Optimizing laboratory medication safety monitoring in patients with juvenile idiopathic arthritis to advance value-based care.

Introduction/objectives: Most children with juvenile idiopathic arthritis (JIA) are treated with medications that require safety monitoring labs. Recommended testing includes a creatinine level. However, 87.

Semaglutide in obesity-related heart failure with preserved ejection fraction and type 2 diabetes across baseline HbA levels (STEP-HFpEF DM): a prespecified analysis of heart failure and metabolic outcomes from a randomised, placebo-controlled trial.

Background: About half of patients with heart failure with mildly reduced or preserved ejection fraction (HFpEF) have type 2 diabetes. In the STEP-HFpEF DM trial of adults with obesity-related HFpEF and type 2 diabetes, subcutaneous once weekly semaglutide 2·4 mg conferred improvements in heart failure-related symptoms and physical limitations, bodyweight, and other heart failure outcomes. We aimed to determine whether these effects of semaglutide differ according to baseline HbA.

Evolution of sleep disordered breathing in infants with achondroplasia.

Purpose: Children with achondroplasia (ACH) are at risk for sudden death in infancy due to sleep disordered breathing (SDB) and foramen magnum stenosis (FMS). Sleep studies and neuroimaging are performed in infants with ACH, but interpretation of infant studies is challenging. We sought to describe baseline data on polysomnography (PSG) indices in infants with achondroplasia as well as effects of age and surgery on these parameters.

Achieving reliable patient reported outcomes collection to measure health care improvement in a learning health network: lessons from pediatric rheumatology care and outcomes improvement network.

Introduction: Data from the Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) registry suggests that reliable collection of patient-reported outcomes (PROs) varies across sites. The objective of this study was to better understand the practices of collecting PROs at PR-COIN sites.

Methods: A REDCap survey was sent to the lead representative for each PR-COIN site.

Rapid and scalable personalized ASO screening in patient-derived organoids.

Personalized antisense oligonucleotides (ASOs) have achieved positive results in the treatment of rare genetic disease. As clinical sequencing technologies continue to advance, the ability to identify patients with rare disease harbouring pathogenic genetic variants amenable to this therapeutic strategy will probably improve. Here we describe a scalable platform for generating patient-derived cellular models and demonstrate that these personalized models can be used for preclinical evaluation of patient-specific ASOs.

Patient physical violence toward healthcare workers at a US children's hospital.

An improved understanding of patient-related violent events toward healthcare workers (HCWs) is a critical step in mitigating patient violence in the pediatric medical hospital setting. Therefore, we sought to describe the timing/setting, potential antecedents to, and management of pediatric patient-related violence toward HCW. Using our electronic health record (EHR), we performed a retrospective study of patient-related physical violent events from 2017 to 2022 among youth hospitalized at our free-standing children's hospital.

Skin cancer treatment environment and survivorship resources among sexual and gender minority patients: results of the OUT National Cancer Survey.

Sexual and gender minority (SGM) cancer survivors face unmet care needs in accessing cancer health information and social support despite high satisfaction with treatment. SGM patients often delay care due to concerns of discrimination in healthcare settings, though the care experiences of SGM skin cancer survivors are less known. SGM individuals, particularly sexual minority men, report higher skin cancer prevalence and related risk behaviors than heterosexual men.

Investigation of a targeted panel of gut microbiome-derived toxins in children with chronic kidney disease.

Background: The gut-kidney axis is implicated in chronic kidney disease (CKD) morbidity. We describe how a panel of gut microbiome-derived toxins relates to kidney function and neurocognitive outcomes in children with CKD, consisting of indoleacetate, 3-indoxylsulfate, p-cresol glucuronide, p-cresol sulfate, and phenylacetylglutamine.

Methods: The Chronic Kidney Disease in Children (CKiD) cohort is a North American multicenter prospective cohort that enrolled children aged 6 months to 16 years with estimated glomerular filtration rate (eGFR) 30-89 ml/min/1.

Explanatory capacity of measures of community context for paediatric injury hospitalisations in the USA.

Objective: Community context influences children's risk for injury. We aimed to measure the explanatory capacity of two ZIP code-level measures-the Child Opportunity Index V.3.

Practical approach to extracardiac findings in CT and MR for the non-radiologist.

Cross-sectional cardiac imaging plays an essential role in the evaluation of pediatric patients with congenital and acquired heart disease. While the focus of cardiac imaging is primarily on evaluating cardiac structures, it also provides valuable insights into extracardiac structures and associated abnormalities. The radiologists and cardiologists interpreting these exams play a critical role in recognizing these findings and providing recommendations for follow-up testing when indicated.

Clinical outcomes and adverse events of Hedgehog pathway inhibitors for advanced basal cell carcinoma patients: A systematic review and meta-analysis.

Background: Basal cell carcinoma (BCC) is the most common form of skin cancer and poses treatment challenges in advanced stages. Treatment options include surgery, radiotherapy, and systemic therapies, but tumor location and prior interventions can limit these methods. Hedgehog pathway inhibitors (HPIs) are used for patients unsuitable for conventional treatments.

Accuracy of pathogen diagnostic codes for acute hematogenous musculoskeletal infections in children.

Administrative databases are powerful tools for pediatric research but lack patient-level microbiology results. This study aimed to determine the accuracy of pathogen discharge diagnosis codes for children hospitalized with acute hematogenous musculoskeletal infections (MSKIs). Medical records for 244 children hospitalized with acute hematogenous MSKIs were manually reviewed to determine which bacterial pathogen, if any, was identified for each MSKI based on microbiology results obtained during the hospitalization.

One is not like the other: health quality of life scores vary in neuromuscular EOS by diagnosis.

Purpose: The etiology of early-onset scoliosis (EOS) has been shown to significantly influence baseline parent-reported health-related quality of life (HrQOL). In combining these etiology groups, we obligatorily lump together many disparate diagnoses, particularly true in the neuromuscular (NM) cohort. We sought to evaluate the influence of underlying neuromuscular diagnosis on the HrQOL at 5 years following surgery for EOS.

Physiologic and structural characterization of desisobutyryl-ciclesonide, a selective glucocorticoid receptor modulator in newborn rats.

Bronchopulmonary dysplasia, the most prevalent chronic lung disease of prematurity, is often treated with glucocorticoids (GCs) such as dexamethasone (DEX), but their use is encumbered with several adverse somatic, metabolic, and neurologic effects. We previously reported that systemic delivery of the GC prodrug ciclesonide (CIC) in neonatal rats activated glucocorticoid receptor (GR) transcriptional responses in lung but did not trigger multiple adverse effects caused by DEX. To determine whether limited systemic metabolism of CIC was solely responsible for its enhanced safety profile, we treated neonatal rats with its active metabolite desisobutyryl-ciclesonide (Des-CIC).

Structural polymorphism and diversity of human segmental duplications.

Segmental duplications (SDs) contribute significantly to human disease, evolution and diversity but have been difficult to resolve at the sequence level. We present a population genetics survey of SDs by analyzing 170 human genome assemblies (from 85 samples representing 38 Africans and 47 non-Africans) in which the majority of autosomal SDs are fully resolved using long-read sequence assembly. Excluding the acrocentric short arms and sex chromosomes, we identify 173.

Treatment of e-cigarette use among hospitalised adolescents and young adults: a protocol for intervention development and evaluation of preliminary efficacy and implementation outcomes in a randomised controlled trial.

Introduction: Hospitalisation represents an opportunity to identify and treat e-cigarette use among adolescents and young adults (AYAs). Knowledge on how to provide this care is lacking. We aim to fill this gap by developing an e-cigarette use intervention and evaluating preliminary efficacy and implementation outcomes among hospitalised AYAs.

Mortality of gastrointestinal cancers attributable to smoking, alcohol, and metabolic risk factors, and its association with socioeconomic development status 2000-2021: GI Cancer Mortality and Risk Factors.

Objective: Gastrointestinal (GI) cancers account for one-third of global cancer mortality, with nearly half being preventable. This study updates the global burden of GI cancers attributed to major risk factors: smoking, alcohol, and metabolic disturbances.

Methods: We utilized data from the Global Burden of Disease Study 2021 to examine trends in death and age-standardized death rates related to GI cancers caused by smoking, alcohol, high body mass index (BMI), and high fasting blood glucose (FBG) from 2000 to 2021.

A comparative study of Merkel cell carcinoma and melanoma incidence and survival in the United States, 2000-2021.

Merkel cell carcinoma (MCC) and melanoma are important contributors to skin cancer mortality in the United States. We evaluated their epidemiology using US cancer registry data. During 2000-2021, 19,444 MCCs and 646,619 melanomas of the skin were diagnosed.

Modern Digital Query Analytics of Patient Education Materials on Acanthosis Nigricans: Systematic Search and Content Analysis.

Background: Online digital materials are integral to patient education and health care outcomes in dermatology. Acanthosis nigricans (AN) is a common condition, often associated with underlying diseases such as insulin resistance. Patients frequently search the internet for information related to this cutaneous finding.

Esophageal Perforation During Transesophageal Echocardiography Managed Conservatively: A Case Report With a Review of the Literature on Management.

Transesophageal echocardiography (TEE) is one of the cornerstones of cardiac imaging in inpatient and intra-operative settings. TEE is considered a safe procedure, but it may result in serious complications, such as esophageal injury, vocal cord paralysis, arrhythmia, hypotension, seizure, and cardiac arrest. Herein, we discuss one of the rare complications, esophageal perforation, and a conservative approach to managing the patient in a 64-year-old female who underwent a TEE prior to a scheduled valvular surgery.

Bacillus subtilis-derived-exopolysaccharide halts depigmentation and autoimmunity in vitiligo.

Vitiligo has a complex multifactorial etiology involving a T-cell mediated autoimmune response to cutaneous melanocytes. Microbial dysbiosis has been assigned a contributing role in vitiligo etiology. Treating vitiligo can be a challenging task and finding novel treatment approaches is crucial.

Pediatric flecainide pharmacogenomics: a roadmap to delivering precision-based care to pediatrics arrhythmias.

Flecainide acetate is a Class 1c anti-arrhythmic with a potent sodium voltage gated channel blockade which is utilized for the second-line treatment of tachyarrhythmias in children and adults. Given its narrow therapeutic index, the individualization of drug therapy is of utmost importance for clinicians. Despite efforts to improve anti-arrhythmic drug therapy, there remain knowledge gaps regarding the impact of variation in the genes relevant to flecainide's disposition and response.