Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial.

Background: Due to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.

Methods: FIREFLY-1 investigated the efficacy (arm 1, n=77), safety, and tolerability (arms 1/2) of tovorafenib (420 mg/m2 once weekly; 600 mg maximum) in patients with BRAF-altered relapsed/refractory pediatric low-grade glioma (pLGG).

Incidence of Pediatric Urinary Tract Infections Before and During the COVID-19 Pandemic.

Importance: Urinary tract infection (UTI) is common in children, but the population incidence is largely unknown. Controversy surrounds the optimal diagnostic criteria and how to balance the risks of undertreatment and overtreatment. Changes in health care use during the COVID-19 pandemic created a natural experiment to examine health care use and UTI diagnosis and outcomes.

NFKB2 haploinsufficiency identified via screening for IFN-α2 autoantibodies in children and adolescents hospitalized with SARS-CoV-2-related complications.

Background: Autoantibodies against type I IFNs occur in approximately 10% of adults with life-threatening coronavirus disease 2019 (COVID-19). The frequency of anti-IFN autoantibodies in children with severe sequelae of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection is unknown.

Objective: We quantified anti-type I IFN autoantibodies in a multicenter cohort of children with severe COVID-19, multisystem inflammatory syndrome in children (MIS-C), and mild SARS-CoV-2 infections.

Diagnostic testing for and detection of physical abuse in infants with brief resolved unexplained events.

Background: A Brief Resolved Unexplained Event (BRUE) can be a sign of occult physical abuse.

Objectives: To identify rates of diagnostic testing able to detect physical abuse (head imaging, skeletal survey, and liver transaminases) at BRUE presentation. The secondary objective was to estimate the rate of physical abuse diagnosed at initial BRUE presentation through 1 year of age.

HEROIC Trials to Answer Pragmatic Questions for Hospitalized Children.

Although the number of randomized controlled trials (RCTs) published each year involving adult populations is steadily rising, the annual number of RCTs published involving pediatric populations has not changed since 2005. Barriers to the broader utilization of RCTs in pediatrics include a lower prevalence of disease, less available funding, and more complicated regulatory requirements. Although child health researchers have been successful in overcoming these barriers for isolated diseases such as pediatric cancer, common pediatric diseases are underrepresented in RCTs relative to their burden.

Patent foramen ovale in children: Unique pediatric challenges and lessons learned from adult literature.

A patent foramen ovale (PFO) is a frequent incidental finding during echocardiography in otherwise healthy children. In most healthy children with a diagnosis of isolated incidental PFO, no further follow-up or intervention is necessary. In some children, PFO is associated with certain clinical syndromes such as cryptogenic stroke, decompression sickness, migraine, and platypnea-orthodeoxia syndrome.

Cumulative In-Hospital Costs Associated With Single-Ventricle Palliation.

Background: In the SVR (Single Ventricle Reconstruction) Trial, 1-year survival in recipients of right ventricle to pulmonary artery shunts (RVPAS) was superior to that in those receiving modified Blalock-Taussig-Thomas shunts (MBTTS), but not in subsequent follow-up. Cost analysis is an expedient means of evaluating value and morbidity.

Objectives: The purpose of this study was to evaluate differences in cumulative hospital costs between RVPAS and MBTTS.

Accuracy of Diagnostic Codes for Identifying Brief Resolved Unexplained Events.

Objectives: To evaluate (ICD-10) coding strategies for the identification of patients with a brief resolved unexplained event (BRUE).

Methods: Multicenter retrospective cohort study, including patients aged <1 year with an emergency department (ED) visit between October 1, 2015, and September 30, 2018, and an ICD-10 code for the following: (1) BRUE; (2) characteristics of BRUE; (3) serious underlying diagnoses presenting as a BRUE; and (4) nonserious diagnoses presenting as a BRUE. Sixteen algorithms were developed by using various combinations of these 4 groups of ICD-10 codes.

Risk Factors and Outcomes After a Brief Resolved Unexplained Event: A Multicenter Study.

Background: The accuracy of the risk criteria for brief resolved unexplained events (BRUEs) from the American Academy of Pediatrics (AAP) is unknown. We sought to evaluate if AAP risk criteria and event characteristics predict BRUE outcomes.

Methods: This retrospective cohort included infants <1 year of age evaluated in the emergency departments (EDs) of 15 pediatric and community hospitals for a BRUE between October 1, 2015, and September 30, 2018.

Nonfasted Liver Stiffness Correlates with Liver Disease Parameters and Portal Hypertension in Pediatric Cholestatic Liver Disease.

Elastographic measurement of liver stiffness is of growing importance in the assessment of liver disease. Pediatric experiences with this technique are primarily single center and limited in scope. The Childhood Liver Disease Research Network provided a unique opportunity to assess elastography in a well-characterized multi-institutional cohort.

The Nerve to Thyrohyoid Muscle as a Novel Donor Nerve for Laryngeal Reinnervation.

Objectives: Recurrent laryngeal nerve (RLN) injury may be a consequence of surgical procedures of the skull base, neck, and chest, with adverse consequences to function and quality of life. Laryngeal reinnervation offers a potentially stable improvement in vocal fold position and tone. The classic donor nerve is the ansa cervicalis, but is not always available due to damage or sacrifice during previous neck surgeries.

Variability in antimicrobial use in pediatric ventilator-associated events.

Objective: To assess variability in antimicrobial use and associations with infection testing in pediatric ventilator-associated events (VAEs).

Design: Descriptive retrospective cohort with nested case-control study.

Setting: Pediatric intensive care units (PICUs), cardiac intensive care units (CICUs), and neonatal intensive care units (NICUs) in 6 US hospitals.

Hypertonic Saline in Bronchiolitis and Type I Error: A Trial Sequential Analysis.

Unlabelled: : media-1vid110.1542/5799873875001PEDS-VA_2018-1144 BACKGROUND AND OBJECTIVES: Meta-analyses of nebulized hypertonic saline (HS) for acute viral bronchiolitis have yielded disparate conclusions. Trial sequential analysis (TSA) is a novel method designed to account for potential sources of error in conventional meta-analysis.

Platelet Inhibition in Shunted Infants on Aspirin at Short and Midterm Follow-Up.

There are few data to guide aspirin therapy to prevent shunt thrombosis in infants. We aimed to determine if aspirin administered at conventional dosing in shunted infants resulted in ≥50% arachidonic acid (AA) inhibition in short and midterm follow-up using thromboelastography with platelet mapping (TEG-PM) and to describe bleeding and thrombotic events during follow-up. We performed a prospective observational study of infants on aspirin following Norwood procedure, aortopulmonary shunt alone, or cavopulmonary shunt surgery.

Gene-Specific Therapy With Mexiletine Reduces Arrhythmic Events in Patients With Long QT Syndrome Type 3.

Background: Long QT syndrome type 3 (LQT3) is a lethal disease caused by gain-of-function mutations in the SCN5A gene, coding for the alpha-subunit of the sodium channel NaV1.5. Mexiletine is used to block late sodium current and to shorten QT interval in LQT3 patients.