Methods for Stratification and Validation Cohorts: A Scoping Review.

Area of Science:

• Biomedical research
• Personalized medicine
• Clinical cohort studies

Background:

• Personalized medicine necessitates large patient cohorts for effective stratification and validation.
• Current practices for designing and managing these cohorts lack standardized methods and tools.
• This deficiency impacts the reproducibility and robustness of personalized medicine research.

Purpose of the Study:

• To conduct a scoping review of the state-of-the-art in methods and tools for designing and managing cohorts in personalized medicine.
• To identify existing practices and highlight areas needing standardization.

Main Methods:

• A comprehensive scoping review was performed using major scientific databases (PubMed, EMBASE, Web of Science, Psycinfo, Cochrane Library).
• Searches focused on reviews concerning tools and methods for cohorts in cancer, stroke, and Alzheimer's disease (2005-April 2020).
• PRISMA guidelines were followed for screening and inclusion of 50 reviews.

Main Results:

• Most included reviews (25/50) detailed data generation methods, and (24/50) discussed data management and analysis tools.
• Significant gaps were identified regarding data quality monitoring and requirements for associated clinical data.
• A scarcity of information and standards was noted for critical aspects like sample size calculation.

Conclusions:

• The current landscape of cohort design and management in personalized medicine reveals a need for standardized guidelines.
• Addressing identified gaps in data quality, monitoring, and sample size calculation is crucial.
• Developing comprehensive guidelines will enhance the reproducibility and robustness of future personalized medicine studies.