Serum bile acids as a prognostic biomarker in biliary atresia following Kasai portoenterostomy.

Background And Aims: In biliary atresia, serum bilirubin is commonly used to predict outcomes after Kasai portoenterostomy (KP). Infants with persistently high levels invariably need liver transplant, but those achieving normalized levels have a less certain disease course. We hypothesized that serum bile acid levels could help predict outcomes in the latter group.

Serial Profiling of Circulating Tumor DNA Identifies Dynamic Evolution of Clinically Actionable Genomic Alterations in High-Risk Neuroblastoma.

Unlabelled: Neuroblastoma evolution, heterogeneity, and resistance remain inadequately defined, suggesting a role for circulating tumor DNA (ctDNA) sequencing. To define the utility of ctDNA profiling in neuroblastoma, 167 blood samples from 48 high-risk patients were evaluated for ctDNA using comprehensive genomic profiling. At least one pathogenic genomic alteration was identified in 56% of samples and 73% of evaluable patients, including clinically actionable ALK and RAS-MAPK pathway variants.

Trial of Anti-BDCA2 Antibody Litifilimab for Systemic Lupus Erythematosus.

Background: Antibody-binding of blood dendritic cell antigen 2 (BDCA2), which is expressed exclusively on plasmacytoid dendritic cells, suppresses the production of type I interferon that is involved in the pathogenesis of systemic lupus erythematosus (SLE). The safety and efficacy of subcutaneous litifilimab, a humanized monoclonal antibody that binds to BDCA2, in patients with SLE have not been extensively studied.

Methods: We conducted a phase 2 trial of litifilimab involving participants with SLE.

Dietary Elimination for the Treatment of Atopic Dermatitis: A Systematic Review and Meta-Analysis.

Background: The influence of diet on atopic dermatitis (AD) is complex, and the use of dietary elimination as a treatment has conflicting views.

Objective: To systematically review the benefits and harms of dietary elimination for the treatment of AD.

Methods: We searched MEDLINE, Embase, AMED, PsycINFO, and the Cochrane Central Register of Controlled Trials from inception to January 18, 2022, without language restrictions, for randomized controlled trials (RCTs) and observational studies comparing dietary elimination and no dietary elimination for the treatment of AD.

A Randomized Trial of Lipid Metabolism Modulation with Fenofibrate for Acute Coronavirus Disease 2019.

Background Abnormal cellular lipid metabolism appears to underlie SARS-CoV-2 cytotoxicity and may involve inhibition of peroxisome proliferator activated receptor alpha (PPARα). Fenofibrate, a PPAR-α activator, modulates cellular lipid metabolism. Fenofibric acid has also been shown to affect the dimerization of angiotensin-converting enzyme 2, the cellular receptor for SARS-CoV-2.

Prevalence of Third-party Tracking on Medical Journal Websites.

This cross-sectional study investigates web tracking prevalence and characteristics on medical journal websites.

Associations Between Low Serum Urate, Body Composition, and Mortality.

Objective: Controversy remains as to whether low serum urate or uric acid (UA) levels contribute to adverse outcomes. We evaluated the relation between low serum UA levels and sarcopenia and assessed whether sarcopenia confounds associations between these low levels and mortality.

Methods: We utilized data from the National Health and Nutrition Examination Survey (1999-2006).

Trial of Anti-BDCA2 Antibody Litifilimab for Cutaneous Lupus Erythematosus.

Background: Blood dendritic cell antigen 2 (BDCA2) is a receptor that is exclusively expressed on plasmacytoid dendritic cells, which are implicated in the pathogenesis of lupus erythematosus. Whether treatment with litifilimab, a humanized monoclonal antibody against BDCA2, would be efficacious in reducing disease activity in patients with cutaneous lupus erythematosus has not been extensively studied.

Methods: In this phase 2 trial, we randomly assigned adults with histologically confirmed cutaneous lupus erythematosus with or without systemic manifestations in a 1:1:1:1 ratio to receive subcutaneous litifilimab (at a dose of 50, 150, or 450 mg) or placebo at weeks 0, 2, 4, 8, and 12.

Urine Uromodulin Is Not Associated With Blood Pressure in the Chronic Kidney Disease in Children Cohort.

Background: Uromodulin regulates activity of the sodium-potassium-two-chloride transporter in the loop of Henle. In adults, higher urine uromodulin levels are associated with greater rise in blood pressure (BP) in response to salt intake. We hypothesized that higher urine uromodulin levels would be associated with higher BP in children with chronic kidney disease, and that there would be an interaction of dietary sodium on this association.

Vitamin D supplementation in children and young adults with persistent proteinuria secondary to glomerular disease.

Background: Vitamin D deficiency is common in glomerular disease. Supplementation may be ineffective due to ongoing urinary losses of vitamin D binding protein. We sought to determine if daily cholecalciferol supplementation would increase vitamin D concentrations in children with glomerular disease and persistent proteinuria, without adverse effects.

Patterns of Use, Outcomes, and Resource Utilization among Recipients of Commercial Axicabtagene Ciloleucel and Tisagenlecleucel for Relapsed/Refractory Aggressive B Cell Lymphomas.

Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are CD19-directed chimeric antigen receptor (CAR) T cell therapies approved for the treatment of relapsed/refractory aggressive B cell lymphomas. We present a multicenter retrospective study among centers that prescribe either commercial product to evaluate usage patterns, safety and efficacy outcomes, and resource utilization. Data collection included all patients from 8 US centers who underwent apheresis between May 1, 2018, and July 31, 2019.

ORIGINS OF THE OPIOID CRISIS AND ITS ENDURING IMPACTS.

Overdose deaths involving opioids have increased dramatically since the 1990s, leading to the worst drug overdose epidemic in U.S. history, but there is limited empirical evidence about the initial causes.

Experience of the COVID-19 pandemic and support for safety-net expansion.

Did individuals' experiences with the harms of the COVID-19 pandemic influence their attitudes towards safety-net programs? To assess this question, we combine rich information about county-level impacts and individual-level perceptions of the early pandemic, repeated measurements of attitudes towards safety-net expansion, and pre-pandemic measurements of related political attitudes. Individuals facing higher county-level impact or greater perceived risks are more likely to support long-term expansions to unemployment insurance and government-provided healthcare when surveyed in June 2020. These differences persist across time, with experiences in the early months of the pandemic remaining strongly predictive of attitudes towards safety-net expansion in early 2021.

KIR-favorable TCR-αβ/CD19-depleted haploidentical HCT in children with ALL/AML/MDS: primary analysis of the PTCTC ONC1401 trial.

We performed a prospective multicenter study of T-cell receptor αβ (TCR-αβ)/CD19-depleted haploidentical hematopoietic cell transplantation (HCT) in children with acute leukemia and myelodysplastic syndrome (MDS), to determine 1-year disease-free survival (DFS) and compare 2-year outcomes with recipients of other donor cell sources. Fifty-one patients aged 0.7 to 21 years were enrolled; donors were killer immunoglobulin-like receptor (KIR) favorable based on ligand mismatch and/or high B content.

Patient-reported outcome measures for life engagement in mental health: a systematic review.

Background: Life engagement in the context of mental health is a broad term that describes positive health aspects relating to cognition, vitality, motivation and reward, and the ability to feel pleasure-concepts that are meaningful to patients. The aim of this systematic literature review was to identify validated patient-reported outcomes (PROs) that can assess any aspect of life engagement in adults, in the field of general mental health.

Methods: This was a systematic literature review of articles in English from the MEDLINE database (date of search: September 9, 2020).

Biomedical Imaging in Experimental Models of Cardiovascular Disease.

Major advances in biomedical imaging have occurred over the last 2 decades and now allow many physiological, cellular, and molecular processes to be imaged noninvasively in small animal models of cardiovascular disease. Many of these techniques can be also used in humans, providing pathophysiological context and helping to define the clinical relevance of the model. Ultrasound remains the most widely used approach, and dedicated high-frequency systems can obtain extremely detailed images in mice.

Neurobehavioral Dimensions of Prader Willi Syndrome: Relationships Between Sleep and Psychosis-Risk Symptoms.

Background: Prader Willi Syndrome (PWS) is a genetic disorder caused by the absence of expression of the paternal copies of maternally imprinted gene(s) located at 15q11-q13. While the physical and medical characteristics of PWS, including short stature, hyperphagia and endocrine dysfunction are well-characterized, systematic investigation of the long-recognized psychiatric manifestations has been recent.

Methods: Here, we report on the first remote (web-based) assessment of neurobehavioral traits, including psychosis-risk symptoms (Prodromal Questionnaire-Brief Version; PQ-B) and sleep behaviors (Pittsburgh Sleep Quality Index), in a cohort of 128 participants with PWS, of whom 48% had a paternal deletion, 36% uniparental disomy, 2.

Cross-sectional characteristics of pediatric-onset discoid lupus erythematosus: Results of a multicenter, retrospective cohort study.

Background: The incidence of systemic lupus in children with discoid lupus is unknown.

Objective: This study assessed the baseline characteristics of patients with pediatric discoid lupus erythematosus (pDLE).

Methods: Medical records at 17 sites were reviewed for pediatric dermatology and rheumatology patients with discoid lupus erythematosus.

Biological impact of iberdomide in patients with active systemic lupus erythematosus.

Objectives: Iberdomide is a high-affinity cereblon ligand that promotes proteasomal degradation of transcription factors Ikaros () and Aiolos (). Pharmacodynamics and pharmacokinetics of oral iberdomide were evaluated in a phase 2b study of patients with active systemic lupus erythematosus (SLE).

Methods: Adults with autoantibody-positive SLE were randomised to placebo (n=83) or once daily iberdomide 0.