The ACTION VAD Registry: A Collective Five-Year Experience.

Background: The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) began in 2018 as a collaborative learning health system committed to improving outcomes in pediatric heart failure, including children and adults with congenital heart disease, supported with ventricular assist devices (VADs). This report describes patient and device characteristics, and outcomes through 1-year post-implant.

Methods: The ACTION VAD registry report was created from data submitted to the ACTION learning network from April 2018-June 2023.

Evaluating ADHD medication trial representativeness: a Swedish population-based study comparing hypothetically trial-eligible and trial-ineligible individuals.

Background: Randomised controlled trials (RCTs) evaluating ADHD medications often use strict eligibility criteria, potentially limiting generalisability to patients in real-world clinical settings. We aimed to identify the proportion of individuals with ADHD who would be ineligible for medication RCTs and evaluate differences in treatment patterns and clinical and functional outcomes between RCT-eligible and RCT-ineligible individuals.

Methods: We used multiple Swedish national registries to identify individuals with ADHD, aged at least 4 years at the age of diagnosis, initiating pharmacological treatment between Jan 1, 2007, and Dec 31, 2019, with follow-up up to Dec 31, 2020.

A Second Look: Retrospective Identification of Thrombotic Microangiopathy in Pediatric Stem Cell Transplant Patients With Veno-Occlusive Disease.

Background: Veno-occlusive disease (VOD) and transplant-associated thrombotic microangiopathy (TA-TMA) remain a diagnostic and therapeutic challenge for patients undergoing hematopoietic stem cell transplant (HSCT). Both VOD and TA-TMA share an underlying etiology of microvascular endothelial damage. Potential under-recognition of TA-TMA in the context of VOD leaves HSCT recipients vulnerable to additional endothelial damage, and risk of end-organ failure.

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).

Regional Anesthesia and Surgical Volume in Children Undergoing Nuss Repair: A Multicenter Review.

Introduction: Regional anesthetic approach and surgical volume have been shown to outcomes in patients undergoing Nuss procedure for pectus excavatum. However, their independent relationship is not described. We investigated how regional anesthesia and surgical volume are associated with length of stay (LOS), postoperative opioid use, operating room utilization, and complications.

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.

Implementation and sustainability of systems change for mental health promotion and substance misuse prevention: a qualitative study.

Background: Social determinants of health have been shown to influence individual mental health and overall well-being. Additionally, populations that experience stigma and/or discrimination because of race, class, gender, or another identity group experience disproportionately higher rates of mental health disorders than populations that do not experience such marginalization. One way to address upstream social determinants that influence mental health is through systems change initiatives.

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.

End-stage and chronic kidney disease in classic bladder exstrophy: A retrospective muti-institutional cohort study.

Introduction: While most children with classic bladder exstrophy (CBE) are born with normal kidneys, some experience renal deterioration in adulthood. Little is known about the incidence of end-stage and chronic kidney disease (ESKD and CKD, respectively) in this population. Our group has recently published on surgical outcomes in a multi-institutional cohort of 216 people with CBE.

Evaluation of a Novel Mechanical Compression Device.

Background: Venous thromboembolism (VTE) is a leading preventable cause of death in hospitalized patients. Current intermittent pneumatic compression (IPC) devices used to provide external mechanical compression for VTE prevention are associated with a multitude of clinical challenges that often result in subtherapeutic use. A comparative study was conducted of the real-world clinical use of a novel mechanical compression device (MCD) and a current IPC device.

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.