An Overview of Current Methods for Real-world Applications to Generalize or Transport Clinical Trial Findings to Target Populations of Interest.

It has been well established that randomized clinical trials have poor external validity, resulting in findings that may not apply to relevant-or target-populations. When the trial is sampled from the target population, generalizability methods have been proposed to address the applicability of trial findings to target populations. When the trial sample and target populations are distinct, transportability methods may be applied for this purpose.

An observational analysis of disease burden in patients with cold agglutinin disease: Results from a large US electronic health record database.

Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia (AIHA). Information regarding the impact of CAD from the patient and health care system perspective is limited. To understand longitudinal trends in outcomes in patients with CAD, including anemia severity, hemolytic status, administration of CAD-related therapies, and health care resource utilization (HCRU).

Data Mining of Free-Text Responses: An Innovative Approach to Analyzing Patient Perspectives on Treatment for Chronic Rhinosinusitis with Nasal Polyps in a Phase IIa Proof-of-Concept Study for Dupilumab.

Purpose: Patient perspective is an important and increasingly sought-after complement to clinical assessment. The aim of this study was to transcribe individual patients' experience of treatment in a dupilumab clinical trial through free-text responses with analysis using natural language processing (NLP) to obtain the unique perspective of patients on disease impact and unmet needs with existing treatment to inform future trial design.

Patients And Methods: Patients with chronic rhinosinusitis with nasal polyps (CRSwNP) who were enrolled in a Phase IIa randomized controlled trial comparing dupilumab with placebo (NCT01920893) were invited to complete a self-assessment of treatment (SAT) tool at the end of treatment, asking, "What is your opinion on the treatment you had during the trial? What did you like or dislike about the treatment?" Free-text responses were analyzed for the overall cohort and according to treatment assignment using natural language processing including sentiment scoring.

Cardiac magnetic resonance features of left dominant arrhythmogenic cardiomyopathy: differential diagnosis with myocarditis.

Cardiac magnetic resonance (CMR) findings suggesting a suspected left-dominant arrhythmogenic cardiomyopathy (LDAC) may be difficult to distinguish from those related to previous myocarditis; however, especially in patients with ventricular arrhythmias (VA) with ECG morphology consistent with a left ventricle (LV) origin differential diagnosis is fundamental. Aim of the study was to identify potential imaging features at CMR specific for LDAC diagnosis. Between January 2011 and December 2019, we enrolled 15 consecutive stable patients with a recent diagnosis of significant VA and ECG morphology consistent with a LV origin, detection of potential LV arrhythmic substrate at CMR and undergoing a clinically-indicated LV endomyocardial biopsy showing tissue abnormalities consistent with the diagnosis of LDAC.