for medically complex infants.

Study Design: Five neonatology units and representatives from Medicaid and Managed Care Organizations participated. Measures included length of stay (LOS) from surgery to discharge, readmissions, and time from surgery to identifying home nursing.

BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.

Objectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.

Methods: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82).

Fundamentals of DNA methylation in development.

DNA methyation is critical to regulation of gene expression especially during developmentally dynamic changes. A large proportion occurs at CpG (a cytosine followed by a guanine nucleotide) sites and impacts gene expression based on location, timing and level of DNA methylation. The spectrum of effects produced by DNA methylation ranges from inhibition to enhancement of gene expression.

Characteristics of Young Children Associated with Diagnostic Utility of the Autism Diagnostic Observation Schedule: A DBPNet Study.

Objective: The aim of this study is to identify characteristics of children for whom a developmental-behavioral pediatrician's (DBP) diagnostic impressions of autism spectrum disorder (ASD) or non-ASD were changed by Autism Diagnostic Observation Schedule (ADOS) results.

Method: A prospective study of children 1½ to <6 years consecutively referred to 8 sites for possible ASD. Cognitive/developmental, language, and adaptive testing varied, as each site followed its usual clinical approach.

Complex Attention-Deficit Hyperactivity Disorder in a 4-Year-Old With Repaired Critical Congenital Heart Disease and Autism Spectrum Disorder.

Background: Congenital heart disease (CHD) is a risk factor for developmental delay and for attention-deficit hyperactivity disorder (ADHD). The Cardiac Neurodevelopmental Outcome Collaborative has developed recommendations for ongoing monitoring of this at-risk population to be able to detect developmental, learning, and behavioral concerns, as they become apparent as a child ages.

Case Presentation: A 4-year-old boy with tetralogy of Fallot with a ventricular septal defect repaired in infancy was followed periodically in the cardiac neurodevelopmental follow-up clinic and diagnosed with autism spectrum disorder as well as additional developmental and medical issues.

Challenges in institutional ethical review process and approval for international multicenter clinical studies in lower and middle-income countries: the case of PARITY study.

Background: One of the greatest challenges to conducting multicenter research studies in low and middle-income countries (LMICs) is the heterogeneity in regulatory processes across sites. Previous studies have reported variations in requirements with a lack of standardization in the Institutional Review Board (IRB) processes between centers, imposing barriers for approval, participation, and development of multicenter research.

Objectives: To describe the regulatory process, variability and challenges faced by pediatric researchers in LMICs during the IRB process of an international multicenter observational point prevalence study (Global PARITY).

Germline de novo alterations of RUNX1T1 in individuals with neurodevelopmental and congenital anomalies.

Runt-related transcription factor 1 translocated to 1 (RUNX1T1; also known as eight-twenty-one [ETO]) encodes a transcription regulator for hematopoietic genes and is well known for its involvement in hematologic malignancies, particularly acute myeloid leukemia (AML). However, its role in congenital disease is less understood. This study provides detailed clinical and molecular information on three cases exhibiting neurodevelopmental and congenital anomalies with germline de novo alterations in RUNX1T1.

Critical Data for Critical Care: A Primer on Leveraging Electronic Health Record Data for Research From Society of Critical Care Medicine's Panel on Data Sharing and Harmonization.

A growing body of critical care research draws on real-world data from electronic health records (EHRs). The bedside clinician has myriad data sources to aid in clinical decision-making, but the lack of data sharing and harmonization standards leaves much of this data out of reach for multi-institution critical care research. The Society of Critical Care Medicine (SCCM) Discovery Data Science Campaign convened a panel of critical care and data science experts to explore and document unique advantages and opportunities for leveraging EHR data in critical care research.

Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study.

Background: As people with cystic fibrosis (PWCF) live longer due to the breakthrough drug elexacaftor-tezacaftor-ivacaftor (ETI), they have questioned whether other CF therapies could be safely discontinued. SIMPLIFY was the first prospective, randomized trial to evaluate non-inferiority of discontinuing versus continuing two therapies. The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study was conducted to understand experiences of PWCF enrolled in SIMPLIFY, including why they joined, perceptions of randomization, decision-making around study withdrawal, and considerations for future discontinuation studies.

Trifolin attenuates hypertension-mediated cardiac injury by inhibiting cardiomyocyte apoptosis: Mechanistic insights and therapeutic potential.

Background: Hypertension-induced cardiac disease is a common complication and a significant contributor to mortality in hypertensive patients, largely due to cardiomyocyte apoptosis. Although Trifolin has been identified as a potential antihypertensive compound, its therapeutic role in hypertension-induced cardiac injury remains uncertain.

Purpose: This study aims to evaluate the protective effects of Trifolin and explore the underlying mechanisms of its action against hypertension-induced cardiac injury.