Neurodevelopmental and Mental Health Outcomes in a National Clinical Sample of Youth With Sex Chromosome Trisomies Compared With Matched Controls.

Objective: To compare the prevalence of neurodevelopmental and mental health diagnoses in a national sample of youth with sex chromosome trisomies (SCTs) with matched controls.

Methods: Patients in PEDSnet and a diagnosis code mapping to 47,XXY/Klinefelter syndrome (n = 1171), 47,XYY/Double Y syndrome (n = 243), or 47,XXX/Trisomy X syndrome (n = 262) were matched with controls using propensity scores. Generalized estimating equations computed odds ratios (OR) with 95% confidence intervals (CI) for the prevalence of diagnoses within the neurodevelopmental and mental health composites, psychotropic medication prescriptions, and encounters with behavioral health and therapy providers.

Response to induction chemotherapy modifies the effect of conventional prognostic factors in high-risk neuroblastoma: A report from the Children's Oncology Group.

Background: Response to induction chemotherapy has been shown to predict outcome in patients with high-risk neuroblastoma (HR-NB), with those achieving a complete response (CR) having superior outcomes.

Methods: We evaluated whether conventional prognostic factors remain prognostic in subsets of patients defined by response to induction. 1244 Patients from four COG high-risk trials were included.

Clinical factors that influence chorionic villus sampling sample size.

Purpose: Our primary objective was to characterize clinical and procedural factors affecting sample size in chorionic villus sampling (CVS).

Methods: This retrospective, single-site cohort study included singleton pregnancies undergoing transabdominal (TA) and transcervical (TC) CVS between 2020 and 2023. Prenatal and maternal data were obtained from the electronic medical record.

Long-term safety and efficacy of Oleogel-S10 (birch bark extract) in epidermolysis bullosa: 24-month results from the Phase III EASE Study.

Background: Epidermolysis bullosa (EB) is a group of rare, severe, genetic disorders characterised by persistent skin fragility and open wounds. EB manifests as cutaneous and mucosal blistering, erosions and impaired wound healing.

Objectives: To determine the long-term efficacy, tolerability and safety of Oleogel-S10 (birch bark extract) in dystrophic (DEB) and junctional (JEB) EB in the 24-months open-label phase (OLP) of the EASE study.

Incomplete remyelination via therapeutically enhanced oligodendrogenesis is sufficient to recover visual cortical function.

Myelin loss induces neural dysfunction and contributes to the pathophysiology of neurodegenerative diseases, injury conditions, and aging. Because remyelination is often incomplete, better understanding endogenous remyelination and developing remyelination therapies that restore neural function are clinical imperatives. Here, we use in vivo two-photon microscopy and electrophysiology to study the dynamics of endogenous and therapeutic-induced cortical remyelination and functional recovery after cuprizone-mediated demyelination in mice.

Looped Penrose Drainages of Acute Hand Infections in Vulnerable Populations.

Purpose: Acute hand infections (AHIs) remain a challenge for hand surgeons and represent a condition for which clinical outcomes are considerably affected by social barriers. We previously described the looped Penrose drainage technique, where a drain is sutured to itself in a loop and the outflow tract of egress is maintained, thus obviating the need for large incisions, wound closure, or repeat packing, thereby reducing the follow-up burden. In the face of escalating numbers of socioeconomically vulnerable patients, especially in urban settings, we aimed to characterize the clinical features and outcomes of this technique in an urban population of patients with AHI.

Supporting community translation of lung cancer screening: A web-based decision aid to support informed decision making.

Background: Results of the National Lung Screening Trial create the potential to reduce lung cancer mortality, but community translation of lung cancer screening (LCS) has been challenging. Subsequent policies have endorsed informed and shared decision-making and using decision support tools to support person-centered choices about screening to facilitate implementation. This study evaluated the feasibility and acceptability of LuCaS CHOICES, a web-based decision aid to support delivery of accurate information, facilitate communication skill development, and clarify personal preferences regarding LCS-a key component of high-quality LCS implementation.

Knowledge, Attitudes, and Practices in Colorectal Cancer Screening in the Philippines.

Background And Objective: Colorectal cancer (CRC) has the third highest incidence in the Philippines. Currently, there is a paucity in literature that is focused on the knowledge, attitudes, and perceptions of Filipinos regarding CRC screening. This is the first study in the Philippines that describes this.

The impact of neoadjuvant therapy in patients with left-sided resectable pancreatic cancer: an international multicenter study.

Purpose: To assess the association between neoadjuvant therapy and overall survival (OS) in patients with left-sided resectable pancreatic cancer (RPC) compared to upfront surgery.

Background: Left-sided pancreatic cancer is associated with worse OS compared to right-sided pancreatic cancer. Although neoadjuvant therapy is currently seen as not effective in patients with RPC, current randomized trials included mostly patients with right-sided RPC.

Early childhood vaccination coverage and patterns by rural-urban commuting area.

Introduction: National surveillance efforts have reported rural-urban disparities in childhood vaccination coverage by metropolitan statistical area designations, measured at the county level. This study's objective was to quantify vaccination trends using more discrete measures of coverage and rurality than prior work.

Methods: Serial, cross-sectional analyses of National Immunization Survey-Child restricted-use data collected in 2015-2021 for US children born 2014-2018 were conducted.

Assessing Clinical Improvement of Infants Hospitalized for Respiratory Syncytial Virus-Related Critical Illness.

Background: Pediatric respiratory syncytial virus (RSV)-related acute lower respiratory tract infection (LRTI) commonly requires hospitalization. The Clinical Progression Scale Pediatrics (CPS-Ped) measures level of respiratory support and degree of hypoxia across a range of disease severity, but it has not been applied in infants hospitalized with severe RSV-LRTI.

Methods: We analyzed data from a prospective surveillance registry of infants hospitalized for RSV-related complications across 39 U.

The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol.

Background: Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are prevalent disease complications in people with cystic fibrosis. These understudied comorbidities significantly impact quality of life. The impact of highly effective modulator therapy (HEMT) in young children with cystic fibrosis (YCwCF) on these disease complications is unknown.

Blood absolute lymphocyte count and trajectory are important in understanding severe COVID-19.

Background: Low blood absolute lymphocyte count (ALC) may predict severe COVID-19 outcomes. Knowledge gaps remain regarding the relationship of ALC trajectory with clinical outcomes and factors associated with lymphopenia.

Methods: Our post hoc analysis of the Therapeutics for Inpatients with COVID-19 platform trial utilized proportional hazards models to assess relationships between Day (D) 0 lymphopenia (ALC < 0.

Team resuscitation for paediatrics (TRAP); application and validation of a paediatric resuscitation quality instrument in non-simulated resuscitations.

Background: Resuscitation of paediatric cardiac and respiratory arrest is a high-stakes and low frequency event in the paediatric emergency department. Resuscitation team performance assessment tools have been developed and validated for use in the simulation environment, but no tool currently exists to evaluate clinical performance in non-simulated, live paediatric resuscitations.

Methods: This is a validation study assessing inter-rater reliability of a novel assessment tool of clinical performance of non-simulated resuscitations, the Team Resuscitation for Paediatrics tool.

Role of preconception nutrition supplements in maternal anemia and intrauterine growth: a systematic review and meta-analysis of randomized controlled trials.

Background: Impaired intrauterine growth, a significant global health problem, contributes to a higher burden of infant morbidity and mortality, mainly in resource-poor settings. Maternal anemia and undernutrition, two important causes of impaired intrauterine growth, are prioritized by global nutrition targets of 2030. We synthesized the evidence on the role of preconception nutrition supplements in reducing maternal anemia and improving intrauterine growth.

Glycogen synthase GYS1 overactivation contributes to glycogen insolubility and malto-oligoglucan-associated neurodegenerative disease.

Polyglucosans are glycogen molecules with overlong chains, which are hyperphosphorylated in the neurodegenerative Lafora disease (LD). Brain polyglucosan bodies (PBs) cause fatal neurodegenerative diseases including Lafora disease and adult polyglucosan body disease (ABPD), for which treatments, biomarkers, and good understanding of their pathogenesis are currently missing. Mutations in the genes for the phosphatase laforin or the E3 ubiquitin ligase malin can cause LD.

Video versus Direct Laryngoscopy for Tracheal Intubation Following Cardiac Arrest: a Secondary Analysis of the DEVICE Trial.

Background: Airway management is a critical component of the care of patients experiencing cardiac arrest, but data from randomized trials on the use of video vs direct laryngoscopy for intubation in the setting of cardiac arrest are limited. Current AHA guidelines recommend placement of an endotracheal tube either during CPR or shortly after return of spontaneous circulation but do not provide guidance around intubation methods, including the choice of laryngoscope.

Research Question: Does use of video laryngoscopy improve the incidence of successful intubation on the first attempt, compared to use of direct laryngoscopy, among adults undergoing tracheal intubation after experiencing cardiac arrest?

Study Design And Methods: This secondary analysis of the Direct versus Video Laryngoscope (DEVICE) trial compared video laryngoscopy versus direct laryngoscopy in the subgroup of patients who were intubated following cardiac arrest.

Characterization of glucagon-like peptide-1 (GLP-1) agonist exposures reported to a single United States poison center.

Introduction: Glucagon-like peptide-1 agonists have gained attention in recent years due to their efficacy in managing type II diabetes mellitus and their emerging role in weight management. The purpose of this study was to characterize glucagon-like peptide-1 agonist exposures reported to a single United States regional poison center over nine years, including causes of exposure, associated clinical effects, and potential areas for improving patient education and safety.

Methods: This retrospective cohort study analyzed all poison center calls involving glucagon-like peptide-1 agonists submitted to a single United States regional poison center from 14 January 2014 to 1 May 2023.

Colorado Immersion Training in Community Engagement: Ten years of learning and doing.

The Colorado Immersion Training in Community Engagement (CIT) program supports a change in the research trajectory of junior faculty, early career researchers, and doctoral students toward Community-Based Participatory Research (CBPR). CIT is within the Community Engagement and Health Equity Core (CEHE) at the Colorado Clinical and Translational Sciences Institute (CCTSI), an NIH-funded Clinical and Translational Science award. This Translational Science Case Study reports on CIT's impacts from 2010 to 2019.

Cardiopulmonary exercise testing in children and adolescents at moderate altitude: New normative valuesh.

Background: Cardiopulmonary exercise tests (CPETs) measure cardiovascular exercise response. Altitude alters exercise parameters, so standard normative datasets (Cooper, Bruce, Burstein) may not accurately predict exercise parameters for data collected at moderate altitude. This study aimed to: 1) establish modern normative exercise values for children/adolescents at moderate altitude and 2) compare these values against the Cooper, Burstein and Bruce models.

What Is in a Name? Depression and Anxiety Symptoms in Collegiate Athletes With and Without a History of Concussion.

Context: Athletes with a history of concussion (CON) have been demonstrated to have heightened levels of anxiety and depression that may continue well beyond the resolution of concussion symptoms. The global events of 2020 resulted in elevated levels of anxiety and depression in the general population, which may have unequally presented in collegiate athletes with (CON) than those without a history of concussion (NoCON). Using a deception design, our survey-based study compared levels of anxiety and depression in CON and NoCON collegiate athletes in response to the pandemic and social injustices.

Injectable Estradiol Use in Transgender and Gender-Diverse Individuals in the U.S.: A Multicenter Retrospective Study.

Context: Guidelines for use of injectable estradiol esters (valerate [EV] and cypionate [EC]) among transgender and gender diverse (TGD) individuals designated male at birth vary considerably, with many providers noting supraphysiologic serum estradiol concentrations based on current dosing recommendations.

Objectives: 1. Determine dose of injectable estradiol (subcutaneous [SC] and intramuscular [IM]) needed to reach guideline-recommended estradiol concentrations for TGD adults using EC/EV.

A multivalent engagement of ENL with MOZ.

The epigenetic cofactor ENL (eleven-nineteen-leukemia) and the acetyltransferase MOZ (monocytic leukemia zinc finger) have vital roles in transcriptional regulation and are implicated in aggressive forms of leukemia. Here, we describe the mechanistic basis for the intertwined association of ENL and MOZ. Genomic analysis shows that ENL and MOZ co-occupy active promoters and that MOZ recruits ENL to its gene targets.

Genomic analysis of comprehensive next generation sequencing data to explore the criteria for MET amplification as an actionable biomarker in NSCLC.

Introduction: MET amplification (METamp) can be a de novo or acquired resistance driver; however, the definition of METamp that best captures patients who may respond to targeted therapy remains debated. We explored the genomic landscape of METamp NSCLC including degree of amplification, co-drivers, amplicon size, and outcomes to MET inhibitors.

Methods: Hybrid-capture NGS-based genomic profiling from 88,547 tissue and 12,428 liquid NSCLC samples were queried for METamp (copy number (CN) ≥ ploidy + 4, or amplification ratio (AmpRatio; [CN/sample ploidy] ≥ 3).