The missed crossing vessel during open pyeloplasty: a potential advantage of the robot-assisted approach in children.

Objective: To investigate whether the panoramic view offered by robot-assisted laparoscopic pyeloplasty (RALP) reduces the likelihood of missing a crossing vessel compared to open pyeloplasty in cases where initial pyeloplasty fails.

Methods: A single institution redo-pyeloplasty database was reviewed for children treated between January 2012 to July 2023. Clinical history, imaging and operative details were reviewed to identify the etiology for the redo procedure.

Highly tunable bimane-based fluorescent probes: design, synthesis, and application as a selective amyloid binding dye.

Small molecule fluorescent probes are indispensable tools for a broad range of biological applications. Despite many probes being available, there is still a need for probes where photophysical properties and biological selectivity can be tuned as desired. Here, we report the rational design and synthesis of a palette of fluorescent probes based on the underexplored bimane scaffold.

Improving access to child abuse evaluations through innovation & telemedicine.

Many children and families do not have access to specialized pediatric health care, including child abuse pediatricians. Medical evaluations in cases of suspected child maltreatment including physical abuse, sexual abuse, and neglect are a critical component of the multidisciplinary team response to these concerns. We review the role of child abuse pediatricians in cases of suspected child maltreatment.

Development of Pyridothiophene Compounds for PET Imaging of α-Synuclein.

Aggregated α-synuclein (α-syn) protein is a pathological hallmark of Parkinson's disease (PD) and Lewy body dementia (LBD). Development of positron emission tomography (PET) radiotracers to image α-syn aggregates has been a longstanding goal. This work explores the suitability of a pyridothiophene scaffold for α-syn PET radiotracers, where 47 derivatives of a potent pyridothiophene (asyn-44; K=1.

A Comparison of Ketamine and Midazolam as First-Line Anesthetic Infusions for Pediatric Status Epilepticus.

Background: Pediatric refractory status epilepticus (RSE) often requires management with anesthetic infusions, but few data compare first-line anesthetics. This study aimed to compare the efficacy and adverse effects of midazolam and ketamine infusions as first-line anesthetics for pediatric RSE.

Methods: Retrospective single-center study of consecutive study participants treated with ketamine or midazolam as the first-line anesthetic infusions for RSE at a quaternary care children's hospital from December 1, 2017, until September 15, 2021.

Outpatient management of established bronchopulmonary dysplasia: An update.

As the incidence of infants with bronchopulmonary dyspasia (BPD) has continued to rise, so has their rate of survival. Their medical management is often complex and requires the use of numerous therapies such as steroids, bronchodilators, diuretics and modalities to deliver supplemental oxygen and positive pressure. It also requires multi-disciplinary care to ensure adequate growth and to optimize neurodevelopmental outcomes.

Hoarding behavior and its association with mental health and functioning in a large youth sample.

Hoarding behavior is prevalent in children and adolescents, yet clinicians do not routinely inquire about it and youth may not spontaneously report it due to stigma. It is unknown whether hoarding behavior, over and above obsessive-compulsive symptoms (OCS), is associated with major clinical factors in a general youth population. This observational study included N = 7054 youth who were not seeking help for mental health problems (ages 11-21, 54% female) and completed a structured interview that included evaluation of hoarding behavior and OCS, as a part of the Philadelphia Neurodevelopmental Cohort between November 2009 and December 2011.

A Novel Brain PET Radiotracer for Imaging Alpha Synuclein Fibrils in Multiple System Atrophy.

Abnormal α-synuclein (α-syn) aggregation characterizes α-synucleinopathies, including Parkinson's disease (PD) and multiple system atrophy (MSA). However, no suitable positron emission tomography (PET) radiotracer for imaging α-syn in PD and MSA exists currently. Our structure-activity relationship studies identified 4-methoxy--(4-(3-(pyridin-2-yl)-3,8-diazabicyclo[3.

The Fontan Udenafil Exercise Longitudinal Trial: Subgroup Analysis.

The Pediatric Heart Network's Fontan Udenafil Exercise Longitudinal (FUEL) Trial (Mezzion Pharma Co. Ltd., NCT02741115) demonstrated improvements in some measures of exercise capacity and in the myocardial performance index following 6 months of treatment with udenafil (87.

Small-bowel plication prevents ileostomy prolapse in young children with inflammatory bowel disease.

Purpose: Young children with medically refractory very early-onset inflammatory bowel disease (VEO-IBD) sometimes benefit from ileostomy diversion alone or may be offered subtotal colectomy with ileostomy. Though generally well-tolerated, ileostomy complications are frequent. Prolapse is particularly frustrating as it can be difficult and painful to reduce, becomes a recurring problem is some patients, and often requires ostomy revision or bowel resection.

Microtubule-Stabilizing 1,2,4-Triazolo[1,5-]pyrimidines as Candidate Therapeutics for Neurodegenerative Disease: Matched Molecular Pair Analyses and Computational Studies Reveal New Structure-Activity Insights.

Microtubule (MT)-stabilizing 1,2,4-triazolo[1,5-]pyrimidines (TPDs) hold promise as candidate therapeutics for Alzheimer's disease (AD) and other neurodegenerative conditions. However, depending on the choice of substituents around the TPD core, these compounds can elicit markedly different cellular phenotypes that likely arise from the interaction of TPD congeners with either one or two spatially distinct binding sites within tubulin heterodimers (, the seventh site and the vinca site). In the present study, we report the design, synthesis, and evaluation of a series of new TPD congeners, as well as matched molecular pair analyses and computational studies, that further elucidate the structure-activity relationships of MT-active TPDs.

Increasing CEUS utilization in the USA: a call to action for the adult and pediatric body imaging community.

Despite its well-established benefits and a powerful body of scientific literature supporting the efficacy of contrast-enhanced ultrasound (CEUS), it has faced challenges in being fully adopted as a diagnostic and problem-solving tool within the USA. This effort, written by experts in performance of and interpretation of CEUS, acts as a roadmap, for those interested in expanding CEUS within their facility. CEUS benefits from a Champion who is knowledgeable and passionate about its performance and who is capable of transferring evidence-based enthusiasm to others.

Small interfering ribonucleic acid for cholesterol lowering - Inclisiran: Inclisiran for cholesterol lowering.

Despite the development of new therapies to lower cardiovascular disease (CVD) risk in recent decades, the trend of reductions in CVD mortality has reversed. New therapies are essential for the prevention of first and recurrent CVD. The importance of lowering low-density lipoprotein cholesterol (LDL-C) in the management and prevention of atherosclerotic CVD (ASCVD) is widely reflected in clinical treatment guidelines; however, most patients with established ASCVD do not achieve guideline-recommended LDL-C targets.

The CAPNET multi-center data set for child physical abuse: Rationale, methods and scope.

Background: The pediatric subspecialty of Child Abuse Pediatrics (CAP) was certified by the American Board of Medical Subspecialties in 2006. Relative to its impact on pediatric health, CAP-focused research has been relatively under-funded. Multi-center networks related to CAP-focused research have made important advances, but have been limited in scope and duration.

Advances in Understanding the Mechanism of Transitional Neonatal Hypoglycemia and Implications for Management.

Our lack of basic knowledge about the basic mechanisms of transitional hypoglycemia and other forms of hypoglycemia in newborns underlies the ongoing controversies over standards for managing these conditions. To address this deficiency, the authors evaluated regulation of insulin secretion in fetal, newborn, and adult rats. The results demonstrate that transitional hypoglycemia in normal neonates and persistent hypoglycemia in high-risk infants both reflect altered beta-cell insulin regulation.

Treatment of Posthemorrhagic Hydrocephalus.

The incidence of intraventricular hemorrhage (IVH) has overall declined to 15% to 20% of preterm infants with birth weight less than 1500 g. One of the major complications of severe IVH is posthemorrhagic ventricular dilation (PHVD). Nearly 10% of all infants with IVH and 20% of infants with severe IVH will develop progressive PHVD requiring surgical intervention to prevent parenchymal damage in the developing brain.

The Development of F Fluorthanatrace: A PET Radiotracer for Imaging Poly (ADP-Ribose) Polymerase-1.

Fluorine 18 (F) fluorthanatrace (F-FTT) is a PET radiotracer for imaging poly (adenosine diphosphate-ribose) polymerase-1 (PARP-1), an important target for a class of drugs known as PARP inhibitors, or PARPi. This article describes the stepwise development of this radiotracer from its design and preclinical evaluation to the first-in-human imaging studies and the initial validation of F-FTT as an imaging-based biomarker for measuring PARP-1 expression levels in patients with breast and ovarian cancer. A detailed discussion on the preparation and submission of an exploratory investigational new drug application to the Food and Drug Administration is also provided.

Early childhood lead exposure and adolescent heart rate variability: A longitudinal cohort study.

Lead is a known neurotoxicant with many detrimental health effects, including neurocognitive deficits and cardiovascular and metabolic disorders. However, few studies have tested the association between lead exposure and the physiological stress response, which in and of itself may act as a precursor to and/or underlying mechanism of detrimental health outcomes. The purpose of this study was to examine the influence of early childhood and early adolescent low-level lead exposure on early adolescent heart rate variability, a widely-used measure of physiological stress.

The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics.

Background: The PARADIGHM registry of adult and pediatric patients with chronic hypoparathyroidism evaluates the long-term safety and effectiveness of treatment with recombinant human parathyroid hormone, rhPTH(1-84), and describes the clinical disease course under conditions of routine clinical practice. In this first report, we detail the registry protocol and describe the baseline characteristics of two adult patient cohorts from an interim database analysis. One cohort after study entry were prescribed rhPTH(1-84), and the other cohort received conventional therapy of calcium and active vitamin D.

Medical Management of Eosinophilic Esophagitis in Pediatric Patients.

Eosinophilic esophagitis is an immune-mediated allergic disease of the esophagus that affects pediatric patients of all ages. The diagnosis is made by esophagogastroduodenoscopy demonstrating eosinophilic infiltrate of the esophagus. Approaches to treatment involve proton pump inhibitors (PPIs), swallowed topical steroid preparations, as well as dietary elimination.

Reconstruction of the external auditory canal: The tragal flap revisited and review of contemporary reconstructive techniques.

Background: Primary malignancies arising in the external auditory canal (EAC) are rare and usually are treated surgically. We review techniques to reconstruct the EAC following ablative surgery, and introduce a rarely utilized tragal skin flap which has particular advantages for reconstruction of limited anterior EAC defects.

Methods: The terms "tragal flap", "external auditory canal", "preauricular tragal flap", "reconstructive techniques" were searched on PubMed and Google Scholar.

What's in a name? Sentinel injuries in abused infants.

Infants are at greatest risk of severe and fatal physical abuse yet they sometimes present for medical care multiple times with abusive injuries prior to being diagnosed with abuse and having protective actions taken. Efforts to identify these infants in a timely manner are critical to prevent repeated, escalating abuse and subsequent harm. Increasing the identification and evaluation of sentinel injuries has been highlighted as a strategy for improving timely detection of abuse in infants.

Evaluating Neonatal Telehealth Programs Using the STEM Framework.

The COVID-19 pandemic has caused an explosive adoption of telehealth in pediatrics . However, there remains substantial variation in evaluation methods and measures of these programs despite introduction of measurement frameworks in the last five years. In addition, for neonatal health care, assessing a telehealth program must measure its benefits and costs for four stakeholder groups - patients, providers, healthcare system, and payers.

Identification of a nanomolar affinity α-synuclein fibril imaging probe by ultra-high throughput screening.

Small molecules that bind with high affinity and specificity to fibrils of the α-synuclein (αS) protein have the potential to serve as positron emission tomography (PET) imaging probes to aid in the diagnosis of Parkinson's disease and related synucleinopathies. To identify such molecules, we employed an ultra-high throughput screening strategy using idealized pseudo-ligands termed exemplars to identify compounds for experimental binding studies. For the top hit from this screen, we used photo-crosslinking to confirm its binding site and studied the structure-activity relationship of its analogs to develop multiple molecules with nanomolar affinity for αS fibrils and moderate specificity for αS over Aβ fibrils.