Revisions to the International Neuroblastoma Response Criteria: A Consensus Statement From the National Cancer Institute Clinical Trials Planning Meeting.

Purpose More than two decades ago, an international working group established the International Neuroblastoma Response Criteria (INRC) to assess treatment response in children with neuroblastoma. However, this system requires modification to incorporate modern imaging techniques and new methods for quantifying bone marrow disease that were not previously widely available. The National Cancer Institute sponsored a clinical trials planning meeting in 2012 to update and refine response criteria for patients with neuroblastoma.

Improving neonatal intubation safety: A journey of a thousand miles.

Neonatal intubation is one of the most common procedures performed by neonatologists, however, the procedure is difficult and high risk. Neonates who endure the procedure often experience adverse events, including bradycardia and severe oxygen desaturations. Because of low first attempt success rates, neonates are often subjected to multiple intubation attempts before the endotracheal tube is successfully placed.

Current challenges in the management of sepsis in ICUs in resource-poor settings and suggestions for the future.

Background: Sepsis is a major reason for intensive care unit (ICU) admission, also in resource-poor settings. ICUs in low- and middle-income countries (LMICs) face many challenges that could affect patient outcome.

Aim: To describe differences between resource-poor and resource-rich settings regarding the epidemiology, pathophysiology, economics and research aspects of sepsis.

Effect of congenital heart disease on 4-year neurodevelopment within multiple-gestation births.

Objectives: We sought to assess the effect of congenital heart disease requiring infant surgery with cardiopulmonary bypass on neurodevelopmental outcomes and growth at 4 years of age, while matching for gestational age, socioeconomic status, maternal gestational conditions, home environment, and parental intelligence by studying multiple-gestation births.

Methods: We performed within-family comparison of 14 multiple-gestation births in which 1 child had congenital heart disease requiring surgery with cardiopulmonary bypass at ≤6 months of age. Between 4 and 5 years of age, a comprehensive neurodevelopmental assessment was performed.

Early and Late Factors Impacting Patient and Graft Outcome in Pediatric Liver Transplantation: Summary of an ESPGHAN Monothematic Conference.

As pediatric liver transplantation comes of age, experts gathered to discuss current paradigms and define gaps in knowledge warranting research to further improve patient and graft outcomes. Identified areas ripe for collaborative research include understanding the molecular and cellular mechanisms of tolerance and the role of donor-specific antibodies, considering ways to expand donor pool, minimizing long-term side effects of immunosuppression, and fine-tuning surgical techniques to minimize biliary and vascular complications.

Thoracoscopic division of vascular rings.

Background/purpose: Vascular rings are traditionally treated via an open thoracotomy. In recent years the use of thoracoscopy has increased. Herein we report our experience with thoracoscopic division of vascular rings in pediatric patients.

Complicating Hemolytic Uremic Syndrome: Survival Without Surgical Intervention.

is an anaerobic bacterium that causes rapidly progressive myonecrosis, bacteremia, and central nervous system infection. It has been reported as a complication of hemolytic uremic syndrome (HUS) in 8 children worldwide; 5 children died, and the 3 reported survivors had surgically treated disease. We present 3 cases of complicating HUS in children, including the first 2 reported cases of survival without surgical intervention.

Guidelines for the management of postoperative obstructive symptoms in children with Hirschsprung disease.

Although most children with Hirschsprung disease ultimately do well, many experience a variety of ongoing problems after pull-through surgery. The most common include obstructive symptoms, soiling, enterocolitis and failure to thrive. The purpose of this guideline is to present a rational approach to the management of postoperative obstructive symptoms in children with Hirschsprung disease.

Tumor Talk and Child Well-Being: Perceptions of "Good" and "Bad" News Among Parents of Children With Advanced Cancer.

Context: Little is known about how parents of children with advanced cancer classify news they receive about their child's medical condition.

Objective: To develop concepts of "good news" and "bad news" in discussions of advanced childhood cancer from parent perspectives.

Methods: Parents of children with advanced cancer cared for at three children's hospitals were asked to share details of conversations in the preceding three months that contained "good news" or "bad news" related to their child's medical condition.

Clinical and Fecal Microbial Changes With Diet Therapy in Active Inflammatory Bowel Disease.

Goal: To determine the effect of the specific carbohydrate diet (SCD) on active inflammatory bowel disease (IBD).

Background: IBD is a chronic idiopathic inflammatory intestinal disorder associated with fecal dysbiosis. Diet is a potential therapeutic option for IBD based on the hypothesis that changing the fecal dysbiosis could decrease intestinal inflammation.

Targeting Functional Impairments in the Treatment of Children and Adolescents with ADHD.

The diagnostic criteria for attention-deficit hyperactivity disorder (ADHD) require both symptoms and impairment to be present. Impairment in functioning is commonly the primary reason for referral, and is also a better predictor of long-term outcomes than ADHD symptoms. And yet, only recently has research begun to examine the impact of ADHD treatments on functional impairment using efficient and psychometrically sound outcome measures.

Patient-centered benefit-risk assessment in duchenne muscular dystrophy.

Introduction: This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored.

Methods: A best-worst scaling survey (BWS) was administered to caregivers and patients.

Cystic Fibrosis: Microbiology and Host Response.

The earliest descriptions of lung disease in people with cystic fibrosis (CF) showed the involvement of 3 interacting pathophysiologic elements in CF airways: mucus obstruction, inflammation, and infection. Over the past 7 decades, our understanding of CF respiratory microbiology and inflammation has evolved with the introduction of new treatments, increased longevity, and increasingly sophisticated laboratory techniques. This article reviews the current understanding of infection and inflammation and their roles in CF lung disease.

A wearable artificial kidney for patients with end-stage renal disease.

Background: Stationary hemodialysis machines hinder mobility and limit activities of daily life during dialysis treatments. New hemodialysis technologies are needed to improve patient autonomy and enhance quality of life.

Methods: We conducted a FDA-approved human trial of a wearable artificial kidney, a miniaturized, wearable hemodialysis machine, based on dialysate-regenerating sorbent technology.

The intestinal microbiome, barrier function, and immune system in inflammatory bowel disease: a tripartite pathophysiological circuit with implications for new therapeutic directions.

We discuss the tripartite pathophysiological circuit of inflammatory bowel disease (IBD), involving the intestinal microbiota, barrier function, and immune system. Dysfunction in each of these physiological components (dysbiosis, leaky gut, and inflammation) contributes in a mutually interdependent manner to IBD onset and exacerbation. Genetic and environmental risk factors lead to disruption of gut homeostasis: genetic risks predominantly affect the immune system, environmental risks predominantly affect the microbiota, and both affect barrier function.

Submucosal nerve diameter of greater than 40 μm is not a valid diagnostic index of transition zone pull-through.

Background: Submucosal nerve hypertrophy is a feature of the transition zone in Hirschsprung disease and has been used as a primary diagnostic feature of transition zone pull-through for patients with persistent obstructive symptoms after their initial surgery. Most published criteria for identification of hypertrophy rely on a nerve diameter of greater than 40μm, based primarily on data from a relatively small number of infants with Hirschsprung disease and controls. The validity of these objective measures has not been validated in appropriate controls for post-pull-through patients.

Kawasaki Disease.

Kawasaki disease is an acute, self-limited vasculitis of unknown etiology that occurs predominantly in infants and children. If not treated early with high-dose intravenous immunoglobulin, 1 in 5 children develop coronary artery aneurysms; this risk is reduced 5-fold if intravenous immunoglobulin is administered within 10 days of fever onset. Coronary artery aneurysms evolve dynamically over time, usually reaching a peak dimension by 6 weeks after illness onset.

2016 Classification Criteria for Macrophage Activation Syndrome Complicating Systemic Juvenile Idiopathic Arthritis: A European League Against Rheumatism/American College of Rheumatology/Paediatric Rheumatology International Trials Organisation Collaborative Initiative.

To develop criteria for the classification of macrophage activation syndrome (MAS) in patients with systemic juvenile idiopathic arthritis (JIA). A multistep process, based on a combination of expert consensus and analysis of real patient data, was conducted. A panel of 28 experts was first asked to classify 428 patient profiles as having or not having MAS, based on clinical and laboratory features at the time of disease onset.

Expert consensus on dynamics of laboratory tests for diagnosis of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis.

Objective: To identify which laboratory tests that change over time are most valuable for the timely diagnosis of macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (sJIA).

Methods: A multistep process, based on a combination of expert consensus and analysis of real patient data, was conducted. A panel of experts was first asked to evaluate 115 profiles of patients with MAS, which included the values of laboratory tests at the pre-MAS visit and at MAS onset, and the change in values between the two time points.

Assessment of Primary Site Response in Children With High-Risk Neuroblastoma: An International Multicenter Study.

Purpose: The International Neuroblastoma Response Criteria (INRC) require serial measurements of primary tumors in three dimensions, whereas the Response Evaluation Criteria in Solid Tumors (RECIST) require measurement in one dimension. This study was conducted to identify the preferred method of primary tumor response assessment for use in revised INRC.

Patients And Methods: Patients younger than 20 years with high-risk neuroblastoma were eligible if they were diagnosed between 2000 and 2012 and if three primary tumor measurements (antero-posterior, width, cranio-caudal) were recorded at least twice before resection.

Spine Computed Tomography Radiation Dose Reduction: Protocol Refinement Based on Measurement Variation at Simulated Lower Radiation Acquisitions.

Study Design: Retrospective dose-simulation comparison.

Objective: To determine if sufficient detail for preoperative analysis of bony anatomy can be acquired at substantially lower doses than those typically used.

Summary Of Background Data: Computed tomography (CT) is a preoperative planning tool for spinal surgery.

Specific carbohydrate diet for pediatric inflammatory bowel disease in clinical practice within an academic IBD center.

Objective: Despite dietary factors being implicated in the pathogenesis of inflammatory bowel disease (IBD), nutritional therapy, outside of exclusive enteral nutrition (EEN), has not had a defined role within the treatment paradigm of pediatric IBD within IBD centers. Based on emerging data, Seattle Children's Hospital IBD Center has developed an integrated dietary program incorporating the specific carbohydrate diet (SCD) into its treatment paradigm. This treatment paradigm uses the SCD as primary therapy as well as adjunctive therapy for the treatment of IBD.

A Cost-Effectiveness Analysis of a Pilot Neonatal Screening Program for Sickle Cell Anemia in the Republic of Angola.

Objective: To assess the cost-effectiveness of a pilot newborn screening (NBS) and treatment program for sickle cell anemia (SCA) in Luanda, Angola.

Study Design: In July 2011, a pilot NBS and treatment program was implemented in Luanda, Angola. Infants identified with SCA were enrolled in a specialized SCA clinic in which they received preventive care and sickle cell education.

Lacerations and Embedded Needles Caused by Epinephrine Autoinjector Use in Children.

Study Objective: Epinephrine autoinjector use for anaphylaxis is increasing. There are reports of digit injections because of incorrect autoinjector use, but no previous reports of lacerations, to our knowledge. We report complications of epinephrine autoinjector use in children and discuss features of these devices, and their instructions for use, and how these may contribute to injuries.