Publishing Trends in the Field of Pediatric Emergency Medicine From 2004 to 2013.

Objective: To identify publishing trends within the field of pediatric emergency medicine between 2004 and 2013.

Methods: We conducted a MEDLINE search of pediatric emergency medicine articles, filtered by clinical trial, published between 2004 and 2013 in ten journals from the fields of pediatrics, emergency medicine, general medicine, and pediatric emergency medicine. Each article was classified by journal type, study design, results (positive or negative/equivocal), age/type of subjects, and major topic (based on the objective of the study).

Outcomes of Spinal Fusion for Cervical Kyphosis in Children with Neurofibromatosis.

Background: Cervical kyphosis may occur with neurofibromatosis type I (NF1) and is often associated with vertebral dysplasia. Outcomes of cervical spinal fusion in patients with NF1 are not well described because of the rarity of the condition. We aimed to (1) characterize the clinical presentation of cervical kyphosis and (2) report the outcomes of posterior and anteroposterior cervical fusion for the condition in these children.

A prospective 5-year study of the frequency of arrhythmias during serial exercise testing and clinical follow-up after Melody valve implant.

Background: Although percutaneous Melody valve implant has become an accepted alternative to surgical pulmonary valve replacement in patients with congenital heart disease, the benefit regarding frequency and severity of arrhythmias remains undefined.

Objective: The purpose of this study was to evaluate the impact of Melody valve implant on the type and frequency of arrhythmias during cardiopulmonary exercise testing (CPET) and subsequent clinical outcome.

Methods: As part of the phase I Melody valve clinical trial, 136 patients with congenital heart disease underwent prospective serial evaluation including CPET before implant, 6 months after implant, and annually thereafter for 5 years.

Orbital Angiogenesis and Lymphangiogenesis in Thyroid Eye Disease: An Analysis of Vascular Growth Factors with Clinical Correlation.

Purpose: The human orbit is an environment that is vulnerable to inflammation and edema in the setting of autoimmune thyroid disease. Our study investigated the tenet that orbital adipose tissue lacks lymphatic vessels and analyzed the clinicopathologic differences between patients with acute and chronic thyroid eye disease (TED). The underlying molecular mediators of blood and lymphatic vessel formation within the orbital fat also were evaluated.

Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's): An ARChiVe Cohort Study.

Objective: To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegener's) (GPA).

Methods: The European Medicines Agency (EMA) classification algorithm was applied by computation to categorical data from patients recruited to the ARChiVe (A Registry for Childhood Vasculitis: e-entry) cohort, with the data censored to November 2015. The EMA algorithm was used to uniquely distinguish children with MPA from children with GPA, whose diagnoses had been classified according to both adult- and pediatric-specific criteria.

Rationale and study design for a phase I/IIa trial of anakinra in children with Kawasaki disease and early coronary artery abnormalities (the ANAKID trial).

Background: Although Kawasaki disease (KD) is the most common cause of acquired heart disease in children and may result in coronary artery aneurysms (CAA) with an attendant risk of myocardial infarction, there is no recommended therapy to halt progression of arterial wall damage and prevent aneurysm formation in the acute phase of the vasculitis. While intravenous immunoglobulin (IVIG) reduces the risk of CAA, up to 20% of KD patients are IVIG resistant and have a higher risk for developing CAA. The IL-1 pro-inflammatory pathway is upregulated in children with acute KD and plays a critical role in the experimental animal model of KD.

Development and Feasibility Testing of a Critical Care EEG Monitoring Database for Standardized Clinical Reporting and Multicenter Collaborative Research.

Purpose: The rapid expansion of the use of continuous critical care electroencephalogram (cEEG) monitoring and resulting multicenter research studies through the Critical Care EEG Monitoring Research Consortium has created the need for a collaborative data sharing mechanism and repository. The authors describe the development of a research database incorporating the American Clinical Neurophysiology Society standardized terminology for critical care EEG monitoring. The database includes flexible report generation tools that allow for daily clinical use.

White Paper on P4 Concepts for Pediatric Imaging.

Over the past decade, innovations in the field of pediatric imaging have been based largely on single-center and retrospective studies, which provided limited advances for the benefit of pediatric patients. To identify opportunities for potential "quantum-leap" progress in the field of pediatric imaging, the ACR-Pediatric Imaging Research (PIR) Committee has identified high-impact research directions related to the P4 concept of predictive, preventive, personalized, and participatory diagnosis and intervention. Input from 237 members of the Society for Pediatric Radiology was clustered around 10 priority areas, which are discussed in this article.

Treatment of Chronic Myeloid Leukemia in Rural Rwanda: Promising Early Outcomes.

Purpose: The burden of cancer is rising in low- and middle-income countries, yet cancer treatment requires resources that are often not available in these settings. Although management of chronic myeloid leukemia (CML) has been described in low- and middle-income countries, few programs involve patients treated in rural settings. We describe characteristics and early outcomes of patients treated for CML at rural district hospitals in Rwanda.

A Case of HDR Syndrome and Ichthyosis: Dual Diagnosis by Whole-Genome Sequencing of Novel Mutations in GATA3 and STS Genes.

Context: Atypical presentations of complex multisystem disorders may elude diagnosis based on clinical findings only. Appropriate diagnostic tests may not be available or available tests may not provide appropriate coverage of relevant genomic regions for patients with complex phenotypes. Clinical whole-exome/-genome sequencing is often considered for complex patients lacking a definitive diagnosis.

Dissecting Allele Architecture of Early Onset IBD Using High-Density Genotyping.

Background: The inflammatory bowel diseases (IBD) are common, complex disorders in which genetic and environmental factors are believed to interact leading to chronic inflammatory responses against the gut microbiota. Earlier genetic studies performed in mostly adult population of European descent identified 163 loci affecting IBD risk, but most have relatively modest effect sizes, and altogether explain only ~20% of the genetic susceptibility. Pediatric onset represents about 25% of overall incident cases in IBD, characterized by distinct disease physiology, course and risks.

Effects of Vitamin D Supplementation on C-peptide and 25-hydroxyvitamin D Concentrations at 3 and 6 Months.

The link between African-Americans' disproportionate rates of diabetes, obesity and vitamin D deficiency may be marked by C-peptide as an indicator of insulin secretion. We hypothesize that vitamin D supplementation will increase C-peptide, a marker of insulin secretion. During 3 winters from 2007-2010, 328 healthy African-Americans (median age, 51 years) living in Boston, MA were randomized into a 4-arm, double-blind trial for 3 months of placebo, 1000, 2000, or 4000 IU of vitamin D3.

A faculty development program integrating cross-cultural care into a gastrointestinal pathophysiology tutorial benefits students, tutors, and the course.

A specific faculty development program for tutors to teach cross-cultural care in a preclinical gastrointestinal pathophysiology course with weekly longitudinal followup sessions was designed in 2007 and conducted in the same manner over a 6-yr period. Anonymous student evaluations of how "frequently" the course and the tutor were actively teaching cross-cultural care were performed. The statements "This tutor actively teaches culturally competent care" and "Issues of culture and ethnicity were addressed" were significantly improved over baseline 2004 data.

Development of phenotype algorithms using electronic medical records and incorporating natural language processing.

Electronic medical records are emerging as a major source of data for clinical and translational research studies, although phenotypes of interest need to be accurately defined first. This article provides an overview of how to develop a phenotype algorithm from electronic medical records, incorporating modern informatics and biostatistics methods.

Measuring Health-Related Quality of Life With the Parental Opinions of Pediatric Constipation Questionnaire.

Objectives: The purpose of this study was to develop a caregiver-completed constipation condition-specific health-related quality of life (HRQL) instrument.

Methods: 410 caregivers of children aged 2-18 years completed the Parental Opinions of Pediatric Constipation (POOPC), the PedsQL Generic Core Scales, PedQL Family Impact Module, Pediatric Symptom Checklist, the Functional Disability Inventory, the Pediatric Inventory for Parents, and a demographic questionnaire. Exploratory and confirmatory factor analyses were conducted to assess the psychometric properties of the POOPC.

Distinct effects of losartan and atenolol on vascular stiffness in Marfan syndrome.

We conducted a randomized, double-blind trial of losartan (100 mg QD) versus atenolol (50 mg QD) for 6 months in adults with Marfan syndrome. Carotid-femoral pulse wave velocity (PWV), central augmentation index (AIx), aortic diameter and left ventricular (LV) function were assessed with arterial tonometry and echocardiography. Thirty-four subjects (18 female; median age 35 years, IQR 27, 45) were randomized.

Feasibility of using CT volume as a predictor of specimen weight in a subgroup of patients with low risk Wilms tumors registered on COG Study AREN03B2: implications for central venous catheter placement.

Objective: Patients with stage I Wilms tumor, age ≤ 2 years, tumor ≤ 550 g may not require therapy beyond nephrectomy. This study's aims were to determine: (1) if a linear relationship exists between tumor weight and computed tomography (CT) estimated volume; (2) describe the accuracy of a slope-intercept equation in estimating weight; and (3) determine the potential impact of weight estimation on port placement decisions.

Materials And Methods: Tumor weight and port placement information were abstracted from 105 patients, age ≤ 2 years, with tumors ± 550 g, enrolled in COG AREN03B2.

Time efficiency and diagnostic agreement of 2-D versus 3-D ultrasound acquisition of the neonatal brain.

The purpose of this study was to compare acquisition time efficiency and diagnostic agreement of neonatal brain ultrasound (US) scans obtained with a 3-D volume US acquisition protocol and the conventional 2-D acquisition protocol. Ninety-one consecutive premature neonatal brain ultrasound scans were prospectively performed on 59 neonates with the conventional 2-D acquisition protocol. Immediately after the 2-D study, a coronal 3-D ultrasound volume was acquired and later reconstructed into axial and sagittal planes.

Clinically inactive disease in a cohort of children with new-onset polyarticular juvenile idiopathic arthritis treated with early aggressive therapy: time to achievement, total duration, and predictors.

Objective: To determine the elapsed time while receiving aggressive therapy to the first observation of clinically inactive disease (CID), total duration of CID and potential predictors of this response in a cohort of children with recent onset of polyarticular juvenile idiopathic arthritis (poly-JIA).

Methods: Eighty-five children were randomized blindly to methotrexate (MTX), etanercept, and rapidly tapered prednisolone (MEP) or MTX monotherapy and assessed for CID over 1 year of treatment. Patients who failed to achieve intermediary endpoints were switched to open-label MEP treatment.

Estimating utility values for vesicoureteral reflux in the general public using an online tool.

Introduction: Cost-utility analyses are useful to study conditions without a widely accepted treatment algorithm; in pediatric urology, one such condition is vesicoureteral reflux (VUR). A necessary component of cost-utility analyses is to accurately calculate the "utility", a numerical surrogate of quality of life, for various health states. Our aims were to determine utility values for representative VUR health states and to verify the feasibility of a novel online platform for utility elicitation in order to reduce the time and expense of such analyses.