Prognostic impact of cooccurring mutations in FLT3-ITD pediatric acute myeloid leukemia.

We sought to define the cooccurring mutational profile of FLT3-ITD-positive (ITDpos) acute myeloid leukemia (AML) in pediatric and young adult patients and to define the prognostic impact of cooperating mutations. We identified 464 patients with FLT3-ITD mutations treated on Children's Oncology Group trials with available sequencing and outcome data. Overall survival, event-free survival (EFS), and relapse risk were determined according to the presence of cooccurring risk stratifying mutations.

Rationale and design of the Children's Oncology Group study AAML1831 integrated cardiac substudies in pediatric acute myeloid leukemia therapy.

Background: Pediatric acute myeloid leukemia (AML) therapy is associated with substantial short- and long-term treatment-related cardiotoxicity mainly due to high-dose anthracycline exposure. Early left ventricular systolic dysfunction (LVSD) compromises anthracycline delivery and is associated with inferior event-free and overall survival in pediatric AML. Thus, effective cardioprotective strategies and cardiotoxicity risk predictors are critical to optimize cancer therapy delivery and enable early interventions to prevent progressive LVSD.

Chest compressions for pediatric organized rhythms: A hemodynamic and outcomes analysis.

Aim: Pediatric cardiopulmonary resuscitation (CPR) guidelines recommend starting CPR for heart rates (HRs) less than 60 beats per minute (bpm) with poor perfusion. Objectives were to (1) compare HRs and arterial blood pressures (BPs) prior to CPR among patients with clinician-reported bradycardia with poor perfusion ("BRADY") vs. pulseless electrical activity (PEA); and (2) determine if hemodynamics prior to CPR are associated with outcomes.

Fifteen Years of Spinal Fusion Outcomes in Children With Cerebral Palsy: Are We Getting Better?

Study Design: Retrospective multicenter study.

Objective: We reviewed 15-year trends in operative factors, radiographic and quality of life outcomes, and complication rates in children with cerebral palsy (CP)-related scoliosis who underwent spinal fusion.

Summary Of Background Data: Over the past two decades, significant efforts have been made to decrease complications and improve outcomes of this population.

Derivation of paediatric blood pressure percentiles from electronic health records.

Background: Identification of abnormal blood pressure (BP) in children requires normative data. We sought to examine the feasibility of using "real-world" office BP data obtained from electronic health records (EHR) to generate age-, sex- and height-specific BP percentiles for children.

Methods: Using data collected 01/01/2009-8/31/2021 from eight large children's healthcare organisations in PEDSnet, we applied a mixed-effects polynomial regression model with random slopes to generate Z-scores and BP percentiles and compared them with currently used normative BP distributions published in the 2017 American Academy of Paediatrics (AAP) Clinical Practise Guidelines (CPG).

Rehydration Rates and Outcomes in Overweight Children With Diabetic Ketoacidosis.

Background And Objectives: The Pediatric Emergency Care Applied Research Network Fluid Therapies Under Investigation in Diabetic Ketoacidosis (DKA) (FLUID) Trial found that rapid fluid infusion does not increase the risk of cerebral injury. Concern persists, however, whether fluid rates should be adjusted for overweight or obese patients. We used the FLUID Trial database to evaluate associations between fluid infusion rate and outcomes in these patients.

The Effect of Udenafil on Heart Rate and Blood Pressure in Adolescents With the Fontan Circulation.

The Fontan Udenafil Exercise Longitudinal (FUEL) trial showed that treatment with udenafil was associated with improved exercise performance at the ventilatory anaerobic threshold in children with Fontan physiology. However, it is not known how the initiation of phosphodiesterase 5 inhibitor therapy affects heart rate and blood pressure in this population. These data may help inform patient selection and monitoring after the initiation of udenafil therapy.

School age and adolescent heart failure following the Norwood procedure.

Background: Heart failure results in significant morbidity and mortality for young children with hypoplastic left heart syndrome (HLHS) following the Norwood procedure. The trajectory in later childhood is not well described.

Methods: We studied the outcome into adolescence of participants enrolled in the Single Ventricle Reconstruction trial who underwent the Fontan procedure or survived to 6 years without having undergone Fontan procedure.

Improving the Procedure of Delivering Serious News: Impact of a Six-Month Curriculum for Second Year Pediatric Residents.

We implemented and studied a novel curriculum that combined role play, didactic education, and the use of a procedure card for asynchronous learning to improve second-year pediatric residents' skills in delivering serious news. Phase 1 established baseline performance with a self-efficacy survey and observed simulation delivering serious news. Phase 2 included directed education of participants with a validated communication skills training framework.

A Telemedicine Solution to Minimize Unnecessary Emergency Department Transfers for Low-acuity Pediatric Orthopaedic Patients: A Model for Cost Minimization.

Introduction: Unnecessary emergency department (ED) transfers represent a notable source of excess costs and misutilization of healthcare resources, particularly with management of acute pediatric musculoskeletal injuries. This study used institutional data to create a model investigating the expected costs of a formal peer-to-peer telemedicine intervention designed to triage pediatric orthopaedic transfers, which we hypothesized would decrease healthcare costs by minimizing unwarranted ED-to-ED transfers.

Methods: In this retrospective modeling analysis, 350 pediatric orthopaedic trauma patients transferred to two in-network referral hospitals from outside facilities were identified and stratified into three groups representing how patients theoretically optimally could have been treated.

Cognition, Academic Achievement, Adaptive Behavior, and Quality of Life in Child and Adolescent Boys with Klinefelter Syndrome.

Objective: Klinefelter syndrome (KS; 47, XXY), the most common sex chromosome aneuploidy in males, is characterized by testicular failure and testosterone deficiency as well as a variety of cognitive, social, and emotional challenges. In the current study, we aimed to clarify the cognitive-behavioral profile of peripubertal boys with KS using measures of cognition, academic achievement, adaptive behavior, and quality of life.

Method: We compared 47 boys with KS (7-16 years of age) with 55 performance IQ-matched boys without KS on measures of cognition (WISC-V), executive function (BRIEF-2), academic achievement (KTEA-3), adaptive behavior (Vineland-3), and quality of life (PROMIS).

A Comparison of Virtual Reality to Traditional Simulation in Health Professions Education: A Systematic Review.

With the increasing availability of virtual reality (VR) and its lower overall costs of use, the objective of this review was to compare VR to traditional simulation in terms of learning outcomes. Studies were included if they met the following criteria: ( a ) research study (of any design), ( b ) focused on learners in health professions, and ( c ) compared VR with traditional simulation. Studies were excluded for the following reasons: ( a ) not a research study, ( b ) focused on learners outside health professions, ( c ) used screen-based or computer-based simulation, ( d ) used a task trainer, and ( e ) did not involve a comparison of VR to traditional simulation.

Association of CPR simulation program characteristics with simulated and actual performance during paediatric in-hospital cardiac arrest.

Aim: To evaluate associations between characteristics of simulated point-of-care cardiopulmonary resuscitation (CPR) training with simulated and actual intensive care unit (ICU) CPR performance, and with outcomes of children after in-hospital cardiac arrest.

Methods: This is a pre-specified secondary analysis of the ICU-RESUScitation Project; a prospective, multicentre cluster randomized interventional trial conducted in 18 ICUs from October 2016-March 2021. Point-of-care bedside simulations with real-time feedback to allow multidisciplinary ICU staff to practice CPR on a portable manikin were performed and quality metrics (rate, depth, release velocity, chest compression fraction) were recorded.

Echocardiographic image collection and evaluation in infants with CHD: lessons learned from the imaging core lab for the Residual Lesion Score study.

Many factors affect patient outcome after congenital heart surgery, including the complexity of the heart disease, pre-operative status, patient specific factors (prematurity, nutritional status and/or presence of comorbid conditions or genetic syndromes), and post-operative residual lesions. The esidual esion core is a novel tool for assessing whether specific residual cardiac lesions after surgery have a measurable impact on outcome. The goal is to understand which residual lesions can be tolerated and which should be addressed prior to leaving the operating room.

Molecular Trojan Horses for treating lysosomal storage diseases.

Lysosomal storage diseases (LSDs) are caused by monogenic mutations in genes encoding for proteins related to the lysosomal function. Lysosome plays critical roles in molecule degradation and cell signaling through interplay with many other cell organelles, such as mitochondria, endoplasmic reticulum, and peroxisomes. Even though several strategies (i.

Natural History of Pediatric Hand and Wrist Ganglion Cysts: Longitudinal Follow-Up of a Prospective, Dual-Center Cohort.

Purpose: This investigation describes the outcomes of pediatric ganglion cysts in a prospective cohort that elected not to undergo cyst aspiration or surgical treatment. Our primary aim was to investigate the rate of spontaneous resolution over time among the subset of patients who did not undergo specific treatments.

Methods: Children (aged ≤18 years) who presented to the clinic with ganglion cysts of the hand or wrist were enrolled in a prospective two-center registry between 2017 and 2021.

Echocardiographic Assessment of Pulmonary Arterial Hypertension Following Inhaled Nitric Oxide in Infants with Severe Bronchopulmonary Dysplasia.

Objectives: Inhaled nitric oxide (iNO) is an effective pulmonary vasodilator. However, the efficacy of iNO in former premature infants with established bronchopulmonary dysplasia (BPD) has not been studied. This study aimed to determine the efficacy of iNO in reducing pulmonary artery pressure in infants with severe BPD as measured by echocardiography.

Intraoperative neuromonitoring predicts postoperative deficits in severe pediatric spinal deformity patients.

Purpose: To evaluate intraoperative monitoring (IOM) alerts and neurologic deficits during severe pediatric spinal deformity surgery.

Methods: Patients with a minimum Cobb angle of 100° in any plane or a scheduled vertebral column resection (VCR) with minimum 2-year follow-up were prospectively evaluated (n = 243). Preoperative, immediate postoperative, and 2-year postoperative neurologic status were reported.

HNRNPC haploinsufficiency affects alternative splicing of intellectual disability-associated genes and causes a neurodevelopmental disorder.

Heterogeneous nuclear ribonucleoprotein C (HNRNPC) is an essential, ubiquitously abundant protein involved in mRNA processing. Genetic variants in other members of the HNRNP family have been associated with neurodevelopmental disorders. Here, we describe 13 individuals with global developmental delay, intellectual disability, behavioral abnormalities, and subtle facial dysmorphology with heterozygous HNRNPC germline variants.

Current Patterns of Probiotic Use in U.S. Neonatal Intensive Care Units: A Multi-Institution Survey.

Objective: Probiotic supplementation is associated with health benefits in preterm infants. The 2021 American Academy of Pediatrics (AAP) statement on probiotic use advised caution, citing heterogeneity and absence of federal regulation. We assessed the impact of the AAP statement and current institution-wide patterns of probiotic use across neonatal intensive care units (NICU) across the United States.

Clinical, neuroradiological, and molecular characterization of mitochondrial threonyl-tRNA-synthetase (TARS2)-related disorder.

Purpose: Biallelic variants in TARS2, encoding the mitochondrial threonyl-tRNA-synthetase, have been reported in a small group of individuals displaying a neurodevelopmental phenotype but with limited neuroradiological data and insufficient evidence for causality of the variants.

Methods: Exome or genome sequencing was carried out in 15 families. Clinical and neuroradiological evaluation was performed for all affected individuals, including review of 10 previously reported individuals.

More Than a Headache: Lived Experience of Migraine in Youth.

Background: Migraine is the leading cause of disability among adolescents and young adults. We aimed to characterize the impact of migraine on the experience of children, adolescents, and caregivers.

Methods: This descriptive qualitative study recruited youth aged four to 18 years with migraine and their caregivers from the multicenter, prospective Pediatric Migraine Registry between 2020 and 2021.