Look above the IRP: predicting abnormal confirmatory testing in patients with esophagogastric junction outflow obstruction.

Background: Esophagogastric junction outflow obstruction (EGJOO) is a manometric diagnosis based on Chicago Classification version 4.0 (CC4.0) that requires confirmatory testing for clinical relevancy.

Emergency department utilization patterns for pediatric urinary stone patients in the United States.

Background: The prevalence of pediatric urolithiasis has increased rapidly, leading to more emergency department (ED) visits across the United States.

Objective: The purpose of this study was to determine emergency care practices for children and adolescents with urinary stones and characteristics associated with management.

Methods: We performed a cross-sectional study of the 2021 Nationwide Emergency Department Sample to identify pediatric patients (≤21 years) presenting to an ED in the United States with a primary diagnosis of urinary stone disease.

Correlation of the clinical rating scale for tremor with a global assessment.

Objective: The Fahn-Tolosa-Marín Clinical Rating Scale for Tremor (CRST) includes three parts (tremor severity, motor task performance, functional disability) and a separate global assessment of impairment completed by both the patient and examiner. Although the CRST is used to determine tremor severity and the efficacy of treatments for tremor, the instrument may not sufficiently capture the patient's perspective. The objective of this study was to determine the association of the CRST subpart and total scores with the global assessment.

Mediators of epilepsy risk after traumatic brain injury: A 20-year U.S. veteran cohort study.

Objective: Traumatic brain injury (TBI) is a significant risk factor for epilepsy, but little work has explored whether risk of epilepsy after TBI may operate through intermediary mechanisms. The objective of this study was to statistically screen for potentially mediating effects among 64 comorbidities for epilepsy risk following TBI among Post-9/11 U.S.

Machine learning-optimized targeted detection of alternative splicing.

RNA sequencing (RNA-seq) is widely adopted for transcriptome analysis but has inherent biases that hinder the comprehensive detection and quantification of alternative splicing. To address this, we present an efficient targeted RNA-seq method that greatly enriches for splicing-informative junction-spanning reads. Local splicing variation sequencing (LSV-seq) utilizes multiplexed reverse transcription from highly scalable pools of primers anchored near splicing events of interest.

Intestinal oxygen utilisation and cellular adaptation during intestinal ischaemia-reperfusion injury.

The gastrointestinal tract can be deranged by ailments including sepsis, trauma and haemorrhage. Ischaemic injury provokes a common constellation of microscopic and macroscopic changes that, together with the paradoxical exacerbation of cellular dysfunction and death following restoration of blood flow, are collectively known as ischaemia-reperfusion injury (IRI). Although much of the gastrointestinal tract is normally hypoxemic, intestinal IRI results when there is inadequate oxygen availability due to poor supply (pathological hypoxia) or abnormal tissue oxygen use and metabolism (dysoxia).

Time-dependent prognostic accuracy measures for recurrent event data.

In many clinical contexts, the event of interest could occur multiple times for the same patient. Considerable advancement has been made on developing recurrent event models based on or that use biomarker information. However, less attention has been given to evaluating the prognostic accuracy of a biomarker or a composite score obtained from a fitted recurrent event-rate model.

Assessing the benefit-risk profile of newer glucose-lowering drugs: A systematic review and network meta-analysis of randomized outcome trials.

Aim: To comprehensively evaluate the benefits and risks of glucagon-like peptide-1 receptor agonists (GLP-1RA), dipeptidyl peptidase 4 inhibitors (DPP4i), and sodium-glucose cotransporter 2 inhibitors (SGLT2i).

Materials And Methods: A systematic search of PubMed, EMBASE, and Cochrane Central Register of Controlled Trials (CENTRAL) from inception to November 2023 to identify randomized cardiovascular and kidney outcome trials that enrolled adults with type 2 diabetes, heart failure, or chronic kidney disease and compared DPP4i, GLP-1RAs, or SGLT2i to placebo. Twenty-one outcomes (e.

Barriers and facilitators to caregiver comfort with health-related social needs data collection in the pediatric clinical setting.

Objective: To identify barriers and facilitators to family-level comfort with health-related social needs (HRSN) data collection and documentation in the pediatric clinical setting.

Study Setting And Design: This qualitative study was nested within a pragmatic randomized controlled trial on social care integration in the pediatric clinical setting. We used a hybrid random-purposive strategy to sample 60 caregivers of pediatric patients ages 0-25 presenting at two primary care clinics and one emergency department affiliated with a large pediatric healthcare system between September 2022 and 2023.

Integrated Analysis of Vonoprazan Safety for Symptomatic Gastro-Oesophageal Reflux Disease or Erosive Oesophagitis.

Background: Patients with erosive oesophagitis, and those with persistent symptomatic non-erosive gastro-oesophageal reflux disease, require long-term maintenance treatment with acid-suppressing agents.

Aim: To evaluate the safety of vonoprazan, a potassium-competitive acid blocker, in an integrated analysis of data from clinical trials in adults.

Methods: We included 14 clinical trials of vonoprazan conducted in multiple countries.

Real-world effectiveness and causal mediation study of BNT162b2 on long COVID risks in children and adolescents.

Background: The impact of pre-infection vaccination on the risk of long COVID remains unclear in the pediatric population. We aim to assess the effectiveness of BNT162b2 on long COVID risks with various strains of the SARS-CoV-2 virus in children and adolescents, using comparative effectiveness methods. We further explore if such pre-infection vaccination can mitigate the risk of long COVID beyond its established protective benefits against SARS-CoV-2 infection using causal mediation analysis.

Cardiovascular Disease Among Women and Birthing Individuals After Delivering a Child With Congenital Heart Disease.

Background: Individuals have a higher risk of cardiovascular disease later in life if they give birth to a child with congenital heart disease (CHD). The mechanism of this association has not been well documented.

Objectives: The authors aimed to describe the prevalence of cardiovascular disease and risk factors in women and birthing individuals 18 to 23 years after delivery of a child with CHD compared to normative data.

Staged Screening Identifies People with Biomarkers Related to Neuronal Alpha-Synuclein Disease.

Objective: Remote identification of individuals with severe hyposmia may enable scalable recruitment of participants with underlying alpha-synuclein aggregation. We evaluated the performance of a staged screening paradigm using remote smell testing to enrich for abnormal dopamine transporter single-photon emission computed tomography imaging (DAT-SPECT) and alpha-synuclein aggregation.

Methods: The Parkinson's Progression Markers Initiative (PPMI) recruited participants for the prodromal cohort who were 60-years and older without a Parkinson's disease diagnosis.

Pancreaticoduodenectomy in trauma patients with grade IV-V duodenal or pancreatic injuries: a post hoc analysis of an EAST multicenter trial.

Introduction: The utility of pancreaticoduodenectomy (PD) for high-grade traumatic injuries remains unclear and data surrounding its use are limited. We hypothesized that PD does not result in improved outcomes when compared with non-PD surgical management of grade IV-V pancreaticoduodenal injuries.

Methods: This is a retrospective, multicenter analysis from 35 level 1 trauma centers from January 2010 to December 2020.

Automated Insulin Delivery in Older Adults with Type 1 Diabetes.

Background: Older adults with type 1 diabetes are at risk for serious hypoglycemia. Automated insulin delivery can reduce risk but has not been sufficiently evaluated in this population.

Methods: We conducted a multicenter, randomized crossover trial in adults older than or equal to 65 years of age with type 1 diabetes.

Letter to the editor in response to Duan X, et al, CYP4A22 loss-of-function causes a new type of vitamin D-dependent rickets (VDDR1C).

Duan X, et al, report that CYP4A22 loss-of-function causes a new form of vitamin D-dependent rickets. Here we describe the basis for our rejection of their proposal and provide evidence that the CYP4A22 variant that they have identified (c.901del, p.

Thighplasty for Transfemoral Amputations and Osseointegration: Lower Rate of Complications Than Thighplasty for Body Contouring.

Background: Modern techniques in lower extremity amputation have made significant advances to improve prosthetic control and soft-tissue envelopes through various techniques, including medial thighplasties. These advances are necessary to enhance the fit and functionality of the prosthesis in transfemoral amputations.

Methods: We performed a retrospective review of all thighplasties performed at our institution in patients with ipsilateral transfemoral amputation from November 2017 to December 2021.

Operationalizing the New Global Definition of ARDS: A Retrospective Cohort Study From South Africa.

Background: A proposed new global definition of ARDS seeks to update the Berlin definition and account for nonintubated ARDS and ARDS diagnoses in resource-variable settings.

Research Question: How do ARDS epidemiologic characteristics change with operationalizing the new global definition of ARDS in a resource-limited setting?

Study Design And Methods: We performed a real-use retrospective cohort study among adult patients meeting criteria for the Berlin definition of ARDS or the global definition of ARDS at ICU admission in two public hospitals in the KwaZulu-Natal Department of Health, South Africa, from January 2017 through June 2022.

Results: Among 5,760 adults (aged ≥ 18 years) admitted to the ICU, 2,027 patients (35.

Testosterone promotes photoreceptor degeneration in the sodium iodate model.

Previously, we found that retinas of young male mice were more damaged than those of young female mice in the sodium iodate (NaIO) model. The purpose of this study was to test whether reducing testosterone levels would be retina-protective. Male C57Bl/6J mice underwent surgical castration or sham surgery, then were given an intraperitoneal injection of NaIO at 25 mg/kg.

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.

Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.