Since the advent of the phrase "subgroup identification," there has been an explosion of methodologies that seek to identify meaningful subgroups of patients with exceptional response in order to further the realization of personalized medicine. However, to perform fair comparison and understand what methods work best under different clinical trials situations, a common platform is needed for comparative effectiveness of these various approaches. In this paper, we describe a comprehensive project that created an extensive platform for evaluating subgroup identification methods as well as a publicly posted challenge that was used to elicit new approaches. We proposed a common data-generating model for creating virtual clinical trial datasets that contain subgroups of exceptional responders encompassing the many dimensions of the problem or null scenarios in which there are no such subgroups. Furthermore, we created a common scoring system for evaluating performance of purported methods for identifying subgroups. This makes it possible to benchmark methodologies in order to understand what methods work best under different clinical trial situations. The findings from this project produced considerable insights and allow us to make recommendations for how the statistical community can better compare and contrast old and new subgroup identification methodologies.
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http://dx.doi.org/10.1002/bimj.202200164 | DOI Listing |
Pathogens
January 2025
Aklilu Lemma Institute of Pathobiology, Addis Ababa University, Addis Ababa P.O. Box 1176, Ethiopia.
Anthrax is a zoonotic disease characterized by rapid onset with usual fatal outcomes in livestock and wildlife. In Ethiopia, anthrax is a persistent disease; however, there are limited data on the isolation and molecular characterization of strains. This study aimed to characterize isolated from animal anthrax outbreaks between 2019 and 2024, from different localities in Ethiopia.
View Article and Find Full Text PDFGenes (Basel)
January 2025
Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padua Medical School, 35128 Padova, Italy.
Since its first pathological description over 65 years ago, hypertrophic cardiomyopathy (HCM), with a worldwide prevalence of 1:500, has emerged as the most common genetically determined cardiac disease. Diagnostic work-up has dramatically improved over the last decades, from clinical suspicion and abnormal electrocardiographic findings to hemodynamic studies, echocardiography, contrast-enhanced cardiac magnetic resonance, and genetic testing. The implementation of screening programs and the use of implantable cardioverter defibrillators (ICDs) for high-risk individuals have notably reduced arrhythmic sudden deaths, altering the disease's mortality profile.
View Article and Find Full Text PDFSci Rep
January 2025
Sanofi R&D - Translational Medicine & Early Development - Translational Precision Medicine, 13 Quai Jules Guesde, 94400, Vitry-sur-Seine, France.
Precision medicine is defined by the U.S. Food & Drug Administration as "an innovative approach to tailoring disease prevention and treatment that considers differences in people's genes, environments, and lifestyles".
View Article and Find Full Text PDFJ Cell Mol Med
January 2025
Department of General Surgery, Nanfang Hospital, Southern Medical University, Guangdong Provincial Engineering Technology Research Center of Minimally Invasive Surgery, Guangzhou, People's Republic of China.
Cellular senescence is a key promoter of tumorigenesis and malignant progression. This study aimed to develop a predictive model for assessing cellular senescence in gastric cancer (GC) outcomes. We identified senescence-related genes and lncRNAs from 375 stomach adenocarcinoma (STAD) patients and established a prognostic senescence score using multivariate Cox regression, validated in testing, TCGA-STAD and the combined TCGA-COAD and READ cohorts.
View Article and Find Full Text PDFIntroduction: Access to care varies by sociodemographic group, with some groups facing higher barriers to care than others. This study will use novel methods to explore barriers and potential solutions as perceived by members of the population groups who are least able to access care. We aim to use rapid yet robust mixed methods that allow us to identify generalisable findings within each programme and testable service modifications to improve equitable access to care; delivering non-tokenistic findings within a matter of weeks.
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