RASopathies.

RASopathies are a group of disorders characterized by mutations in the RAS-MAPK pathway. RAS-MAP signaling plays a critical role in cell differentiation, proliferation, and survival. Germline mutations can result in distinctive syndromes, including Noonan syndrome, Costello syndrome, and neurofibromatosis type 1.

Interprofessional ECMO telerounding: a novel approach to neonatal ECMO clinical participation and education.

Objective: Determine the feasibility, strengths, and barriers of offering extracorporeal membrane oxygenation (ECMO) telerounding to neonatal intensive care unit (NICU) care providers.

Study Design: NICU providers were invited to join ECMO rounds by teleconference. Data were collected on telerounding participation and ECMO concepts discussed.

Pediatric Emergency Medicine Simulation Curriculum: Bacterial Tracheitis.

Introduction: Pediatric bacterial tracheitis is a rare but life-threatening upper airway infection with mortality rates estimated as high as 20%, typically affecting children between 6 months and 12 years old. Given such high mortality rates, we felt it was important to train medical personnel to evaluate and manage this condition.

Methods: This simulation-based curriculum was developed for health care professionals involving the evaluation and management of an 8-year-old male with symptoms of fever, stridor, worsening barking cough, and increased work of breathing.

Concussion Symptom Treatment and Education Program: A Feasibility Study.

Objective: To evaluate the feasibility and potential benefits of a manualized, brief cognitive-behavioral therapy-based intervention program for children and adolescents with persistent postconcussive symptoms.

Setting: Two outpatient pediatric concussion programs in the United States.

Participants: Patients aged 8 to 17 years who sustained concussions between 2 and 12 months prior to enrollment.

Pediatric Emergency Medicine Didactics and Simulation (PEMDAS): Serotonin Syndrome.

Introduction: Serotonin syndrome is caused by an accumulation of serotonin in the body from drug interactions or overdose of serotonergic medications, including commonly used antidepressants. Symptoms can be life-threatening and encompass both neurologic and cardiovascular toxicity, including agitation, seizure, tachycardia, rhabdomyolysis, and hyperthermia.

Methods: This simulation case was developed for pediatric emergency medicine fellows and emergency medicine residents in the pediatric emergency department and can be altered to accommodate other learners.

Pathological response in children and adults with large unresected intermediate-grade or high-grade soft tissue sarcoma receiving preoperative chemoradiotherapy with or without pazopanib (ARST1321): a multicentre, randomised, open-label, phase 2 trial.

Background: Outcomes for children and adults with advanced soft tissue sarcoma are poor with traditional therapy. We investigated whether the addition of pazopanib to preoperative chemoradiotherapy would improve pathological near complete response rate compared with chemoradiotherapy alone.

Methods: In this joint Children's Oncology Group and NRG Oncology multicentre, randomised, open-label, phase 2 trial, we enrolled eligible adults (aged ≥18 years) and children (aged between 2 and <18 years) from 57 hospitals in the USA and Canada with unresected, newly diagnosed trunk or extremity chemotherapy-sensitive soft tissue sarcoma, which were larger than 5 cm in diameter and of intermediate or high grade.

Educational Perspectives: Educational Strategies to Improve Outcomes from Neonatal Resuscitation.

Since 1987, the Neonatal Resuscitation Program (NRP) course has taught the cognitive, technical, and behavioral skills required to effectively resuscitate newborns. To remain relevant and effective, the NRP course needs to continually evolve and embrace evidence-based educational strategies proven to improve outcomes from resuscitation. In this Educational Perspectives article, 6 educational strategies that can be applied to neonatal resuscitation education are reviewed.

Forehead location and large segmental pattern of facial port-wine stains predict risk of Sturge-Weber syndrome.

Background: Children with forehead port-wine stains (PWSs) are at risk of Sturge-Weber syndrome (SWS). However, most will not develop neurologic manifestations.

Objective: To identify children at greatest risk of SWS.

Office-Based Screening for Sexually Transmitted Infections in Adolescents.

Almost 1 in 4 adolescents have a sexually transmitted infection (STI). These infections are preventable through safe sexual practices and routine screening. Pediatricians are the first line of clinical care for adolescents and are well positioned to offer sexual and reproductive health care counseling and services to their patients; yet, there is a paucity of sexual health screening provided at routine health supervision visits.

Editorial: Research in Real-World Settings: Challenging the Limits of Experimental Design.

As underscored by Ponguta and colleagues in this issue of the Journal, displacement secondary to war, poverty, and unsafe living conditions has affected large segments of the world's population, placing millions of children and their parents in highly stressful living conditions. Positive parenting, parenting that is warm, loving, and responsive to a child's needs, can mitigate the negative effects of early adversity and poverty on a child's behavioral, emotional, and cognitive development and is one factor that can be targeted in otherwise almost immutable circumstances. Programs to support parents and to enhance positive parenting skills have proved effective across multiple countries and environments, including low- and middle-income countries; however, efforts to rigorously test these programs in areas of highest need and psychosocial instability are rare.

Echocardiographic surveillance in children after tetralogy of Fallot repair: Adherence to guidelines?

Background: Longitudinal clinical surveillance by transthoracic echocardiography (TTE) is an established practice in children with repaired tetralogy of Fallot (TOF). Non-Invasive Imaging Guidelines recommends a list of reporting elements that should be addressed during routine TTE in this population. In this study, we assessed the adherence to these recommendations.

Initial treatment of pediatric differentiated thyroid cancer: a review of the current risk-adaptive approach.

Differentiated thyroid cancer in children is a rare disease, accounting for only 1.4% of all pediatric malignancies. The diagnosis, biological behavior and treatment of differentiated thyroid cancer in children is different from that in adults.

Transcranial Doppler Ultrasound During Critical Illness in Children: Survey of Practices in Pediatric Neurocritical Care Centers.

Objectives: The scope of transcranial Doppler ultrasound in the practice of pediatric neurocritical care is unknown. We have surveyed pediatric neurocritical care centers on their use of transcranial Doppler and analyzed clinical management practices.

Design: Electronic-mail recruitment with survey of expert centers using web-based questionnaire.

Neurodevelopmental Outcomes in Preschool and School Aged Children With Biliary Atresia and Their Native Liver.

Objectives: The aim of the study was to assess neurodevelopmental outcomes among children with biliary atresia (BA) surviving with their native liver at ages 3 to 12 years and evaluate variables that associate with neurodevelopment.

Methods: Participants (ages 3-12 years) in a prospective, longitudinal, multicenter study underwent neurodevelopmental testing with Weschler Preschool and Primary Scale of Intelligence, 3rd edition (WPPSI-III, ages 3-5 years) and Weschler Intelligence Scale for Children, 4th edition (WISC-IV, ages 6-12 years). Continuous scores were analyzed using Kolmogorov-Smironov tests compared with a normal distribution (mean = 100 ± 15).

Safety and Effectiveness of Adalimumab in Patients With Polyarticular Course of Juvenile Idiopathic Arthritis: STRIVE Registry Seven-Year Interim Results.

Objective: To evaluate safety and effectiveness of adalimumab (ADA) in polyarticular-course juvenile idiopathic arthritis (JIA) in the STRIVE registry.

Methods: STRIVE enrolled patients with polyarticular-course JIA into 2 arms based on treatment with methotrexate (MTX) alone or ADA with/without MTX (ADA ± MTX). Adverse events (AEs) per 100 patient-years of observation time were analyzed by registry arm.

Apgar score of 0 at 10 min and survival to 1 year of age: a retrospective cohort study in Washington state.

Objective: To determine 1-year survival in a cohort of newborns with an Apgar score of 0 at 5 and 10 min of age.

Study Design: A retrospective cohort study of the Washington State Comprehensive Hospital Abstract Reporting System from 2005 to 2014.

Results: Of 879,340 births, 199 (0.

Dermatologic care of sexual and gender minority/LGBTQIA youth, Part 2: Recognition and management of the unique dermatologic needs of SGM adolescents.

Sexual and gender minority (SGM) individuals, including lesbian, gay, bisexual, transgender/gender diverse, questioning/queer, intersex, and asexual (LGBTQIA) persons, represent a historically underserved population within the field of medicine, though their unique health needs are increasingly recognized. Part 2 of this two-part review will address unique concerns regarding acne, tanning behavior, sexually transmitted infections, and other health disparities among SGM adolescents. A more comprehensive understanding of the dermatologic needs of SGM youth will better allow pediatric dermatologists to actively and compassionately care for this health disparity population.

Dermatologic care of sexual and gender minority/LGBTQIA youth, Part I: An update for the dermatologist on providing inclusive care.

Sexual and gender minority (SGM) persons, including lesbian, gay, bisexual, transgender/gender diverse, questioning/queer, intersex, and asexual (LGBTQIA) individuals, represent a historically underserved population within the field of medicine, though their unique health needs are increasingly recognized. Unfortunately, our understanding of these needs as they relate to dermatology is still nascent, particularly with respect to children and adolescents. This two-part review will discuss the dermatologic care of SGM youth, with Part 1 providing practical advice for dermatologists seeking to provide more culturally mindful and accessible care for SGM children and adolescents.

Sofosbuvir and Ribavirin Therapy for Children Aged 3 to <12 Years With Hepatitis C Virus Genotype 2 or 3 Infection.

Currently, the only approved hepatitis C virus (HCV) treatment for children aged <12 years is pegylated interferon plus ribavirin. In an open-label study, we evaluated the safety and efficacy of sofosbuvir plus ribavirin for 12 weeks in children aged 3 to <12 years chronically infected with genotype 2 or for 24 weeks in patients with genotype 3. Patients aged 3 to <6 years weighing <17 kg received sofosbuvir 150 mg, and patients aged 3 to <6 years weighing ≥17 kg and all patients aged 6 to <12 years received sofosbuvir 200 mg once daily.

Ledipasvir-Sofosbuvir for 12 Weeks in Children 3 to <6 Years Old With Chronic Hepatitis C.

For children under 12 years of age who have chronic hepatitis C virus (HCV) infection, there are currently no approved treatments with direct-acting antiviral agents. We therefore evaluated the safety and efficacy of ledipasvir-sofosbuvir in HCV-infected children aged 3 to <6 years. In an open-label study, patients 3 to <6 years old chronically infected with HCV genotype 1 (n = 33) or 4 (n = 1) received weight-based doses of combined ledipasvir-sofosbuvir as granules (33.

Outcomes of low-intensity biopsy surveillance for rejection in paediatric cardiac transplantation.

Background: Significant inter-centre variability in the intensity of endomyocardial biopsy surveillance for rejection following paediatric cardiac transplantation has been reported. Our aim was to determine if low-intensity biopsy surveillance with two scheduled biopsies in the first year would produce outcomes similar to published registry outcomes.

Methods: A retrospective study of paediatric recipients transplanted between 2008 and 2014 using a low-intensity biopsy protocol consisting of two surveillance biopsies at 3 and 12-13 months in the first post-transplant year, then annually thereafter.