Risk Model Development and Validation for Prediction of Coronary Artery Aneurysms in Kawasaki Disease in a North American Population.

Background Accurate prediction of coronary artery aneurysms ( CAAs ) in patients with Kawasaki disease remains challenging in North American cohorts. We sought to develop and validate a risk model for CAA prediction. Methods and Results A binary outcome of CAA was defined as left anterior descending or right coronary artery Z score ≥2.

Epicardial Echocardiography in Pediatric and Congenital Heart Surgery.

Epicardial echocardiography (e-echo) is a useful approach to intraoperative imaging for the smallest patients and in those with contraindications to transesophageal echocardiography (TEE). The e-echo has additional advantages that include improved visualization of anterior and vascular structures and three-dimensional capabilities. In this review, we describe the advantages and disadvantages of e-echo versus TEE for pediatric and congenital heart surgery with the goal of enhancing the utility and feasibility of the former among congenital heart surgeons.

Will Teens Go Red? Low Cardiovascular Disease Awareness Among Young Women.

Background The American Heart Association Go Red for Women campaign has improved awareness of cardiovascular disease ( CVD ) among adult women aged 25 years and older. Little is known about awareness among younger women. Methods and Results We assessed awareness of CVD and prevention efforts among 331 young women aged 15 to 24 years using the American Heart Association National Women's Health Study survey.

Ascending Aorta Size at Birth Predicts White Matter Microstructure in Adolescents Who Underwent Fontan Palliation.

Background In neonates with single ventricle, smaller ascending aorta diameter is associated with cerebral white matter ( WM ) microstructural abnormalities. We sought to determine whether this association persists into adolescence. Methods and Results Ascending aorta Z scores were obtained from first postnatal echocardiogram.

Ombitasvir/Paritaprevir/Ritonavir With or Without Dasabuvir and With or Without Ribavirin for Adolescents With HCV Genotype 1 or 4.

In adults, treatment of hepatitis C virus (HCV) infection with ombitasvir (OBV)/paritaprevir (PTV)/ritonavir (r) with or without dasabuvir (DSV) and ±ribavirin (RBV) results in high rates of sustained virologic response (SVR). However, these regimens have not been investigated in adolescents. This ongoing, open-label, phase 2/3 study evaluated the pharmacokinetics, safety, and efficacy of OBV/PTV/r+DSV±RBV treatment for 12 weeks in adolescents infected with HCV genotype (GT) 1 without cirrhosis (part 1) and the safety and efficacy of OBV/PTV/r±DSV±RBV treatment for 12 or 24 weeks in adolescents infected with GT1 or GT4 without cirrhosis or with compensated cirrhosis (parts 1 and 2).

Association of Initially Normal Coronary Arteries With Normal Findings on Follow-up Echocardiography in Patients With Kawasaki Disease.

Importance: American Heart Association guidelines recommend echocardiography in Kawasaki disease at baseline, 1 to 2 weeks, and 4 to 6 weeks after treatment to detect coronary artery abnormalities. However, these examinations are expensive and may require sedation in young children, which is burdensome and carries some risk.

Objective: To assess the benefit of additional echocardiographic imaging at 6 weeks in patients with uncomplicated Kawasaki disease who had previously normal coronary arteries.

The Emerging Role of Neurodiagnostic Informatics in Integrated Neurological and Mental Health Care.

Mental, neurological, and neurodevelopmental (MNN) disorders impose an enormous burden of disease globally. Many MNN disorders follow a developmental trajectory. Thus, defining symptoms of MNN disorders may be conceived as the end product of a long developmental process.

A twenty-two-year experience with Hymenoptera venom immunotherapy in a US pediatric tertiary care center 1996-2018.

Background: The rate of systemic reactions (SRs) to venom immunotherapy (VIT) in children has not been well evaluated.

Objective: To evaluate the rate of SRs to VIT in pediatric patients age 5 to 18 years who were treated with a standard protocol.

Methods: A retrospective chart review was conducted to identify patients who received VIT at Boston Children's Hospital from 1996 through 2018.

The classification of ATP-binding cassette subfamily A member 3 mutations using the cystic fibrosis transmembrane conductance regulator classification system.

Importance: The ATP-binding cassette subfamily A member 3 (ABCA3) protein plays a vital role in surfactant homeostasis. Mutations in the gene lead to the development of interstitial lung disease. In the most severe manifestation, mutations can lead to a fatal respiratory distress syndrome in neonates.

Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome.

Objective: To measure the efficacy of mecasermin (recombinant human insulin-like growth factor 1, rhIGF-1), for treating symptoms of Rett syndrome (RTT) in a pediatric population using a double-blind crossover study design.

Methods: Thirty girls with classic RTT in postregression stage were randomly assigned to placebo or rhIGF-1 in treatment period 1 and crossed over to the opposite assignment for period 2 (both 20 weeks), separated by a 28-week washout period. The primary endpoints were as follows: Anxiety Depression and Mood Scale (ADAMS) Social Avoidance subscale, Rett Syndrome Behaviour Questionnaire (RSBQ) Fear/Anxiety subscale, Parent Target Symptom Visual Analog Scale (PTSVAS) top three concerns, Clinical Global Impression (CGI), Parent Global Impression (PGI), and the Kerr severity scale.

Graph theory analysis of cortical thickness networks in adolescents with d-transposition of the great arteries.

Objective: Adolescents with d-transposition of the great arteries (d-TGA) who had the arterial switch operation in infancy have been found to have structural brain differences compared to healthy controls. We used cortical thickness measurements obtained from structural brain MRI to determine group differences in global brain organization using a graph theoretical approach.

Methods: Ninety-two d-TGA subjects and 49 controls were scanned using one of two identical 1.

Systemic Reactions in Pediatric Patients Receiving Standardized Allergen Subcutaneous Immunotherapy with and without Seasonal Dose Adjustment.

Background: The 2003 Joint Task Force on Practice Parameters recommended standardizing allergen subcutaneous immunotherapy (SCIT). Data from longitudinal surveillance survey in North America reported a systemic reaction (SR) rate of 0.1% to 0.

Comparison of the mRNA expression profile of B-cell receptor components in normal CD5-high B-lymphocytes and chronic lymphocytic leukemia: a key role of ZAP70.

The B-cell receptor (BCR) signaling pathway is of great importance for B-cell survival and proliferation. The BCR expressed on malignant B-CLL cells contributes to the disease pathogenesis, and its signaling pathway is currently the target of several therapeutic strategies. Although various BCR alterations have been described in B-CLL at the protein level, the mRNA expression levels of tyrosine kinases in B-CLL compared to that in normal CD5-high and CD5-low B-lymphocytes remain unknown.

Longitudinal MRI quantification of muscle degeneration in Duchenne muscular dystrophy.

Objective: The aim of this study was to evaluate the usefulness of magnetic resonance imaging (MRI) in detecting the progression of Duchenne muscular dystrophy (DMD) by quantification of fat infiltration (FI) and muscle volume index (MVI, a residual-to-total muscle volume ratio).

Methods: Twenty-six patients (baseline age: 5-12 years) with genetically proven DMD were longitudinally analyzed with lower limb 3T MRI, force measurements, and functional tests (Gowers, 10-m time, North Star Ambulatory Assessment, 6-min walking test). Five age-matched controls were also examined, with a total of 85 MRI studies.

Organizational topology of brain and its relationship to ADHD in adolescents with d-transposition of the great arteries.

Objective: Little is currently known about the impact of congenital heart disease (CHD) on the organization of large-scale brain networks in relation to neurobehavioral outcome. We investigated whether CHD might impact ADHD symptoms via changes in brain structural network topology in a cohort of adolescents with d-transposition of the great arteries (d-TGA) repaired with the arterial switch operation in early infancy and referent subjects. We also explored whether these effects might be modified by apolipoprotein E (APOE) genotype, as the APOE ε2 allele has been associated with worse neurodevelopmental outcomes after repair of d-TGA in infancy.

Circulating Adipokines and Vascular Function: Cross-Sectional Associations in a Community-Based Cohort.

Adipokines may be potential mediators of the association between excess adiposity and vascular dysfunction. We assessed the cross-sectional associations of circulating adipokines with vascular stiffness in a community-based cohort of younger adults. We related circulating concentrations of leptin and leptin receptor, adiponectin, retinol-binding protein 4, and fatty acid-binding protein 4 to vascular stiffness measured by arterial tonometry in 3505 Framingham Third Generation cohort participants free of cardiovascular disease (mean age 40 years, 53% women).

Role of the clathrin adaptor PICALM in normal hematopoiesis and polycythemia vera pathophysiology.

Clathrin-dependent endocytosis is an essential cellular process shared by all cell types. Despite this, precisely how endocytosis is regulated in a cell-type-specific manner and how this key pathway functions physiologically or pathophysiologically remain largely unknown. PICALM, which encodes the clathrin adaptor protein PICALM, was originally identified as a component of the CALM/AF10 leukemia oncogene.

Rational use of antihypertensive medications in children.

Hypertension has traditionally been regarded as an uncommon diagnosis in childhood and adolescence; however, there is compelling evidence to suggest that its prevalence is on the rise, particularly in those with obesity. As a result, pediatricians increasingly are asked to evaluate and manage patients with elevated blood pressure. An increased emphasis on conducting drug trials in children over the last 15 years has yielded important advances with respect to evidence-based data regarding the efficacy and safety of antihypertensive medications in children and adolescents.

Validity and generalizability of the Withdrawal Assessment Tool-1 (WAT-1) for monitoring iatrogenic withdrawal syndrome in pediatric patients.

Critically ill pediatric patients frequently receive prolonged analgesia and sedation to provide pain relief and facilitate intensive care therapies. Iatrogenic withdrawal syndrome occurs when these drugs are stopped abruptly or weaned too rapidly. We investigated the validity and generalizability of the Withdrawal Assessment Tool-1 (WAT-1) in children during weaning of analgesics and sedatives.