The Adverse Event Landscape of Stem Cell Transplant: Evidence for AGVHD Driving Early Transplant Associated Toxicities.

Although unrelated-donor (URD) hematopoietic cell transplantation (HCT) is associated with many toxicities, a detailed analysis of adverse events, as defined by the Common Terminology Criteria for Adverse Events (CTCAE), has not previously been curated. This represents a major unmet need, especially as it relates to assessing the safety of novel agents. We analyzed a detailed AE database from the "ABA2" randomized, double-blind, placebo-controlled clinical trial of abatacept for acute graft-versus-host disease (AGVHD) prevention, for which the FDA mandated a detailed AE assessment through Day +180, and weekly neutrophil and platelet counts through Day +100.

A survey of practice in the anesthetic management of adolescent idiopathic scoliosis spine fusion by the North American Pediatric Spine Anesthesiologists Collaborative.

Background: Adolescent Idiopathic Scoliosis (AIS) affects 2%-4% of the general pediatric population. While surgical correction remains one of the most common orthopedic procedures performed in pediatrics, limited consensus exists on the perioperative anesthetic management.

Aims: To examine the current state of anesthetic management of typical AIS spine fusions at institutions which have a dedicated pediatric orthopedic spine surgeon.

How to measure patient and family important outcomes in extremely preterm infants: A scoping review.

Aim: Parents of children born preterm have identified outcomes to be measured for audit and research at 18-24 months of age: child well-being, quality of life/function, socio-emotional/behavioural outcomes, respiratory, feeding, sleeping, and caregiver mental health. The aim was to identify the best tools to measure these seven domains.

Methods: Seven working groups completed literature reviews and evaluated potential tools to measure these outcomes in children aged 18-24 months.

Persistent DNA Methylation Changes across the First Year of Life and Prenatal Exposure in a Canadian Prospective Birth Study.

Background: Evidence suggests that prenatal air pollution exposure alters DNA methylation (DNAm), which could go on to affect long-term health. It remains unclear whether DNAm alterations present at birth persist through early life. Identifying persistent DNAm changes would provide greater insight into the molecular mechanisms contributing to the association of prenatal air pollution exposure with atopic diseases.

Analysis of three-dimensional spine growth for vertebral body tethering patients at 2 and 5 years post operatively.

Purpose: Scoliosis can be treated with vertebral body tethering (VBT) as a motion-sparing procedure. However, the knowledge of how growth is affected by a tether spanning multiple levels is unclear in the literature. Three-dimensional true spine length (3D-TSL) is a validated assessment technique that accounts for the shape of the spine in both the coronal and sagittal planes.

Optimizing the approach to monitoring allograft inflammation using serial urinary CXCL10/creatinine testing in pediatric kidney transplant recipients.

Background: Urinary CXCL10/creatinine (uCXCL10/Cr) is proposed as an effective biomarker of subclinical rejection in pediatric kidney transplant recipients. This study objective was to model implementation in the clinical setting.

Methods: Banked urine samples at a single center were tested for uCXCL10/Cr to validate published thresholds for rejection diagnosis (>80% specificity).

The financial and social impacts of the COVID-19 pandemic on youth with eating disorders, their families, clinicians and the mental health system: a mixed methods cost analysis.

Background: The onset of the COVID-19 pandemic has had an adverse impact on children, youth, and families with eating disorders (EDs). The COVID-19 pandemic exacerbated pre-existing personal and financial costs to youth, caregivers, and health professionals accessing or delivering ED services. The objectives of this mixed methods study were to (1) understand the indirect, direct medical and non-medical costs reported by youth, caregivers, and clinicians; (2) understand how the COVID-19 pandemic may have impacted these costs, and (3) explore implications of these costs with regards to barriers and resources to inform future decisions for the ED system of care.

Comprehensive whole-genome sequence analyses provide insights into the genomic architecture of cerebral palsy.

We performed whole-genome sequencing (WGS) in 327 children with cerebral palsy (CP) and their biological parents. We classified 37 of 327 (11.3%) children as having pathogenic/likely pathogenic (P/LP) variants and 58 of 327 (17.

Paediatric meningitis in the conjugate vaccine era and a novel clinical decision model to predict bacterial aetiology.

Objectives: The aims of this study were to assess aetiology and clinical characteristics in childhood meningitis, and develop clinical decision rules to distinguish bacterial meningitis from other similar clinical syndromes.

Methods: Children aged <16 years hospitalised with suspected meningitis/encephalitis were included, and prospectively recruited at 31 UK hospitals. Meningitis was defined as identification of bacteria/viruses from cerebrospinal fluid (CSF) and/or a raised CSF white blood cell count.

Clinical Genetic Testing for Atrial Fibrillation: Are We There Yet?

Important progress has been made toward unravelling the complex genetics underlying atrial fibrillation (AF). Initial studies were aimed to identify monogenic causes; however, it has become increasingly clear that the most common predisposing genetic substrate for AF is polygenic. Despite intensive investigations, there is robust evidence for rare variants for only a limited number of genes and cases.

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Evaluating Outcome Prediction via Baseline, End-of-Treatment, and Delta Radiomics on PET-CT Images of Primary Mediastinal Large B-Cell Lymphoma.

Objectives: Accurate outcome prediction is important for making informed clinical decisions in cancer treatment. In this study, we assessed the feasibility of using changes in radiomic features over time (Delta radiomics: absolute and relative) following chemotherapy, to predict relapse/progression and time to progression (TTP) of primary mediastinal large B-cell lymphoma (PMBCL) patients.

Material And Methods: Given the lack of standard staging PET scans until 2011, only 31 out of 103 PMBCL patients in our retrospective study had both pre-treatment and end-of-treatment (EoT) scans.

Acute and Persistent Postoperative Functional Decline in Children with Severe Neurological Impairment: A Qualitative, Exploratory Study.

Background: Children with severe neurologic impairment (SNI) regularly require major surgery to manage their underlying conditions. Anecdotal evidence suggests that children with SNI experience unexpected and persistent postoperative functional changes long after the postoperative recovery period; however, evidence from the perspective of caregivers is limited. The purpose of the study was to explore the functional postoperative recovery process for children with SNI.

Antimicrobial Stewardship in Pediatric Emergency Medicine: A Narrative Exploration of Antibiotic Overprescribing, Stewardship Interventions, and Performance Metrics.

Antibiotic overprescribing is prevalent in pediatric emergency medicine, influenced by clinician-caregiver dynamics and diagnostic uncertainties, and poses substantial risks such as increasing antibacterial resistance, adverse drug reactions, and increased healthcare expenditures. While antimicrobial stewardship programs have proven effective in optimizing antibiotic use within inpatient healthcare settings, their implementation in pediatric emergency medicine presents specific challenges. Existing biomarkers like white blood cell count, C-reactive protein, procalcitonin, and presepsin have limitations in their ability to distinguish (serious) bacterial infections from other etiologies of fever.

Fetal Hemodynamics, Early Survival, and Neurodevelopment in Patients With Cyanotic Congenital Heart Disease.

Background: Fetuses with cyanotic congenital heart disease (CHD) exhibit profound fetal circulatory disturbances that may affect early outcomes.

Objectives: This study sought to investigate the relationship between fetal hemodynamics and early survival and neurodevelopmental (ND) outcomes in patients with cyanotic CHD.

Methods: In this longitudinal observational study, fetuses with cyanotic CHD underwent late gestational fetal cardiovascular magnetic resonance (CMR) to measure vessel blood flow and oxygen content.

Transient Receptor Potential Melastatin 7 (TRPM7) Ion Channel Inhibitors: Preliminary SAR and Conformational Studies of Xenicane Diterpenoids from the Hawaiian Soft Coral .

Waixenicin A, a xenicane diterpene from the octocoral , is a selective, potent inhibitor of the TRPM7 ion channel. To study the structure-activity relationship (SAR) of waixenicin A, we isolated and assayed related diterpenes from . In addition to known waixenicins A () and B (), we purified six xenicane diterpenes, 7,8-epoxywaixenicins A () and B (), 12-deacetylwaixenicin A (), waixenicin E (), waixenicin F (), and 20-acetoxyxeniafaraunol B ().

Size and Location of Preterm Brain Injury and Associations With Neurodevelopmental Outcomes.

Background And Objectives: We examined associations of white matter injury (WMI) and periventricular hemorrhagic infarction (PVHI) volume and location with 18-month neurodevelopment in very preterm infants.

Methods: A total of 254 infants born <32 weeks' gestational age were prospectively recruited across 3 tertiary neonatal intensive care units (NICUs). Infants underwent early-life (median 33.

Competence By Design: a transformational national model of time-variable competency-based postgraduate medical education.

Postgraduate medical education is an essential societal enterprise that prepares highly skilled physicians for the health workforce. In recent years, PGME systems have been criticized worldwide for problems with variable graduate abilities, concerns about patient safety, and issues with teaching and assessment methods. In response, competency based medical education approaches, with an emphasis on graduate outcomes, have been proposed as the direction for 21st century health profession education.

An oocyte-specific Cas9-expressing mouse for germline CRISPR/Cas9-mediated genome editing.

Cas9 transgenes can be employed for genome editing in mouse zygotes. However, using transgenic instead of exogenous Cas9 to produce gene-edited animals creates unique issues including ill-defined transgene integration sites, the potential for prolonged Cas9 expression in transgenic embryos, and increased genotyping burden. To overcome these issues, we generated mice harboring an oocyte-specific, Gdf9 promoter driven, Cas9 transgene (Gdf9-Cas9) targeted as a single copy into the Hprt1 locus.

Clinical perspective on topical vaccination strategies.

Vaccination is one of the most successful measures in modern medicine to combat diseases, especially infectious diseases, and saves millions of lives every year. Vaccine design and development remains critical and involves many aspects, including the choice of platform, antigen, adjuvant, and route of administration. Topical vaccination, defined herein as the introduction of a vaccine to any of the three layers of the human skin, has attracted interest in recent years as an alternative vaccination approach to the conventional intramuscular administration because of its potential to be needle-free and induce a superior immune response against pathogens.