Transamniotic Delivery of Coagulation Factor VIII mRNA: A Step Toward a Potential Novel Strategy for the Perinatal Management of Hemophilia A.

Hemophilia A is an X-linked monogenic disease resulting in insufficient pro-coagulant factor VIII (FVIII) levels. Hemophiliac infants are at risk for life-threatening hemorrhage, especially during birth. No perinatal treatment for Hemophilia A is currently available.

Harassment and Burnout in Academic Pediatric Anesthesiology.

Background: High-profile cases of sexual harassment (SH) have drawn attention to SH in the workplace via the #MeToo movement. Many studies demonstrate SH occurring in medical training and practice. Experiencing SH correlates with long-term personal and professional detrimental effects.

Adjuvant conditioning enhances neutrophil function while inducing a suppressive peritoneal macrophage phenotype.

Adjuvants are widely used to boost the immune response during vaccination protocols. Our group has previously reported that repeated intraperitoneal administration of alum in mice, known as adjuvant conditioning (AC), creates an immunosuppressive environment that delays allogeneic graft rejection through NLRP3-dependent MDSC expansion. However, little is known about the effects of AC on the reprogramming of peritoneal cavity cells, particularly the different peritoneal macrophage populations, and the impact on the adaptive immune response.

Preferences of Adolescents and Young Adults With Epilepsy and Caregivers on Reproductive Health Counseling by Neurologists: A Concept Mapping Study.

Objective: To ascertain reproductive health counseling priorities of adolescent and young adult women with epilepsy (AWWE) and caregivers during neurology visits.

Methods: We recruited AWWE aged 14-26 years and caregivers from institutional neurology clinics, a research registry, and epilepsy listservs for a Concept Mapping study. Participants (1) brainstormed topics important for counseling of AWWE about reproductive health, (2) sorted topics into categories and rated their importance (on a five-point Likert scale) for AWWE aged 14-17 and 18-26 years, and (3) met to interpret study findings.

Elements of Visuopathy of Prematurity Are Unified by Intermittent or Sustained Systemic Inflammation.

We hypothesize that the major pathologies associated with the visual system in preterm infants, retinopathy of prematurity (ROP), cerebral visual impairment (CVI), and neurodevelopmental impairment (NDI), are unified by a common etio-pathogenesis involving intermittent and/or sustained systemic inflammation (ISSI). We refer to the resulting adverse visual outcomes (AVO) as "visuopathy of prematurity" (VOP). We present the published evidence supporting an etio-pathogenic paradigm centered around ISSI that begins before birth (early phase 1), is exacerbated in the newborn period (intermediate phase 2), and culminates in adverse visual and neurodevelopmental outcomes (late phase 3).

The PKU Patient Registry: Development of a patient-driven registry and initial outcomes.

A patient registry facilitates collection of data on a group of patients with similar conditions. While some registries collect clinician-input data, patient-entered registries prioritize the perspective of patients and families. To better support research for phenylketonuria (PKU), National PKU Alliance (NPKUA) launched the PKU Patient Registry in 2017 to collect patient-entered lived experience and natural history data.

Factors associated with lateralized cognitive dysfunction using the Cognitive Lateralization Rating Index in pediatric epilepsy.

Introduction: Optimizing cognitive outcomes of pediatric epilepsy surgery requires understanding of risk for change in function, typically based on hemispheric lateralization of language skills. Identification of cognitive lateralization in children is complicated by disease in the setting of ongoing functional development. A quantitative method for assessing lateralization, the Cognitive Lateralization Rating Index (CLRI), was used as a systematic way to assess lateralized cognitive dysfunction in a sample of pediatric epilepsy surgery candidates.

Eight PICU Follow-Up Programs in the United States Established From 2013 to 2022: Report From the Pediatric Outcomes Studies After PICU (POST-PICU) Investigators.

Objectives: Children surviving critical illness are at risk for new morbidities collectively termed "post-intensive care syndrome-pediatrics" (PICS-p). Because PICU teams are familiar with PICS-p and motivated to improve patient outcomes, intensivists are ideally positioned to improve access to PICU follow-up care. We aimed to describe various models of care developed by existing U.

Depressive symptoms in youth with refractory epilepsy: Exploration of seizure, sociodemographic and cognitive factors.

Introduction: Rates of depressive symptoms in children and youth with epilepsy (CYE) vary among studies, ranging from 12 to 41%. The current study examined depressive symptoms in a large, multi-site sample of CYE who completed measures of emotional and behavioral functioning as part of a pre-surgical neuropsychological evaluation.

Methods: CYE ranged in age from 5 to 18 years old, had refractory epilepsy (n = 416), and underwent neuropsychological assessment, including standardized assessment of intellectual (IQ), emotional, and behavioral functioning using caregiver-proxy.

On the importance of communication, advocacy, and information accuracy in destigmatizing mpox disease.

Disease names that convey the location of discovery, the discovering scientists, the species of discovery, or the most impacted populations have been increasingly recognized as problematic-often leading to or amplifying xenophobia, disrepute, and stigma. In this context, in 2022 the World Health Organization proposed naming diseases after their causative pathogen or symptomatology instead. This recent guidance has been retrospectively applied to a disease at the center of an epidemic rife with intolerance and misinformation, predominantly affecting the already-stigmatized LGBTQ + community: mpox (formerly known as 'monkeypox').

Comparative analysis of signal quality and usability for a novel wireless, wearable EEG sensor.

Objective: This study details the design, efficacy, and usability of a novel wearable, wireless electroencephalography (EEG) sensor designed for extended-duration clinical monitoring in any environment.

Methods: Simultaneous EEG recordings from REMI sensors and a conventional scalp-EEG recording system were conducted across two cohorts: 1) participants undergoing routine epilepsy seizure monitoring and 2) healthy volunteers performing tasks to induce common EEG artifacts. Comparative time and spectral-based analyses were conducted between the recording modalities.

Enhancing Drug Development for Paediatric Pulmonary Hypertension-An Integrative Perspective.

As with adult pulmonary hypertension (PH), high morbidity and mortality persist with diverse types of paediatric PH. Despite major advances in pharmacologic therapies based on extensive studies in adult PH, few drugs have been comprehensively studied in neonates, infants, and children, leaving current paediatric PH care largely dependent on small observational studies and extrapolation of evidence from adult clinical trials. Challenges in developing successful clinical trials in children include the need to define distinct disease phenotypes with well-characterised natural history and outcomes, the lack of established age- and disease-specific study endpoints, small and heterogeneous paediatric populations, and the common off-label use of PH-targeted drug therapies without regulatory approval.

A Technical Assessment of a Commercial GFAP Lateral Flow Assay to Establish Proof-of-Concept for Use in Traumatic Brain Injury.

Glial fibrillary acidic protein (GFAP) is an emerging biomarker for the detection of acute intracranial pathology following acute brain injuries such as traumatic brain injury (TBI), stroke, and hypoxic-ischaemic encephalopathy. We undertake a proof-of-concept technical assessment of a commercial lateral flow test (LFT) for the detection of GFAP [the Upfront DX LVOne GFAP lateral flow assay (LFA)], against GFAP concentrations measured using a gold-standard assay [Single Molecule Arrays (Simoa®)-based Human Neurology 4-Plex B assay] in a TBI population. The ability of the LVOne GFAP LFA for identification of samples with GFAP concentrations above the manufacturer's reported lower limit of detection (≥ 0.

Generative AI in healthcare: challenges to patient agency and ethical implications.

Clinical research is no longer a monopolistic environment wherein patients and participants are the sole voice of information. The introduction and acceleration of AI-based methods in healthcare is creating a complex environment where human-derived data is no longer the sole mechanism through which researchers and clinicians explore and test their hypotheses. The concept of self-agency is intimately tied into this, as generative data does not encompass the same person-lived experiences as human-derived data.

Using solid-state MRI and a double-tuned RF coil to quantify bone matrix and mineral densities in rat bones.

Quantitative information on the composition of bone, specifically the content of calcium phosphate mineral and organic matrix, is essential for accurate diagnosis of metabolic bone diseases such as osteoporosis, osteomalacia, and renal osteodystrophy, as well as for differentiating among these conditions. Conventional MRI fails to provide this information because these substances are solid and, therefore, yield no signal in conventional MRI scans, which typically employ spin or gradient echoes. In this report, we show how phosphorus and proton solid-state MRI yield the desired compositional information in bone specimens with ZTE and WASPI pulse sequences, respectively, coupled with the use of a two-port double-tuned solenoidal RF coil.

The Effect of Hypotension on Cerebral Metabolism and Perfusion in Adults Undergoing Cardiopulmonary Bypass: A Prospective Cohort Study.

Background: Current blood pressure management strategies cannot accommodate large interindividual variations in cerebral autoregulation, which may result in inadvertent cerebral ischemia. A novel optical neuromonitoring device was developed to explore the relationships between blood pressure and cerebral metabolism and hemodynamics during hypotension on cardiopulmonary bypass and the transition on bypass in cardiac surgery.

Methods: Forty-five elective adult patients were monitored by a hybrid optical device incorporating broadband near-infrared spectroscopy for monitoring changes in tissue oxygen saturation and the oxidative state of cytochrome c oxidase (oxCCO) in the brain along with diffuse correlation spectroscopy for measuring a cerebral blood flow index.

Differing patterns of face processing in infants at elevated likelihood of autism.

Typical development shows early biases in face attention during infancy, characterized by face inversion effects and increased attention to the left side of the face. In autism spectrum disorder (ASD), face scanning patterns often differ, with reduced inversion effects and left-side biases. The current study examined inversion effects, side biases, and pupil responses in EL and TL infants at 7, 10, and 13 months using linear mixed modeling.

Adjuvant conditioning shapes the adaptive immune response promoting immunotolerance via NLRP3/interleukin-1.

Although adjuvants typically enhance immune responses, we show that repeated alum administration-termed adjuvant conditioning (AC)-induces an immunosuppressive environment that delays allogeneic graft rejection by expanding myeloid-derived suppressor cells (MDSCs). AC-induced MDSCs suppress antigen-specific adaptive responses both and , a process dependent on NLRP3 and IL-1 signaling. Allogeneic pancreatic islets transplanted into AC-treated NLRP3 mice are not protected, confirming the necessity of NLRP3.

Understanding and improving the mental health of refugees and asylum-seekers: Reflections from the closing panel of the 2024 International Society for Traumatic Stress Studies Annual Meeting.

Forcible displacement due to war and persecution has reached unprecedented heights across the globe. The mental health impact of trauma and displacement on refugee communities is profound. Although there are several evidence-based therapies that are efficacious in reducing symptoms of posttraumatic stress disorder and depression in refugees, many refugees do not have access to these, and, for those who do, a significant proportion do not respond.

Co-occurring Anxiety in a Child With Autism and ADHD.

KM is an 11-year-old autistic boy followed by a developmental-behavioral pediatrician (DBP) practicing within a multidisciplinary autism center. He had been prescribed various attention-deficit hyperactivity disorder (ADHD) medications over the years, most recently dextroamphetamine-amphetamine extended-release capsule 10 mg daily.KM initially presented to the DBP for diagnostic confirmation of autism and ADHD at the age of 7 years.

Complex ADHD in a Child With Tic Disorder.

Jaxon is a healthy 12-year-old boy who is referred to your clinic for medication management. He was diagnosed with ADHD using a validated questionnaire at age 9 years. He is currently prescribed OROS methylphenidate 54 mg capsules once daily in the morning, which he has taken for 3 years.

Extremity rhabdomyosarcoma in children, adolescents and young adults: A report from Children's Oncology Group trials.

Background: Long-term survival and prognostic factors for patients with extremity rhabdomyosarcoma (RMS) treated on contemporary Children's Oncology Group (COG) trials are unknown.

Methods: Data of extremity RMS patients enrolled on COG trials from 1998 to 2014 were analyzed to estimate event-free survival (EFS) and overall survival (OS), and factors associated with survival.

Results: The authors identified 264 extremity RMS patients, 159 (60%) localized and 105 (40%) metastatic.