Is Endomyocardial Biopsy a Safe and Useful Procedure in Children with Suspected Cardiomyopathy?

Endomyocardial biopsy (EMB) is a common procedure used to aid in the diagnosis, prognosis and treatment of suspected pediatric cardiomyopathy. In suspected cardiomyopathy, no multicenter experience has previously reported on the safety and utility of EMBs. Retrospectively, adverse event (AE) and patient and procedural characteristics were obtained at seven institutions participating in the Congenital Cardiac Catheterization Outcomes Project for both a cardiomyopathy (n = 158) and a post-transplant surveillance (n = 2665) cohort.

Comparison of Extracellular Matrix Patch and Standard Patch Material in the Pulmonary Arteries.

After pulmonary artery (PA) patch augmentation, surgical or catheterization reintervention to address PA stenosis is not uncommon. Multiple patch materials are available and the need for reintervention may be related to characteristics of the patch material. In this retrospective chart review of patients who underwent PA augmentation from 2004 through 2013, we compare outcomes after PA augmentation with standard patch (SP) material and extracellular matrix (ECM) patch material.

Pathogenetics of alveolar capillary dysplasia with misalignment of pulmonary veins.

Alveolar capillary dysplasia with misalignment of pulmonary veins (ACDMPV) is a lethal lung developmental disorder caused by heterozygous point mutations or genomic deletion copy-number variants (CNVs) of FOXF1 or its upstream enhancer involving fetal lung-expressed long noncoding RNA genes LINC01081 and LINC01082. Using custom-designed array comparative genomic hybridization, Sanger sequencing, whole exome sequencing (WES), and bioinformatic analyses, we studied 22 new unrelated families (20 postnatal and two prenatal) with clinically diagnosed ACDMPV. We describe novel deletion CNVs at the FOXF1 locus in 13 unrelated ACDMPV patients.

Is outpatient robotic pyeloplasty feasible?

With increased experience, many laparoscopic procedures have evolved from mandatory same-day admission to the outpatient setting. Given the shorter operative time and length of stay, the potential to perform robotic surgery as an outpatient procedure exists. We sought to describe our initial experience with performing robotic-assisted laparoscopic pyeloplasty (RP) on children in an outpatient setting.

Pediatric Cardiac Intensive Care Society 2014 Consensus Statement: Pharmacotherapies in Cardiac Critical Care Immune Therapy.

Objective: In this Consensus Statement, we review the etiology and pathophysiology of inflammatory processes seen in critically ill children with cardiac disease. Immunomodulatory therapies aimed at improving outcomes in patients with myocarditis, heart failure, and transplantation are extensively reviewed.

Data Sources: The author team experience and along with an extensive review of the medical literature were used as data sources.

Aiming to Preserve Pulmonary Valve Function in Tetralogy of Fallot Repair: Comparing a New Approach to Traditional Management.

Pulmonary valve (PV) incompetence following transannular patch (TAP) repair of tetralogy of Fallot (TOF) results in long-term morbidity and mortality. Valve-sparing repairs have recently gained recognition; however, they may be associated with residual pulmonary stenosis (PS) in patients with small PV z scores. We sought to determine whether a repair that increases the PV annulus and augments the valve leaflet with a biomaterial would result in annular growth and in longer duration of valve competence compared with TAP.

Use of Glucuronidated Mycophenolic Acid Levels for Therapeutic Monitoring in Pediatric Lupus Nephritis Patients.

Background/objectives: Mycophenolate mofetil (MMF) is used to treat pediatric-onset lupus nephritis (pLN). Data are equivocal on the use of plasma mycophenolic acid (MPA) levels as a measure of efficacy and predictor of therapeutic outcomes in pLN. Glucuronidated MPA (MPA-G) is an inactive metabolite that is a marker of adequate absorption and normal metabolism of MMF.

Improved Splenic Function After Hematopoietic Stem Cell Transplant for Sickle Cell Disease.

Background: Splenic dysfunction is a significant complication of sickle cell disease (SCD). Hematopoietic stem cell transplant (HSCT) is a proven cure for SCD; however, its long-term effect on splenic function is not well characterized.

Procedure: We conducted a retrospective cohort study of pediatric patients who had HSCT for SCD at two transplant centers.

Vaccination Rates for Measles, Mumps, Rubella, and Influenza Among Children Presenting to a Pediatric Emergency Department in New York City.

We compared measles, mumps, rubella (MMR), and influenza vaccination rates of children presenting to a Pediatric Emergency Department (PED) in New York City with rates from national assessments. MMR and influenza vaccination rates in this PED population were generally comparable to community rates, but lower than Healthy People 2020 targets.

Safety and Efficacy of Belimumab to Treat Systemic Lupus Erythematosus in Academic Clinical Practices.

Objective: To evaluate the use and efficacy of belimumab in academic practices. Belimumab is a human monoclonal antibody that inhibits soluble B lymphocyte stimulator and has been approved for the treatment of adults with systemic lupus erythematosus (SLE).

Methods: Invitations to participate and complete a 1-page questionnaire for each patient prescribed belimumab were sent to 16 physicians experienced in SLE phase III clinical trials.

Scientific Statement on the Diagnostic Criteria, Epidemiology, Pathophysiology, and Molecular Genetics of Polycystic Ovary Syndrome.

Polycystic ovary syndrome (PCOS) is a heterogeneous and complex disorder that has both adverse reproductive and metabolic implications for affected women. However, there is generally poor understanding of its etiology. Varying expert-based diagnostic criteria utilize some combination of oligo-ovulation, hyperandrogenism, and the presence of polycystic ovaries.

Value and limitations of clinical practice guidelines in neonatology.

Given the overwhelming size of the neonatal literature, clinicians must rely upon expert panels such as the Committee on Fetus and Newborn in the USA and the National Institute for Healthcare and Excellence in the UK for guidance. Guidelines developed by expert panels are not equivalent to evidence-based medicine and are not rules, but do provide evidence-based recommendations (when possible) and at minimum expert consensus reports. The standards used to develop evidence-based guidelines differ among expert panels.

The Epidemiology of Anaphylaxis.

Anaphylaxis is a dramatic expression of systemic allergy. The lifetime prevalence of anaphylaxis is currently estimated at 0.05-2 % in the USA and ~3 % in Europe.

Belimumab in systemic lupus erythematosus: a perspective review.

Belimumab (Benlysta(®)) is a fully humanized monoclonal antibody that inhibits B-lymphocyte stimulator (also known as B cell activating factor of the tumor necrosis factor family) and was approved by the US Food and Drug Administration and the European Medicines Evaluation Agency for treatment of autoantibody-positive systemic lupus erythematosus (SLE) in adults. This review discusses the key findings of the phase III trials, post hoc analyses, and real-world postmarketing use of belimumab in the routine care of SLE patients. It also highlights the safety profile of belimumab and gives insight into its potential use to treat childhood-onset SLE.

Outcomes and Predictors of Perinatal Mortality in Fetuses With Ebstein Anomaly or Tricuspid Valve Dysplasia in the Current Era: A Multicenter Study.

Background: Ebstein anomaly and tricuspid valve dysplasia are rare congenital tricuspid valve malformations associated with high perinatal mortality. The literature consists of small, single-center case series spanning several decades. We performed a multicenter study to assess the outcomes and factors associated with mortality after fetal diagnosis in the current era.

Balloon valvuloplasty for congenital aortic stenosis: Multi-center safety and efficacy outcome assessment.

Objective: To describe contemporary outcomes of balloon aortic valvuloplasty (BAVP) performed in 22 US centers.

Background: BAVP constitutes first-line therapy for congenital aortic stenosis (cAS) in many centers.

Methods: We used prospectively-collected data from two active, multi-institutional, pediatric cardiac catheterization registries.

Norwood Stage I Palliation in Patients Less Than or Equal to 2.5 kg: Outcomes and Risk Analysis.

Background: Hospital mortality of stage I palliation in patients with hypoplastic left heart syndrome and weighing less than or equal to 2.5 kg remains high. We aimed to (1) assess outcomes in this specific population in a dedicated neonatal cardiac program and (2) determine factors associated with poor outcomes.

Longitudinal Strain by Speckle Tracking Echocardiography in Pediatric Heart Transplant Recipients.

Introduction: Heart transplant recipients are at risk for developing left ventricular (LV) dysfunction. While traditional echocardiographic parameters have limitations, speckle tracking echocardiography (STE) is a novel technique shown to be more sensitive and accurate in adult studies for evaluating ventricular systolic and diastolic function.

Design: Pediatric heart transplant recipients undergoing routine cardiac catheterization were included.

Repair of Tetralogy of Fallot in Children Less Than 4 kg Body Weight.

We reviewed our experience of surgical repair of Tetralogy of Fallot (TOF) in children weighing less than or equal to 4 kg to compare outcome of early palliation versus complete repair as the initial surgical approach. Seventy-six patients, weighing ≤ 4 kg, with TOF surgery between January 2005 and September 2013 were included in this single-center retrospective study. Twenty-five patients who underwent initial shunt procedure followed by later full repair were compared to 51 patients who had primary full repair for differences in baseline characteristics and outcomes.

Fetal Growth and Neurodevelopmental Outcome in Congenital Heart Disease.

We evaluated differences in growth between fetuses with and without congenital heart disease (CHD) and tested associations between growth and early childhood neurodevelopment (ND). In this prospective cohort study, fetuses with hypoplastic left heart syndrome (HLHS), transposition of the great arteries (TGA), and tetralogy of Fallot (TOF) and controls had biparietal diameter (BPD), head (HC) and abdominal circumference (AC), femur length (FL), and estimated fetal weight (EFW) recorded serially during pregnancy at 18-26 weeks GA (F1), at 27-33 weeks GA (F2), and at 34-40 weeks GA (F3). CHD subjects underwent Bayley Scales of Infant Development-III ND testing at 18 months.