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Bayesian Sample Size Calculation in Small n, Sequential Multiple Assignment Randomized Trials (snSMART).
Pharm Stat
January 2025
Department of Biostatistics, School of Public Health, University of Michigan, Ann Arbor, Michigan, USA.
A recent study design for clinical trials with small sample sizes is the small n, sequential, multiple assignment, randomized trial (snSMART). An snSMART design has been previously proposed to compare the efficacy of two dose levels versus placebo. In such a trial, participants are initially randomized to receive either low dose, high dose or placebo in stage 1.
View Article and Find Full Text PDFMycophenolate mofetil: an update on its mechanism of action and effect on lymphoid tissue.
Front Immunol
January 2025
Department of Pharmacology and Toxicology, Faculty of Veterinary Medicine, University of Warmia and Mazury in Olsztyn, Olsztyn, Poland.
Introduction: Mycophenolate mofetil (MMF) is an immunosuppressive drug administered in the management of both autoimmune diseases and organ transplantation. The main aims of the study were: (a) to obtain information regarding the safety of using MMF in respect of its effect on normal T and B cells in lymphoid tissues; (b) to investigate whether the generation of inducible Foxp3-expressing regulatory T cells (Treg) might constitute additional mechanisms underlying the immunosuppressive properties of MMF.
Methods: The effect of MMF ( studies) and its active metabolite, mycophenolic acid, ( studies) on murine CD4 and CD8 T cells as well as B cells was determined, regarding: (a) absolute count, proliferation and apoptosis of these cells ( studies); (b) absolute count of these cells in the head and neck lymph nodes, mesenteric lymph nodes and the spleen ( studies).
Advances in genetic diagnosis and therapy of hereditary heart disease: a bibliometric review from 2004 to 2024.
Front Med (Lausanne)
January 2025
Key Laboratory of Obstetric and Gynecologic and Pediatric Diseases and Birth Defects of Ministry of Education, Department of Radiology, West China Second University Hospital, Sichuan University, Chengdu, China.
Hereditary heart disease (HHD) is a series of cardiac disorders associated with monogenic or polygenic abnormalities and is one of the leading causes of sudden death, particularly in young adults. The updated European Cardiology guideline for cardiomyopathies provides the first comprehensive summary of genotyping, imaging, and therapy recommendations for inherited cardiomyopathies, but still lacks a comprehensive discussion of research advances and future trends in genetic diagnosis and therapy of HHD. Our research aims to fill this gap.
View Article and Find Full Text PDFA Cross-sectional Survey of Physicians to Understand Biomarker Testing and Treatment Patterns in Patients with Prostate Cancer in the USA, EU5, Japan, and China.
Eur Urol Open Sci
January 2025
Merck & Co. Inc, Rahway, NJ, USA.
Background And Objective: Treatment landscape in advanced prostate cancer (PC) is evolving. There is limited understanding of the factors influencing decision-making for genetic/genomic testing and the barriers to recommending testing and treatment in international real-world clinical practice following the approval of poly-adenosine diphosphate-ribose polymerase inhibitors (PARPi) for metastatic castration-resistant PC (mCRPC). This work aims to assess genetic/genomic testing patterns and methods, including for homologous recombination repair mutation (HRRm), and treatment decisions among physicians caring for patients with PC across the USA, Europe, and Asia.
View Article and Find Full Text PDFNew COVID-19 vaccination recommendations in Spain: Optimizing for next seasons.
Enferm Infecc Microbiol Clin
January 2025
Centro Nacional de Gripe de Valladolid (GISRS/WHO), Spain.
Despite high initial vaccination rates, Spain's current COVID-19 vaccination coverage in recommended groups does not meet WHO targets. For the upcoming season, challenges include revising vaccination age, updating risk groups, and unifying criteria with flu vaccine co-administration. European Commission's advance purchase agreements limit access to certain vaccines, and the need for vaccines effective against current variants adds administrative complexities.
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