Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.

The MEK inhibitor selumetinib induces objective responses and provides clinical benefit in children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs). To evaluate whether similar outcomes were possible in adult patients, in whom PN growth is generally slower than in pediatric patients, we conducted an open-label phase 2 study of selumetinib in adults with NF1 PNs. The study was designed to evaluate objective response rate (primary objective), tumor volumetric responses, patient-reported outcomes and pharmacodynamic effects in PN biopsies.

Comparative analysis of injuries related to self-harm, assault, and intimate partner violence: insights from U.S. Emergency Departments (2005-2021).

Background: Emergency departments are on the front lines of non-fatal self-harm injury (SHI). This study identifies patterns in patients presenting to emergency departments with SHI compared with patients presenting with assault and intimate partner violence.

Methods: Using the National Electronic Injury Surveillance System All Injury Program database, we analyzed SHI cases in the emergency department from 2005 to 2021 and examined demographic characteristics, injury mechanism and anatomic location, emergency department disposition and temporal patterns relative to cases involving assault and intimate partner violence.

Neutrophil NADPH oxidase promotes bacterial eradication and regulates NF-κB-Mediated inflammation via NRF2 signaling during urinary tract infections.

The precise role of neutrophil-derived reactive oxygen species (ROS) in combating bacterial uropathogens during urinary tract infections (UTI) remains largely unexplored. In this study, we elucidate the antimicrobial significance of NADPH oxidase 2 (NOX2)-derived ROS, as opposed to mitochondrial ROS, in facilitating neutrophil-mediated eradication of uropathogenic Escherichia coli (UPEC), the primary causative agent of UTI. Furthermore, NOX2-derived ROS regulate NF-κB-mediated inflammatory responses in neutrophils against UPEC by inducing the release of nuclear factor erythroid 2-related factor 2 (Nrf2) from its inhibitor, Kelch-like ECH-associated protein 1 (Keap1).

Best practice peri-extubation bundle reduces neonatal and infant extubation failure after cardiac surgery.

Introduction: Extubation failure after neonatal cardiac surgery is associated with increased intensive care unit length of stay, morbidity, and mortality. We performed a quality improvement project to create and implement a peri-extubation bundle, including extubation readiness testing, spontaneous breathing trial, and high-risk criteria identification, using best practices at high-performing centers to decrease neonatal and infant extubation failure by 20% from a baseline of 15.7% to 12.

Adaptation of the German language version of the QUAlity of life assessment of spina bifida for adults (QUALAS-A-G).

Introduction: Spina bifida (SB) affects almost all activities in daily life and therefore also health-related quality of life (HRQOL). To assess the HRQOL of adults with SB, a self-reported QUAlity of Life Assessment of Spina bifida in Adults (QUALAS-A) was validated in English. The purpose of this study was to develop and validate a German version of QUALAS-A.

Medical Student Mentorship Program in Diagnostic Radiology: The Value of Resident-Led Radiology Mentoring in Early Medical Education.

Mentorship is an often insufficiently acknowledged yet powerful tool to recruit medical students to various specialties and provide guidance as they advance through the stages of their medical career. While the number of specialty-specific mentorship programs available to medical students across the country is on the rise, the utility of trainee-led mentorship is yet to be explored. The purpose of this study is to share a single-institution's experience with implementing a trainee-led radiology mentorship program.

Plastic bronchitis in an adult.

Plastic bronchitis is rare in adult pulmonology and has a wide range of aetiology. Cast analysis is key in narrowing down the differential diagnosis of plastic bronchitis. If suspected of having lymphocytic PB, complete imaging to evaluate thoracic lymphatics is important to find out the potential causes for PB.

2023 Year in Review: High-Flow Nasal Cannula for COVID-19.

COVID-19, caused by SARS-CoV-2 infection, led to a pandemic of acute respiratory illness that is ongoing. High-flow nasal cannula (HFNC) is a commonly used form of respiratory support during acute respiratory distress and is used to treat patients with COVID-19 in many centers. Due to the novel nature of COVID-19 at the onset of the pandemic, evidence to support the use and best practices of HFNC for treating patients with COVID-19 was lacking.

Ovarian Tissue Cryopreservation in Pediatric Centers across the United States: Practice Patterns and Barriers.

Study Objective: To evaluate practice patterns in ovarian tissue cryopreservation (OTC) provision METHODS: US providers practicing or developing OTC in pediatric programs were invited to participate in a survey disseminated via the Oncofertility Consortium.

Results: Twenty-seven programs representing a wide geographic area responded, largely representing academic institutions (85.2%).

Predictors of Selective Serotonin Reuptake Inhibitor Treatment Failure in Persons With Cystic Fibrosis.

Introduction: Depression and anxiety are common in persons with cystic fibrosis (PwCF). Genetic polymorphisms in CYP2C19 and CYP2D6 are well-established predictors of selective serotonin reuptake inhibitors (SSRIs) treatment failure yet have not been studied specifically in PwCF. The purpose of this study was to determine the rate of SSRI failure in PwCF and to identify factors that predict treatment failure.

Experiences and preferences of women with CAH and parents about disclosure of childhood surgery.

Introduction: Complete disclosure of childhood genital surgery to patients with congenital adrenal hyperplasia (CAH) is a critical part of CAH care. There are no guidelines or uniform recommendations on the timing and content of surgical disclosure discussions.

Objective: Our objective was to describe the experiences and preferences of females with CAH and parents of females with CAH who underwent childhood genital surgery regarding surgical disclosure.

A Multimodal Educational Boot Camp for Training Fellows in Pediatric Extracorporeal Membrane Oxygenation (ECMO).

Introduction: Pediatric extracorporeal membrane oxygenation (ECMO) management presents unique challenges in acute care settings, requiring specialized expertise to manage critically ill children. Medical and surgical fellows often manage these patients, but prior residency training rarely provides sufficient ECMO exposure. We developed and evaluated a multimodal pediatric ECMO boot camp for new fellows.

Pharmacological Management of Pediatric Critical Asthma.

Pediatric critical asthma, or formerly known as status asthmaticus, is a common pediatric condition encountered in emergency departments, hospital wards, and pediatric intensive care units. Systemic corticosteroids and inhaled bronchodilators are evidence-based, initial treatments for patients with pediatric critical asthma. If clinical symptoms do not improve, pediatric practitioners often prescribe adjunctive medications including inhaled anticholinergics, intravenous ketamine, intravenous magnesium, intravenous short acting beta 2 agonists, and intravenous methylxanthines (such as aminophylline).

An Assessment of Neonatal Nurses' Knowledge and Provision of Child Passenger Safety Information.

Background: Nurses who care for newborn infants in the hospital have an opportunity to serve as a resource for child passenger safety (CPS). Literature lacks information on what CPS knowledge and provision of information exists for neonatal nurses.

Objective: In this study, we assessed neonatal nurses' knowledge and provision of CPS information.

A Patient-Prioritized Research Agenda for Clinical Trials in Kidney Stone Disease.

Purpose: To ensure that research on kidney stones provides meaningful impact for the kidney stone community, patients and caregivers should be engaged as stakeholders in clinical trial design, starting at study inception. This project aimed to elicit, refine, and prioritize research ideas from kidney stone stakeholders to develop a patient-centered research agenda for clinical trials.

Materials And Methods: The Kidney Stone Engagement Core, a group of patients, caregivers, advocates, clinicians, and researchers, executed an iterative process of surveys and focus groups to elicit and refine research themes, which were then translated into research questions.

A National Simulation-Based Study of Pediatric Critical Care Transport Teams Performance.

Objectives: To assess pediatric critical care transport (CCT) teams' performance in a simulated environment and to explore the impact of team and center characteristics on performance.

Study Design: This observational, multicenter, simulation-based study enlisted a national cohort of pediatric transport centers. Teams participated in 3 scenarios: nonaccidental abusive head injury, sepsis, and cardiac arrest.

Derivation and Validation of an Optimal Neutrophil Gelatinase-Associated Lipocalin Cutoff to Predict Stage 2/3 Acute Kidney Injury (AKI) in Critically Ill Children.

Introduction: Acute kidney injury (AKI) defined by changes in serum creatinine (SCr), or oliguria is associated with increased morbidity and mortality in children who are critically ill. We derived and validated a clinical cutoff value for urine neutrophil gelatinase-associated lipocalin (NGAL), in a prospective multicenter study of children who were critically ill. We report the clinical performance of urine NGAL (uNGAL) to aid in pediatric AKI risk assessment.

Biomarker Changes in Response to Tofacitinib Treatment in Patients With Polyarticular-Course Juvenile Idiopathic Arthritis.

Objective: We examine levels of candidate blood-based biomarkers (CBBs) in patients with juvenile idiopathic arthritis (JIA) treated with tofacitinib.

Methods: Patients with JIA who participated in clinical trial NCT02592434 received tofacitinib from baseline to week 18. Serial serum samples were assayed for CBBs (S100A8/9, S100A12, interleukin-18 [IL-18], serum amyloid A, resistin, vascular endothelial growth factor, angiopoietin-1, angiopoietin-2, matrix metalloproteinase 8 [MMP8], MMP2, tissue inhibitor of metalloproteinases 1, leptin, chemokine [C-X-C motif] ligand 9, soluble IL-2 receptor, intercellular adhesion molecule 1, soluble tumor necrosis factor receptor, IL-6, IL-23, monocyte chemotactic protein 1, chemokine [C-C motif] ligand 18 [CCL18], and CCL20).

Fertility preservation in pediatric central nervous system tumors: A report from the Children's Oncology Group.

The Oncofertility Consortium Pediatric Initiative Network has published recommendations about the risks of infertility due to gonadotoxic therapy. We abstracted gonadotoxic therapies from central nervous system (CNS) Children's Oncology Group (COG) protocols between 2000 and 2022. We assigned them as unknown, minimal, significant, or high levels of increased risk for gonadal dysfunction/infertility.