Sweat chloride reflects CFTR function and correlates with clinical outcomes following CFTR modulator treatment.

Background: Highly effective CFTR modulators improve CFTR function and lead to dramatic improvements in health outcomes in many people with cystic fibrosis (pwCF). The relationship between measures of CFTR function, such as sweat chloride concentration, and clinical outcomes in pwCF treated with CFTR modulators is poorly defined. We conducted analyses to better understand the relationships between sweat chloride and CFTR function in vitro, and between sweat chloride and clinical outcomes following CFTR modulator treatment.

Efficacy and safety of LAU-7b in a Phase 2 trial in adults with cystic fibrosis.

Background: Lung inflammation is associated with tissue damage in cystic fibrosis (CF). LAU-7b, a novel oral drug candidate, was shown to control inflammation and stabilize CFTR protein in the epithelial membrane during inflammatory stress in preclinical models of CF.

Methods: A double-blind, randomized, placebo-controlled Phase 2 study was conducted to evaluate efficacy and safety of LAU-7b in adults with CF.

Inpatient allergy delabeling of pediatric patients with low-risk penicillin allergy status through direct oral amoxicillin challenge.

Less than 5% of children who report penicillin allergy have clinically pertinent type 1 immunoglobulin E mediated hypersensitivity reaction by using direct oral amoxicillin challenge. Several pathways have been developed to delabel penicillin allergy by using direct oral amoxicillin challenge, mostly in the outpatient settings, but there is relative scarcity on published outcomes of these pathways, especially in the inpatient pediatric settings. This study aimed to evaluate the performance of an institutionally derived inpatient penicillin allergy screening tool.

A Women's health perspective on managing obesity.

While the prevalence of obesity in US men and women is nearly equivalent, obesity management in women requires a different approach that considers age and life stage in development including sexual maturation/reproduction, menopause and post-menopause. In this review, the diagnosis and treatment of obesity using lifestyle modification, pharmacotherapy and metabolic and bariatric surgery are discussed from a women's health perspective, with emphasis on management during pregnancy and post-partum.

Initial Insights into the Genetic Variation Associated with Metformin Treatment Failure in Youth with Type 2 Diabetes.

Metformin is the first-line treatment for type 2 diabetes (T2D) in youth but with limited sustained glycemic response. To identify common variants associated with metformin response, we used a genome-wide approach in 506 youth from the Treatment Options for Type 2 Diabetes in Adolescents and Youth (TODAY) study and examined the relationship between T2D partitioned polygenic scores (pPS), glycemic traits, and metformin response in these youth. Several variants met a suggestive threshold ( < 1 × 10), though none including published adult variants reached genome-wide significance.

Global PARITY: Study Design for a Multi-Centered, International Point Prevalence Study to Estimate the Burden of Pediatric Acute Critical Illness in Resource-Limited Settings.

Background: The burden of pediatric critical illness and resource utilization by children with critical illness in resource limited settings (RLS) are largely unknown. Without specific data that captures key aspects of critical illness, disease presentation, and resource utilization for pediatric populations in RLS, development of a contextual framework for appropriate, evidence-based interventions to guide allocation of limited but available resources is challenging. We present this methods paper which describes our efforts to determine the prevalence, etiology, hospital outcomes, and resource utilization associated with pediatric acute, critical illness in RLS globally.

Understanding Breastfeeding Barriers at an Urban Pediatric Practice.

Breastfeeding is the optimal nutrition for infants given the numerous health benefits that are conferred on mothers, infants, and society in a dose-dependent manner. However, low breastfeeding rates and racial breastfeeding inequities persist for the African American (AA) community due to historic structural racism. The issue is especially salient at the Rainbow Center for Women and Children, an urban health center in Cleveland, Ohio where approximately 90% of their mothers are AA, WIC-eligible, and publicly insured.

Pediatric Diabetic Retinopathy: Updates in Prevalence, Risk Factors, Screening, and Management.

Purpose Of Review: Diabetic retinopathy (DR) is a microvascular complication of diabetes mellitus and a major cause of vision loss worldwide. The purpose of this review is to provide an update on the prevalence of diabetic retinopathy in youth, discuss risk factors, and review recent advances in diabetic retinopathy screening.

Recent Findings: While DR has long been considered a microvascular complication, recent data suggests that retinal neurodegeneration may precede the vascular changes associated with DR.

Long-term amikacin liposome inhalation suspension in cystic fibrosis patients with chronic P. aeruginosa infection.

Background: . In CLEAR-108-a phase 3, randomised, open-label study-once-daily amikacin liposome inhalation suspension (ALIS) was noninferior to twice-daily tobramycin inhalation solution (TIS) in improving lung function in patients with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa infection after 3 treatment cycles (28 days on/28 days off). The CLEAR-110 extension study (ClinicalTrials.

Long-Term Impact of Ivacaftor on Healthcare Resource Utilization Among People with Cystic Fibrosis in the United States.

Introduction: Ivacaftor was first approved in 2012 for the treatment of a select population of individuals with cystic fibrosis (CF), a rare, life-shortening genetic disease. Reductions in healthcare resource utilization (HCRU) associated with ivacaftor have been observed during limited follow-up and for selected outcomes in real-world studies. This study aimed to further describe the long-term impact of ivacaftor treatment on multiple measures of HCRU among people with CF (pwCF).

Sex Differences in the Outcomes of Mild Traumatic Brain Injury in Children Presenting to the Emergency Department.

Sex differences after concussion have been studied largely in high school and college athletes, often without reference to comparison groups without concussion. This study sought to evaluate sex differences in outcomes among all children and adolescents presenting to the Emergency Department (ED) for either mild traumatic brain injury (TBI) or orthopedic injury (OI), regardless of mechanism of injury. The study involved a concurrent cohort, prospective study design with longitudinal follow-up.

The First Genome-Wide Association Study for Type 2 Diabetes in Youth: The Progress in Diabetes Genetics in Youth (ProDiGY) Consortium.

The prevalence of type 2 diabetes in youth has increased substantially, yet the genetic underpinnings remain largely unexplored. To identify genetic variants predisposing to youth-onset type 2 diabetes, we formed ProDiGY, a multiethnic collaboration of three studies (TODAY, SEARCH, and T2D-GENES) with 3,006 youth case subjects with type 2 diabetes (mean age 15.1 ± 2.

Assessment of White Matter Integrity after Pediatric Traumatic Brain Injury.

White matter (WM) abnormalities, such as atrophy and hyperintensities (WMH), can be accessed via magnetic resonance imaging (MRI) after pediatric traumatic brain injury (TBI). Several methods are available to classify WM abnormalities (i.e.

Narrative Performance Level Assignments at Initial Entrustment and Graduation: Integrating EPAs and Milestones to Improve Learner Assessment.

Purpose: To determine which narrative performance level for each general pediatrics entrustable professional activity (EPA) reflects the minimum level clinical competency committees (CCCs) felt should be associated with graduation as well as initial entrustment and compare expected narrative performance levels (ENPLs) for each EPA with actual narrative performance levels (ANPLs) assigned to residents at initial entrustment.

Method: A series of 5 narratives, corresponding to the 5 milestone performance levels, were developed for each of the 17 general pediatrics EPAs. In academic year (AY) 2015-2016, the CCCs at 22 Association of Pediatric Program Directors Longitudinal Educational Assessment Research Network member sites reported ENPLs for initial entrustment and at time of graduation.

A National Approach to Pediatric Sepsis Surveillance.

Pediatric sepsis is a major public health concern, and robust surveillance tools are needed to characterize its incidence, outcomes, and trends. The increasing use of electronic health records (EHRs) in the United States creates an opportunity to conduct reliable, pragmatic, and generalizable population-level surveillance using routinely collected clinical data rather than administrative claims or resource-intensive chart review. In 2015, the US Centers for Disease Control and Prevention recruited sepsis investigators and representatives of key professional societies to develop an approach to adult sepsis surveillance using clinical data recorded in EHRs.

Parenting styles as a predictor of long-term psychosocial outcomes after traumatic brain injury (TBI) in early childhood.

This study sought to determine whether parenting styles predict long-term psychosocial outcomes after traumatic brain injury in young children. The study involved a concurrent cohort, prospective design, with longitudinal assessments up to early adolescence. Participants included 126 children with moderate to severe traumatic brain injury or orthopedic injury, ages 3 to 6 years 11 months, recruited between 2003 and 2006.

Home Oxygen and 2-Year Outcomes of Preterm Infants With Bronchopulmonary Dysplasia.

Objectives: To compare medical and developmental outcomes over the first 2 years of life in extremely preterm infants with bronchopulmonary dysplasia (BPD) who were discharged on supplemental oxygen via nasal cannula with outcomes of infants with a similar severity of respiratory illness who were discharged breathing in room air.

Methods: We performed a propensity score-matched cohort study. Eligible infants were born at <27 weeks' gestation, were receiving supplemental oxygen or respiratory support at 36 weeks' postmenstrual age, and were assessed at 18 to 26 months' corrected age.

Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor/ivacaftor.

Background: Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. However, long-term survival benefits of lumacaftor/ivacaftor (LUM/IVA) cannot yet be quantified. Simulation models can provide predictions about long-term health outcomes.

A Comparison of 2 Online Parent Skills Training Interventions for Early Childhood Brain Injury: Improvements in Internalizing and Executive Function Behaviors.

Objective: To examine the effectiveness of a web-based parenting intervention (Internet-Based Interacting Together Everyday: Recovery After Childhood TBI [I-InTERACT]) and an abbreviated version (Express) in reducing executive dysfunction and internalizing problems among young children following traumatic brain injury (TBI).

Method: Parents of 113 children (ages 3-9 years) who had sustained a TBI were randomized to 1 of 3 treatment groups: I-InTERACT, Express, or an Internet Resource Comparison (IRC) group. Parents who participated in either I-InTERACT or Express completed self-guided web sessions and received live coaching of their parenting skills via videoconferencing with a therapist.

A quantitative method for the radiological assessment of skeletal maturity using the distal femur.

Aims: Using 90% of final height as a benchmark, we sought to develop a quick, quantitative and reproducible method of estimating skeletal maturity based on topographical changes in the distal femoral physis.

Patients And Methods: Serial radiographs of the distal femoral physis three years prior to, during, and two years following the chronological age associated with 90% of final height were analyzed in 81 healthy children. The distance from the tip of the central peak of the distal femoral physis to a line drawn across the physis was normalized to the physeal width.