Acetaminophen for the patent ductus arteriosus: has safety been adequately demonstrated?

Patent ductus arteriosus (PDA) is the most common cardiovascular condition diagnosed in premature infants. Acetaminophen was first proposed as a potential treatment for PDA in 2011. Since that time acetaminophen use among extremely preterm neonates has increased substantially.

Generation of cerebral cortical GABAergic interneurons from pluripotent stem cells.

The cerebral cortex functions by the complex interactions of intrinsic and extrinsic neuronal activities, glial actions, and the effects of humoral factors. The intrinsic neuronal influences are mediated by two major subclasses: excitatory glutamatergic neurons that generally have axonal projections extending beyond the neuron's locality and inhibitory GABAergic neurons that generally project locally. These interneurons can be grouped based on morphological, neurochemical, electrophysiological, axonal targeting, and circuit influence characteristics.

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.

Periodic limb movements and restless legs syndrome in children with a history of prematurity.

Introduction: Little is known about the pediatric population at an increased risk of restless legs syndrome (RLS) and periodic limb movement disorder (PLMD). Polysomnographic data from the Caffeine for Apnea of Prematurity-Sleep (CAPS) study showed a high prevalence of elevated periodic limb movement index (PLMI) in a cohort of ex-preterm children, but the clinical importance of this finding, such as association with RLS, is unknown. We hypothesized that ex-preterm children would have a high prevalence of RLS and PLMD.

Location of In-Hospital Cardiac Arrest in the United States-Variability in Event Rate and Outcomes.

Background: In-hospital cardiac arrest (IHCA) is a major public health problem with significant mortality. A better understanding of where IHCA occurs in hospitals (intensive care unit [ICU] versus monitored ward [telemetry] versus unmonitored ward) could inform strategies for reducing preventable deaths.

Methods And Results: This is a retrospective study of adult IHCA events in the Get with the Guidelines-Resuscitation database from January 2003 to September 2010.

Relationship between obstructive sleep apnea cardiac complications and sleepiness in children with Down syndrome.

Objective/background: Children with Down syndrome (DS) have a high rate of pulmonary hypertension and sleepiness. They also have a high prevalence of obstructive sleep apnea syndrome (OSAS). We hypothesized that OSAS was associated with cardiovascular dysfunction and sleepiness in children with DS, and that this dysfunction was partly reversible.

Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle.

Gene augmentation therapy as a strategy to treat alpha-1 antitrypsin (AAT) deficiency has reached phase 2 clinical testing in humans. Sustained serum levels of AAT have been observed beyond one year after intramuscular administration of a recombinant adeno-associated virus (rAAV) vector expressing the AAT gene. In this study, sequential muscle biopsies obtained at 3 and 12 months after vector injection were examined for the presence of rAAV vector genomes.

Effects of Multiple Ventilation Courses and Duration of Mechanical Ventilation on Respiratory Outcomes in Extremely Low-Birth-Weight Infants.

Importance: Extubation failure is common in extremely preterm infants. The current paucity of data on the adverse long-term respiratory outcomes associated with reinitiation of mechanical ventilation prevents assessment of the risks and benefits of a trial of extubation in this population.

Objective: To evaluate whether exposure to multiple courses of mechanical ventilation increases the risk of adverse respiratory outcomes before and after adjustment for the cumulative duration of mechanical ventilation.

Loss of PIKfyve in platelets causes a lysosomal disease leading to inflammation and thrombosis in mice.

PIKfyve is essential for the synthesis of phosphatidylinositol-3,5-bisphosphate [PtdIns(3,5)P2] and for the regulation of endolysosomal membrane dynamics in mammals. PtdIns(3,5)P2 deficiency causes neurodegeneration in mice and humans, but the role of PtdIns(3,5)P2 in non-neural tissues is poorly understood. Here we show that platelet-specific ablation of PIKfyve in mice leads to accelerated arterial thrombosis, and, unexpectedly, also to inappropriate inflammatory responses characterized by macrophage accumulation in multiple tissues.

Patient reported outcomes as indicators of pediatric health care quality.

Health care reform has increased demand for pediatric health care quality evaluations, particularly those that assess the impact of care on patient and population health outcomes. Many of today's most common childhood conditions are characterized by symptoms, behaviors, and functional limitations that are best assessed as patient reported outcomes (PROs). Although they remain greatly underutilized, PROs have the potential to improve pediatric health care quality assessment at the point of care and through system-level performance evaluations.

Feasibility of comprehensive, unattended ambulatory polysomnography in school-aged children.

Study Objectives: Although unattended ambulatory polysomnography (PSG) is frequently performed in adults, few studies have been performed in children. The objective of this study was to evaluate the feasibility of comprehensive, ambulatory PSG, including electroencephalography, in school-aged children in the home environment.

Methods: A total of 201 children, born premature with birth weights of 500-1,250 grams, currently aged 5-12 years and living in Canada and Australia, underwent unattended ambulatory PSG.

Prevalence of periodic limb movements during sleep in normal children.

Study Objectives: Although the American Academy of Sleep Medicine (AASM) mandates that periodic limb movements during sleep (PLMS) be scored on every polysomnogram, and considers a periodic limb movement index (PLMI) > 5/h abnormal in children, there is a lack of community-derived data regarding the prevalence of PLMS in children, and no data to support this cutoff value. Therefore, the aim of this study was to determine the prevalence of PLMS in a sample of normal children.

Design: Retrospective study.

Adeno-associated virus delivery of broadly neutralizing antibodies.

Purpose Of Review: In the present review, we will discuss the emerging field of vector-mediated antibody gene transfer as an alternative HIV vaccine. This approach is an improvement over classical passive immunization strategies that administer antibodies to the host to provide protection from infection. With vector-mediated gene transfer, the antibody gene is delivered to the host, resulting in long-term endogenous antibody expression from the injected muscle that confers protective immunity.

Medial prefrontal cortex processes threatening stimuli in juvenile rats.

To survive, all mammalian species must recognize and respond appropriately to threatening stimuli. In adults, the prelimbic medial prefrontal cortex (mPFC) appears to be involved in fear expression, whereas the infralimbic mPFC mediates fear extinction. In juvenile rats (PN26), the mPFC receives information on potential predators but does not act on it.

Open fetal surgery for central bronchial atresia.

Mainstem or lobar bronchial atresia is associated with massive pulmonary hyperplasia, contralateral pulmonary hypoplasia, non-immune hydrops and a fatal fetal prognosis. Open fetal surgery currently provides a potential therapeutic option for management of a fetus with this diagnosis. We present 2 cases of open fetal surgery for bronchial atresia at the level of the mainstem and lobar bronchus.

Longitudinal neurodevelopmental and neuromotor outcome in congenital diaphragmatic hernia patients in the first 3 years of life.

Objective: The objective of this study was to longitudinally evaluate the neurodevelopmental (ND) outcome in congenital diaphragmatic hernia (CDH) survivors during the first 3 years of life.

Study Design: The study cohort consists of 47 CDH survivors that were enrolled in our prospective, follow-up program between July 2004 and September 2010, and underwent serial ND evaluations during the first 3 years of life. ND outcomes were evaluated using the Bayley Scales of Infant Development (BSID)-II or BSID-III.

Fetal MRI-calculated total lung volumes in the prediction of short-term outcome in giant omphalocele: preliminary findings.

Objective: To determine the value of fetal MRI-calculated total lung volumes (TLV) in the prediction of short-term outcome in patients with giant omphalocele (GO).

Material And Methods: We reviewed all cases of GO undergoing fetal MRI after 21 weeks' gestation and receiving postnatal care at our institution between 2003 and 2010. Observed/expected (O/E) TLV was calculated using age-matched TLV normograms [Radiology 2001;219:236-241].

Candidate proteins, metabolites and transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical study.

Background: Spinal Muscular Atrophy (SMA) is a neurodegenerative motor neuron disorder resulting from a homozygous mutation of the survival of motor neuron 1 (SMN1) gene. The gene product, SMN protein, functions in RNA biosynthesis in all tissues. In humans, a nearly identical gene, SMN2, rescues an otherwise lethal phenotype by producing a small amount of full-length SMN protein.

Early neurodevelopmental outcome of infants with high-risk fetal lung lesions.

Objective: To evaluate the neurodevelopmental outcome of infants with high-risk fetal lung lesions defined as (1) requiring fetal intervention and/or ex utero intrapartum therapy (EXIT), or (2) acute respiratory decompensation postnatally necessitating emergent resection within 48 h of life.

Methods: We reviewed the medical records of 13 consecutive patients with high-risk fetal lung lesions who were enrolled in our prospective interdisciplinary follow-up program. Neurodevelopmental status was evaluated using the Bayley Scales of Infant Development-III (children ≤3 years, n = 12), or the Wechsler Preschool and Primary Scale of Intelligence-III (children ≥4 years, n = 1).

Abnormal brain development and maturation on magnetic resonance imaging in survivors of severe congenital diaphragmatic hernia.

Purpose: The aim of the study was to evaluate the incidence of abnormal brain maturation in survivors of severe congenital diaphragmatic hernia (CDH).

Material And Methods: Between July 2004 and December 2009, 50 CDH survivors underwent detailed brain magnetic resonance (MR) imaging before discharge. Magnetic resonance images were analyzed to evaluate the presence of structural brain abnormalities and to calculate overall brain maturation using the total maturation score (TMS).

Urban primary care physicians' perceptions about initiation of controller medications during a pediatric emergency department visit for asthma.

Objective: This study aimed to identify the beliefs and attitudes of primary care providers (PCPs) regarding emergency department (ED) physicians' initiation of controller medications for children with persistent asthma symptoms during an immediate ED visit.

Methods: We performed semistructured interviews and a focus group with a purposive sample of PCPs of asthmatic patients to assess attitudes toward the National Asthma and Education Prevention Program recommendations regarding ED-based initiation of controller medications. Interviews and a focus group were digitally recorded, transcribed, and entered into qualitative software for coding and analysis.