Meaningful Endpoints for Idiopathic Pulmonary Fibrosis (IPF) Clinical Trials: Emphasis on 'Feels, Functions, Survives'. Report of a Collaborative Discussion in a Symposium with Direct Engagement from Representatives of Patients, Investigators, the National Institutes of Health, a Patient Advocacy Organization, and a Regulatory Agency.

Idiopathic pulmonary fibrosis (IPF) carries significant mortality and unpredictable progression, with limited therapeutic options. Designing trials with patient-meaningful endpoints, enhancing the reliability and interpretability of results, and streamlining the regulatory approval process are of critical importance to advancing clinical care in IPF. A landmark in-person symposium in June 2023 assembled 43 participants from the US and internationally, including patients with IPF, investigators, and regulatory representatives, to discuss the immediate future of IPF clinical trial endpoints.

The Canadian Study of Arterial Inflammation in Patients with Diabetes and Recent Vascular Events, Evaluation of Colchicine Effectiveness (CADENCE): protocol for a randomised, double-blind, placebo-controlled trial.

Background: Inflammation is a key mediator in the development and progression of the atherosclerotic disease process as well as its resultant complications, like myocardial infarction (MI), stroke and cardiovascular (CV) death, and is emerging as a novel treatment target. Trials involving anti-inflammatory medications have demonstrated outcome benefit in patients with known CV disease. In this regard, colchicine appears to hold great promise.

Metabolic activity of the left and right atria are differentially altered in patients with atrial fibrillation and LV dysfunction.

Background: Alterations in atrial metabolism may play a role in the perpetuation of atrial fibrillation (AF). This study sought to compare F-fluorodeoxyglucose (FDG) uptake on PET, in patients with LV dysfunction versus those without AF.

Methods: Seventy-two patients who underwent myocardial viability assessment were evaluated.

Solitary iliac branch endoprosthesis placement for iliac artery aneurysms.

Background: Isolated iliac artery aneurysms (IAAs), accounting for 2% to 7% of all abdominal aneurysms, are often treated with the use of iliac branched endografts. Although outside the manufacturer's instructions for use, iliac branched devices can be used solely, without the adjunctive placement of an endovascular aneurysm repair device, for the treatment of an isolated IAA. In the present study, we have described the outcomes of the use of the Gore iliac branched endoprosthesis (IBE; W.

Generation of an iPSC line (AKOSi004-A) from fibroblasts of a female adult NPC1 patient, carrying the compound heterozygous mutation p.Val1023Serfs*15/p.Gly992Arg and of an iPSC line (AKOSi005-A) from a female adult control individual.

Niemann-Pick disease Type C (NPC) is a rare progressive neurodegenerative disorder with an incidence of 1:120,000 caused by mutations in the NPC1 or NPC2 gene leading to a massive cholesterol accumulation. Here, we describe the generation of induced pluripotent stem cells (iPSCs) of an affected female adult individual carrying the NPC1 mutation p.Val1023Serfs*15/p.

Regional Distribution of Fluorine-18-Flubrobenguane and Carbon-11-Hydroxyephedrine for Cardiac PET Imaging of Sympathetic Innervation.

Objectives: The aim of this study was to investigate the regional distribution of novel F-labeled positron emission tomographic (PET) tracer flubrobenguane (FBBG) (whose longer half-life could enable more widespread use) to assess myocardial presynaptic sympathetic nerve function in humans in comparison to [C]meta-hydroxyephedrine (HED).

Background: The sympathetic nervous system (SNS) is vitally linked to cardiovascular regulation and disease. SNS imaging has shown prognostic value.

Computed tomography coronary angiography for patients with heart failure (CTA-HF): a randomized controlled trial (IMAGE-HF 1C).

Aims: This randomized controlled trial sought to determine the financial impact of an initial diagnostic strategy of coronary computed tomography angiography (CCTA) in patients with heart failure (HF) of unknown aetiology. Invasive coronary angiography (ICA) is used to investigate HF patients. CCTA may be a non-invasive cost-effective alternative to ICA.

OUTSMART HF: A Randomized Controlled Trial of Routine Versus Selective Cardiac Magnetic Resonance for Patients With Nonischemic Heart Failure (IMAGE-HF 1B).

Background: Cardiac magnetic resonance (CMR) is a recommended imaging test for patients with heart failure (HF); however, there is a lack of evidence showing incremental benefit over transthoracic echocardiography. Our primary hypothesis was that routine use of CMR will yield more specific diagnoses in nonischemic HF. Our secondary hypothesis was that routine use of CMR will improve patient outcomes.

Correction to: PET imaging of sympathetic innervation with [F]Flubrobenguane vs [C]mHED in a patient with ischemic cardiomyopathy.

At time of initial publication, the USAN Council had assigned the generic name for LMI1195 as Flurobenguan. However, the Council has since changed and finalized this compound name as Flubrobenguane which is recommended as the generic name to be used in the future.

[18F]-Fluorodeoxyglucose PET/CT imaging as a marker of carotid plaque inflammation: Comparison to immunohistology and relationship to acuity of events.

Background: [18F]-fluorodeoxyglucose (FDG) uptake imaged with positron emission tomography (PET) and computed tomography (CT) may serve as a biomarker of plaque inflammation. This study evaluated the relationship between carotid plaque FDG uptake and a) intraplaque expression of macrophage and macrophage-like cellular CD68 immunohistology; b) intraplaque inflammatory burden using leukocyte-sensitive CD45 immunohistology; c) symptomatic patient presentation; d) time from last cerebrovascular event.

Methods: 54 patients scheduled for carotid endarterectomy underwent FDG PET/CT imaging.

Prognostic utility of splenic response ratio in dipyridamole PET myocardial perfusion imaging.

Background: Cardiac magnetic resonance perfusion studies with adenosine stress have shown that splenic response can identify patients with inadequate pharmacologic stress. We investigate the incremental prognostic impact of splenic response ratio (SRR) in patients with normal Rubidium (Rb)-82 PET myocardial perfusion imaging (MPI).

Methods: Consecutive patients undergoing dipyridamole Rb-82 PET MPI for the evaluation of coronary artery disease were screened.

Recent Trends in Baccalaureate-Prepared Registered Nurses in U.S. Acute Care Hospital Units, 2004-2013: A Longitudinal Study.

Purpose: To examine the trends in baccalaureate (bachelor of science in nursing)-prepared registered nurses (BSN RNs) in U.S. acute care hospital units and to project the growth in the number of BSN RNs by 2020.

N-Terminal Pro B-Type Natriuretic Peptide and High-Sensitivity Cardiac Troponin T Levels Are Related to the Extent of Hibernating Myocardium in Patients With Ischemic Heart Failure.

Background: Increased N-terminal pro b-type natriuretic peptide (NT-proBNP) and high-sensitivity cardiac troponin T (hs-cTnT) can identify patients with heart failure (HF) who are at increased risk of cardiac events. The relationship of these biomarkers to the extent of hibernating myocardium and scar has not been previously characterized in patients with ischemic left ventricular dysfunction and HF.

Methods: Patients with ischemic HF meeting recruitment criteria and undergoing perfusion and fluorodeoxyglucose-positron emission tomography to define myocardial hibernation and scar were included in the study.

Subgroup identification of early preterm birth (ePTB): informing a future prospective enrichment clinical trial design.

Background: Despite the widely recognized association between the severity of early preterm birth (ePTB) and its related severe diseases, little is known about the potential risk factors of ePTB and the sub-population with high risk of ePTB. Moreover, motivated by a future confirmatory clinical trial to identify whether supplementing pregnant women with docosahexaenoic acid (DHA) has a different effect on the risk subgroup population or not in terms of ePTB prevalence, this study aims to identify potential risk subgroups and risk factors for ePTB, defined as babies born less than 34 weeks of gestation.

Methods: The analysis data (N = 3,994,872) were obtained from CDC and NCHS' 2014 Natality public data file.

Confirmatory Factor Analysis Alternative: Free, Accessible CBID Software.

New software that performs Classical and Bayesian Instrument Development (CBID) is reported that seamlessly integrates expert (content validity) and participant data (construct validity) to produce entire reliability estimates with smaller sample requirements. The free CBID software can be accessed through a website and used by clinical investigators in new instrument development. Demonstrations are presented of the three approaches using the CBID software: (a) traditional confirmatory factor analysis (CFA), (b) Bayesian CFA using flat uninformative prior, and (c) Bayesian CFA using content expert data (informative prior).

A novel method for expediting the development of patient-reported outcome measures and an evaluation across several populations.

Item response theory (IRT) models provide an appropriate alternative to the classical ordinal confirmatory factor analysis (CFA) during the development of patient-reported outcome measures (PROMs). Current literature has identified the assessment of IRT model fit as both challenging and underdeveloped (Sinharay & Johnson, 2003; Sinharay, Johnson, & Stern, 2006). This study evaluates the performance of Ordinal Bayesian Instrument Development (OBID), a Bayesian IRT model with a probit link function approach, through applications in two breast cancer-related instrument development studies.

Long-Term Follow-Up of Outcomes With F-18-Fluorodeoxyglucose Positron Emission Tomography Imaging-Assisted Management of Patients With Severe Left Ventricular Dysfunction Secondary to Coronary Disease.

Background: Whether viability imaging can impact long-term patient outcomes is uncertain. The PARR-2 study (Positron Emission Tomography and Recovery Following Revascularization) showed a nonsignificant trend toward improved outcomes at 1 year using an F-18-fluorodeoxyglucose positron emission tomography (PET)-assisted strategy in patients with suspected ischemic cardiomyopathy. When patients adhered to F-18-fluorodeoxyglucose PET recommendations, outcome benefit was observed.

A novel method for expediting the development of patient-reported outcome measures and an evaluation of its performance via simulation.

Background: Developing valid and reliable patient-reported outcome measures (PROMs) is a critical step in promoting patient-centered health care, a national priority in the U.S. Small populations or rare diseases often pose difficulties in developing PROMs using traditional methods due to small samples.

Caffeine intake during pregnancy and adverse birth outcomes: a systematic review and dose-response meta-analysis.

Caffeine is commonly consumed during pregnancy, crosses the placenta, with fetal serum concentrations similar to the mother's, but studies of birth outcome show conflicting findings. We systematically searched Medline and Embase for relevant publications. We conducted meta-analysis of dose-response curves for associations between caffeine intake and spontaneous abortion, stillbirth, preterm delivery, low birth weight and small for gestational age (SGA) infants.

Reliability and Validity of the NDNQI® Injury Falls Measure.

Although remarkable efforts have been made to improve patient fall reporting through the utilization of standardized definitions, injury falls reporting has rarely been examined. This study used an overall intra-class correlation coefficient (ICC) estimate and factor analysis to assess the reliability and validity of the National Database of Nursing Quality Indicators® (NDNQI®) falls with injury measure. Data were collected from an online Fall Injury Level Survey that was administered to 1,159 NDNQI site coordinators (39.

Effects of short-term continuous positive airway pressure on myocardial sympathetic nerve function and energetics in patients with heart failure and obstructive sleep apnea: a randomized study.

Background: Heart failure with reduced ejection fraction and obstructive sleep apnea (OSA), 2 states of increased metabolic demand and sympathetic nervous system activation, often coexist. Continuous positive airway pressure (CPAP), which alleviates OSA, can improve ventricular function. It is unknown whether this is due to altered oxidative metabolism or presynaptic sympathetic nerve function.

Computed tomographic coronary angiography for patients with heart failure (CTA-HF): a randomized controlled trial (IMAGE HF Project 1-C).

Background: The prevalence of heart failure (HF) is rising in industrialized and developing countries. Though invasive coronary angiography (ICA) remains the gold standard for anatomical assessment of coronary artery disease in HF patients, alternatives are being sought. Computed tomographic coronary angiography (CTA) has emerged as an accurate non-invasive diagnostic tool for coronary artery disease (CAD) and has been demonstrated to have prognostic value.

Routine versus selective cardiac magnetic resonance in non-ischemic heart failure - OUTSMART-HF: study protocol for a randomized controlled trial (IMAGE-HF (heart failure) project 1-B).

Background: Imaging has become a routine part of heart failure (HF) investigation. Echocardiography is a first-line test in HF given its availability and it provides valuable diagnostic and prognostic information. Cardiac magnetic resonance (CMR) is an emerging clinical tool in the management of patients with non-ischemic heart failure.