EEG-based brain age prediction in infants-toddlers: Implications for early detection of neurodevelopmental disorders.

The infant brain undergoes rapid developmental changes in the first three years of life. Understanding these changes through the prediction of chronological age using neuroimaging can provide insights into typical and atypical brain development. We utilized 938 resting-state EEG recordings from 457 typically developing infants, 2 to 38 months old, to develop age prediction models.

Gaps in the Assessment and Care of Neurodevelopmental and Psychiatric Conditions Associated With Dystrophinopathy.

Introduction/aims: While dystrophinopathies are primarily characterized by progressive muscle weakness with onset during childhood, dystrophin also plays a role in brain development. This study aimed to characterize how neurodevelopmental and psychiatric disorders are currently identified and managed in clinical care of those with Becker and Duchenne muscular dystrophy (BDMD).

Methods: Parent Project Muscular Dystrophy (PPMD) disseminated surveys to caregivers and health care providers (HCPs) in the United States to assess the frequency and management of neurodevelopmental and psychiatric disorders of those with dystrophinopathy.

Primary Versus Secondary Immune Thrombocytopenia (ITP): A Meeting Report from the 2023 McMaster ITP Summit.

The McMaster Immune Thrombocytopenia (ITP) Summit was an educational seminar from leading experts in immune thrombocytopenia and related disorders geared towards hematologists, internists, immunologists, and clinical and translational scientists. The focus of the Summit was to review the mechanisms, diagnosis and treatment of primary versus secondary ITP. Specific objectives were to describe the unique features of secondary ITP, and to review its mechanisms in the context of autoimmune disease and infection.

Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study.

Background: Systemic juvenile idiopathic arthritis-related lung disease (sJIA-LD) is a severe complication in patients with treatment-refractory systemic juvenile idiopathic arthritis (sJIA). The objective of this study was to evaluate the effect of allogeneic haematopoietic stem-cell transplantation (HSCT) in a cohort of children with sJIA-LD.

Methods: This international, retrospective cohort study was performed in nine hospitals across the USA and Europe in children with sJIA-LD who had received allogeneic HSCT.

Building global collaborative research networks in paediatric critical care: a roadmap.

Paediatric critical care units are designed for children at a vulnerable stage of development, yet the evidence base for practice and policy in paediatric critical care remains scarce. In this Health Policy, we present a roadmap providing strategic guidance for international paediatric critical care trials. We convened a multidisciplinary group of 32 paediatric critical care experts from six continents representing paediatric critical care research networks and groups.

Conduction System Pacing Following a Double Switch Operation for Congenitally Corrected Transposition of the Great Arteries.

The use of conduction system pacing (CSP) in adults with congenital heart disease (CHD) is growing, however data remain limited. In patients with congenitally corrected transposition of the great arteries following the double switch operation, existing CSP tools and techniques require modification to allow for the anterior displacement of the atrioventricular node and proximal conduction system in addition to navigating the tortuous route of the atrial redirection. We report the successful use of CSP focusing on the technique of delivery tool modification to allow stability on the basal septum for deployment to the area of the distal His bundle and proximal left bundle branch.

Selective termination: a life-saving procedure for complicated monochorionic gestations.

Monochorionic twin pregnancies are a subset of twin pregnancies that face potential complications related to a shared circulation between the fetuses. These complications are related to anastomotic placental vessels connecting the cardiovascular systems of the two fetuses, which can result in significant sequela if one twin experiences intrauterine death. The sudden cardiovascular collapse in this scenario leads to a massive blood shift away from the healthy co-twin, significantly jeopardizing its life and long-term neurodevelopmental outcome.

Access to Pediatric Bed Capacity According to Social Determinants of Health: All Beds Are Not Created Equal.

Objective: To study pediatric inpatient hospital capacity and resources, characterizing differences according to social determinants of health (SDoH) using market share techniques.

Study Design: This cross-sectional study uses non-elective inpatient discharges (≥1 month to ≤19 years) from Healthcare Cost and Utilization Project and American Hospital Association surveys to derive hospital capacity and resources/capability. We include US hospitals with ≥1 pediatric bed and ≥1 pediatric discharge and calculate per bed capital, expenditure, and staffing, transfer rates, payer-mix, and adjusted central line-associated blood stream infection (CLABSI) rate.

is implicated in central nervous system, orofacial and maxillofacial anomalies.

Background: Previous studies in mouse, and zebrafish embryos show strong expression in progenitor cells of neuronal and neural crest tissues suggesting its involvement in neural crest specification. However, the role of human transcription factor activator protein 2 ( in human embryonic central nervous system (CNS), orofacial and maxillofacial development is unknown.

Methods: Through a collaborative work, exome survey was performed in families with congenital CNS, orofacial and maxillofacial anomalies.

Substance use during pregnancy and postpartum among individuals with perinatally-acquired HIV in the United States.

Use of cannabis and alcohol were common during pregnancy and the first year postpartum among people with HIV in the United States (2007-2019), but there were no major differences in substance use during pregnancy based on mode of HIV acquisition. The relatively high prevalence of substance use in this population, particularly postpartum alcohol and cannabis use, warrants further attention.

Firearm and Motor Vehicle Pediatric Deaths-Intersections of Age, Sex, Race, and Ethnicity.

Importance: Injuries from firearms and motor vehicle crashes (MVCs) are the leading causes of death among US children and youths aged 0 to 19 years. Examining the intersections of age group, sex, race, and ethnicity is essential to focus prevention efforts.

Objective: To examine firearm and motor vehicle fatality rates by population subgroups and analyze changes over time.

Methodology for pediatric head computed tomography image segmentation and volumetric calculation using a tablet computer and stylus pen.

Purpose: This study presents a MATrix LABoratory (MATLAB)-based methodology for calculating intracranial volumes from head computed tomography (CT) data and compares it with established methods.

Methods: Regions of interest (ROI) were manually segmented on CT images using a stylus pen, facilitated by mirroring a computer desktop onto a tablet. The volumetric process involved three main steps: (1) calculating the volume of a single voxel, (2) counting the total number of voxels within the segmented ROI, and (3) multiplying this voxel count by the single-voxel volume.

Neuronal constitutive endolysosomal perforations enable α-synuclein aggregation by internalized PFFs.

Endocytosis, required for the uptake of receptors and their ligands, can also introduce pathological aggregates such as α-synuclein (α-syn) in Parkinson's Disease. We show here the unexpected presence of intrinsically perforated endolysosomes in neurons, suggesting involvement in the genesis of toxic α-syn aggregates induced by internalized preformed fibrils (PFFs). Aggregation of endogenous α-syn in late endosomes and lysosomes of human iPSC-derived neurons (iNs), seeded by internalized α-syn PFFs, caused the death of the iNs but not of the parental iPSCs and non-neuronal cells.

Survivin modulates stiffness-induced vascular smooth muscle cell motility.

Arterial stiffness is a key contributor to cardiovascular diseases, including atherosclerosis, restenosis, and coronary artery disease, it has been characterized to be associated with the aberrant migration of vascular smooth muscle cells (VSMCs). However, the underlying molecular mechanisms driving VSMC migration in stiff environments remain incompletely understood. We recently demonstrated that survivin, a member of the inhibitor of apoptosis protein family, is highly expressed in both mouse and human VSMCs cultured on stiff polyacrylamide hydrogels, where it modulates stiffness-mediated cell cycle progression and proliferation.

High resolution kinematic approach for quantifying impaired mobility of dystrophic zebrafish larvae.

Dystrophin-deficient zebrafish larvae are a small, genetically tractable vertebrate model of Duchenne muscular dystrophy well suited for early stage therapeutic development. However, current approaches for evaluating their impaired mobility, a physiologically relevant therapeutic target, are characterized by low resolution and high variability. To address this, we used high speed videography and deep learning-based markerless motion capture to develop linked-segment models of larval escape response (ER) swimming.

The human RAD52 complex undergoes phase separation and facilitates bundling and end-to-end tethering of RAD51 presynaptic filaments.

Human RAD52 is a prime target for synthetical lethality approaches to treat cancers with deficiency in homologous recombination. Among multiple cellular roles of RAD52, its functions in homologous recombination repair and protection of stalled replication forks appear to substitute those of the tumor suppressor protein BRCA2. However, the mechanistic details of how RAD52 can substitute BRCA2 functions are only beginning to emerge.

transcription factors regulate promoter activity by a universal, homeostatic mechanism.

Transcription factors (TFs) may activate or repress gene expression through an interplay of different mechanisms, including RNA polymerase (RNAP) recruitment, exclusion, and initiation. TFs often have drastically different regulatory behaviors depending on promoter context and interacting cofactors. However, the detailed mechanisms by which each TF affects transcription and produce promoter-dependent regulation is unclear.

Exercise testing in clinical context: Reference ranges for interpreting anaerobic threshold as an outcome for congenital heart disease patients.

Background: Change in the oxygen consumption (VO) at the ventilatory anaerobic threshold (VAT) is an important outcome in research studies of children with congenital heart disease (CHD). The range of values reported by different raters for any given VAT is needed to contextualize a change in VAT in intervention studies.

Methods: Sixty maximal cardiopulmonary exercise tests (CPET) for CHD patients 8-21 years old were independently reviewed by six exercise physiologists and four pediatric cardiologists.

Computational modeling of superparamagnetic nanoparticle-based (affinity) diagnostics.

Introduction: Magnetic nanoparticles (MNPs), particularly iron oxide nanoparticles (IONPs), are renowned for their superparamagnetic behavior, allowing precise control under external magnetic fields. This characteristic makes them ideal for biomedical applications, including diagnostics and drug delivery. Superparamagnetic IONPs, which exhibit magnetization only in the presence of an external field, can be functionalized with ligands for targeted affinity diagnostics.

Phase II trial of blood-brain barrier permeable peptide-paclitaxel conjugate ANG1005 in patients with recurrent high-grade glioma.

Background: This study is a phase II clinical trial to evaluate the efficacy, safety, and tolerability of the blood-brain barrier (BBB) permeable peptide-paclitaxel conjugate ANG1005 in patients with recurrent high-grade glioma (HGG) (NCT01967810).

Methods: Seventy-three patients were enrolled in 3 separate arms-recurrent glioblastoma (GBM) (Arm 1), bevacizumab refractory GBM (Arm 2), and grade 3 anaplastic gliomas (AGs) (Arm 3). The study was started in October 2013, and the data were locked on September 29, 2017.