Tumour mimics in paediatric neuroimaging.

Distinguishing tumours from other conditions is a primary challenge in paediatric neuro-radiology. This paper aims to describe mimics, which are non-neoplastic conditions that have features similar to a neoplastic process caused by a non-neoplastic entity, and chameleons, which are uncommon presentations of brain tumours that are mistaken for other diagnoses. By doing so, we aim to raise awareness of these conditions and prevent inappropriate investigations or treatment in children.

Challenges in institutional ethical review process and approval for international multicenter clinical studies in lower and middle-income countries: the case of PARITY study.

Background: One of the greatest challenges to conducting multicenter research studies in low and middle-income countries (LMICs) is the heterogeneity in regulatory processes across sites. Previous studies have reported variations in requirements with a lack of standardization in the Institutional Review Board (IRB) processes between centers, imposing barriers for approval, participation, and development of multicenter research.

Objectives: To describe the regulatory process, variability and challenges faced by pediatric researchers in LMICs during the IRB process of an international multicenter observational point prevalence study (Global PARITY).

Germline de novo alterations of RUNX1T1 in individuals with neurodevelopmental and congenital anomalies.

Runt-related transcription factor 1 translocated to 1 (RUNX1T1; also known as eight-twenty-one [ETO]) encodes a transcription regulator for hematopoietic genes and is well known for its involvement in hematologic malignancies, particularly acute myeloid leukemia (AML). However, its role in congenital disease is less understood. This study provides detailed clinical and molecular information on three cases exhibiting neurodevelopmental and congenital anomalies with germline de novo alterations in RUNX1T1.

Critical Data for Critical Care: A Primer on Leveraging Electronic Health Record Data for Research From Society of Critical Care Medicine's Panel on Data Sharing and Harmonization.

A growing body of critical care research draws on real-world data from electronic health records (EHRs). The bedside clinician has myriad data sources to aid in clinical decision-making, but the lack of data sharing and harmonization standards leaves much of this data out of reach for multi-institution critical care research. The Society of Critical Care Medicine (SCCM) Discovery Data Science Campaign convened a panel of critical care and data science experts to explore and document unique advantages and opportunities for leveraging EHR data in critical care research.

Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study.

Background: As people with cystic fibrosis (PWCF) live longer due to the breakthrough drug elexacaftor-tezacaftor-ivacaftor (ETI), they have questioned whether other CF therapies could be safely discontinued. SIMPLIFY was the first prospective, randomized trial to evaluate non-inferiority of discontinuing versus continuing two therapies. The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study was conducted to understand experiences of PWCF enrolled in SIMPLIFY, including why they joined, perceptions of randomization, decision-making around study withdrawal, and considerations for future discontinuation studies.

Trifolin attenuates hypertension-mediated cardiac injury by inhibiting cardiomyocyte apoptosis: Mechanistic insights and therapeutic potential.

Background: Hypertension-induced cardiac disease is a common complication and a significant contributor to mortality in hypertensive patients, largely due to cardiomyocyte apoptosis. Although Trifolin has been identified as a potential antihypertensive compound, its therapeutic role in hypertension-induced cardiac injury remains uncertain.

Purpose: This study aims to evaluate the protective effects of Trifolin and explore the underlying mechanisms of its action against hypertension-induced cardiac injury.

An Untold Story: The Feelings of Pediatric Residents Early in the COVID-19 Pandemic and What They Can Teach Us Today.

Objective: To understand the feelings of pediatrics residents early in the COVID-19 pandemic and to offer insights still relevant today.

Methods: We performed a thematic analysis exploring resident feelings early in the pandemic using free-text responses on a national survey distributed between May and June 2020. We analyzed responses from the following multi-part free text question embedded in the larger survey, "Which of the following feelings have you experienced in your role as a pediatric resident during the COVID-19 pandemic" with response prompts including relief, guilt, pride, sadness, worry, fear, and other.

Case Report: A delicate equilibrium of exocrine pancreatic recovery and hepatotoxicity with elexacaftor/tezacaftor/ivacaftor therapy in a pediatric patient with cystic fibrosis.

This case report presents a comprehensive evaluation of the complex balance of therapeutic benefits and potential risks associated with the cystic fibrosis transmembrane conductance regulator (CFTR) modulator elexacaftor/tezacaftor/ivacaftor (ETI) therapy in managing an eight-year-old male with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). While ETI therapy significantly enhanced exocrine pancreatic function, it led to hepatotoxicity, necessitating therapy discontinuation. Attempts to restart ETI at reduced doses were unsuccessful due to persistent hepatic dysfunction.

Does the Daily Practice of a Structured Voice Exercise Protocol Affect the Fitness Instructor's Self-Perceived Vocal Effort, Vocal Fatigue, and Voice Handicap?

Purpose: Identify an optimal "Fitpro Voice Protocol" and display the ease of applicability of this protocol in fitness professional's daily life. This study also aimed to demonstrate the impact of this protocol on the fitpro's vocal quality, vocal fatigue, vocal effort, and self-perceived voice handicap.

Methods: Thirty-six group fitness instructors teaching at least four classes per week were randomized into three voice protocol groups.

Case Report: An association of left ventricular outflow tract obstruction with 5p deletions.

Introduction: 5p deletion syndrome, also called Cri-du-chat syndrome 5p is a rare genetic syndrome with reports up to 36% of patients are associated with congenital heart defects. We investigated the association between left outflow tract obstruction and Cri-du-chat syndrome.

Methods: A retrospective review of the abnormal microarray cases with congenital heart defects in Children's Hospital of Pittsburgh and the Cytogenomics of Cardiovascular Malformations Consortium.

Piezo1 restrains proinflammatory response but is essential in T cell-mediated immunopathology.

Piezo1 is a mechanosensitive, nonselective Ca2+ channel which is broadly expressed in CD4+ T cells. Using lineage-specific Piezo1 knockout mice (Piezo1cKO), we show that loss of Piezo1 in CD4+ T cells significantly increased IFNγ and IL-17 production in vitro under TH1 and TH17 polarizing conditions, respectively. Despite their intrinsic proinflammatory phenotype, Piezo1cKO T cells are incapable of establishing disease in vivo in three separate adoptive transfer (AT) T cell-mediated inflammatory mouse models, including experimental autoimmune encephalomyelitis, inflammatory bowel disease (IBD), and graft versus-host disease.

Recognisable Neuroradiological Findings in Five Neurogenetic Disorders.

The rate of discovery and increased understanding of genetic causes for neurodevelopmental disorders has peaked over the past decade. It is well recognised that some genes show marked variability in neuroradiological phenotypes, and inversely, some radiological phenotypes are associated with several different genetic conditions. However, some readily recognisable brain magnetic resonance imaging (MRI) patterns, especially in the context of corresponding associated clinical findings, should prompt consideration of a pathogenic variant in a specific gene or gene pathway.

The Epidemiology of Pediatric Shoulder Dislocations in the United States.

Background: Shoulder dislocations are the most common of all major joint dislocations and are a frequent cause of emergency department (ED) presentations. While the epidemiology of shoulder dislocations has been characterized in adults, it has not yet been done for children in the United States. The primary goal of this study was to calculate the incidence rate of pediatric shoulder dislocations in the United States.

Reducing Disparities in Breastfeeding: "Breast for Success" Excels a Second Time.

Breastfeeding is a key public health priority with known racial inequities. Despite the well-described and far-reaching health benefits of breastfeeding for mothers and infants, rates of breastfeeding initiation, continuation, and exclusivity lag meaningfully among African American and Black (AA/B) women compared with other racial and ethnic groups due in main to current and historical structural racism. The study objective was to assess the replicability of Breast for Success (BFS) on breastfeeding rates among home-visited low-income predominantly AA/B mothers.

GRADE Notes 4: how to use GRADE when there is "no" evidence? A case study of using unpublished registry data.

Objectives: Trustworthy guidelines rely on systematic reviews of the best available published evidence. The GRADE (Grading of Recommendations Assessment, Development, and Evaluation) Working Group has provided guidance about developing evidence-based recommendations when published direct evidence is lacking. In this article, we provide a case example as an alternate solution to generate primary data using registries prior to collecting expert evidence.