This book provides insight and practical illustrations of how advanced statistical concepts can be applied in prognostic medical research.

A sensible strategy to all three aspects (development, validation, updating) is relevant to provide up-to-date prognostic models that can reliably support medical practice Includes supplementary material: sn.pub/extras Request lecturer material: sn.pub/lecturer-material

Autorentext

Ewout Steyerberg worked for 25 years at Erasmus Medical Center in Rotterdam before moving to Leiden where he is now Professor of Clinical Biostatistics and Medical Decision Making and chair of the Department of Biomedical Data Sciences at Leiden University Medical Center. His research has covered a broad range of methodological and medical topics, which is reflected in hundreds of peer-reviewed methodological and applied publications. His methodological expertise is in the design and analysis of randomized controlled trials, cost-effectiveness analysis, and decision analysis. His methodological research focuses on the development, validation and updating of prediction models, as reflected in a textbook (Springer, 2009). His medical fields of application include oncology, cardiovascular disease, internal medicine, pediatrics, infectious diseases, neurology, surgery and traumatic brain injury.

Klappentext

This book provides insight and practical illustrations on how modern statistical concepts and regression methods can be applied in medical prediction problems, including diagnostic and prognostic outcomes. Many advances have been made in statistical approaches towards outcome prediction, but these innovations are insufficiently applied in medical research. Old-fashioned, data hungry methods are often used in data sets of limited size, validation of predictions is not done or done simplistically, and updating of previously developed models is not considered. A sensible strategy is needed for model development, validation, and updating, such that prediction models can better support medical practice.

Clinical prediction models presents a practical checklist with seven steps that need to be considered for development of a valid prediction model. These include preliminary considerations such as dealing with missing values; coding of predictors; selection of main effects and interactions for a multivariable model; estimation of model parameters with shrinkage methods and incorporation of external data; evaluation of performance and usefulness; internal validation; and presentation formats. The steps are illustrated with many small case-studies and R code, with data sets made available in the public domain. The book further focuses on generalizability of prediction models, including patterns of invalidity that may be encountered in new settings, approaches to updating of a model, and comparisons of centers after case-mix adjustment by a prediction model.

The text is primarily intended for clinical epidemiologists and biostatisticians. It can be used as a textbook for a graduate course on predictive modeling in diagnosis and prognosis. It is beneficial if readers are familiar with common statistical models in medicine: linear regression, logistic regression, and Cox regression. The book is practical in nature. But it provides a philosophical perspective ondata analysis in medicine that goes beyond predictive modeling. In this era of evidence-based medicine, randomized clinical trials are the basis for assessment of treatment efficacy. Prediction models are key to individualizing diagnostic and treatment decision making.

Ewout Steyerberg (1967) is Professor of Medical Decision Making, in particular prognostic modeling, at Erasmus MCUniversity Medical Center Rotterdam, the Netherlands. His work on prediction models was stimulated by various research grants including a fellowship from the Royal Netherlands Academy of Arts and Sciences. He has published over 250 peer-reviewed articles in collaboration with many clinical researchers, both in methodological and medical journals.

Inhalt
Applications of prediction models.- Study design for prediction models.- Statistical Models for Prediction.- Overfitting and optimism in prediction models.- Choosing between alternative statistical models.- Dealing with missing values.- Case study on dealing with missing values.- Coding of Categorical and Continuous Predictors.- Restrictions on candidate predictors.- Selection of main effects.- Assumptions in regression models:Additivity and linearity.- Modern estimation methods.- Estimation with external information.- Evaluation of performance.- Clinical Usefulness.- Validation of Prediction Models.- Presentation formats.- Patterns of external validity.- Updating for a new setting.- Updating for multiple settings.- Prediction of a binary outcome:30-day mortality after acute myocardial infarction.- Case study on survival analysis:prediction of secondary cardiovascular events.- Lessons from case studies.