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Absolute Risk: Methods and Applications in Clinical Management and Public Health provides theory and examples to demonstrate the importance of absolute risk in counseling patients, devising public health strategies, and clinical management. The book provides sufficient technical detail to allow statisticians, epidemiologists, and clinicians to build, test, and apply models of absolute risk. Features: Provides theoretical basis for modeling absolute risk, including competing risks and cause-specific and cumulative incidence regression Discusses various sampling designs for estimating absolute risk and criteria to evaluate models Provides details on statistical inference for the various sampling designs Discusses criteria for evaluating risk models and comparing risk models, including both general criteria and problem-specific expected losses in well-defined clinical and public health applications Describes many applications encompassing both disease prevention and prognosis, and ranging from counseling individual patients, to clinical decision making, to assessing the impact of risk-based public health strategies Discusses model updating, family-based designs, dynamic projections, and other topics Ruth M. Pfeiffer is a mathematical statistician and Fellow of the American Statistical Association, with interests in risk modeling, dimension reduction, and applications in epidemiology. She developed absolute risk models for breast cancer, colon cancer, melanoma, and second primary thyroid cancer following a childhood cancer diagnosis. Mitchell H. Gail developed the widely used "Gail model" for projecting the absolute risk of invasive breast cancer. He is a medical statistician with interests in statistical methods and applications in epidemiology and molecular medicine. He is a member of the National Academy of Medicine and former President of the American Statistical Association. Both are Senior Investigators in the Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health.
This book provides a groundbreaking introduction to the likelihood inference for correlated survival data via the hierarchical (or h-) likelihood in order to obtain the (marginal) likelihood and to address the computational difficulties in inferences and extensions. The approach presented in the book overcomes shortcomings in the traditional likelihood-based methods for clustered survival data such as intractable integration. The text includes technical materials such as derivations and proofs in each chapter, as well as recently developed software programs in R (“frailtyHL”), while the real-world data examples together with an R package, “frailtyHL” in CRAN, provide readers with useful hands-on tools. Reviewing new developments since the introduction of the h-likelihood to survival analysis (methods for interval estimation of the individual frailty and for variable selection of the fixed effects in the general class of frailty models) and guiding future directions, the book is of interest to researchers in medical and genetics fields, graduate students, and PhD (bio) statisticians.
Survival data analysis is a very broad field of statistics, encompassing a large variety of methods used in a wide range of applications, and in particular in medical research. During the last twenty years, several extensions of "classical" survival models have been developed to address particular situations often encountered in practice. This book aims to gather in a single reference the most commonly used extensions, such as frailty models (in case of unobserved heterogeneity or clustered data), cure models (when a fraction of the population will not experience the event of interest), competing risk models (in case of different types of event), and joint survival models for a time-to-event endpoint and a longitudinal outcome. Features Presents state-of-the art approaches for different advanced survival models including frailty models, cure models, competing risk models and joint models for a longitudinal and a survival outcome Uses consistent notation throughout the book for the different techniques presented Explains in which situation each of these models should be used, and how they are linked to specific research questions Focuses on the understanding of the models, their implementation, and their interpretation, with an appropriate level of methodological development for masters students and applied statisticians Provides references to existing R packages and SAS procedure or macros, and illustrates the use of the main ones on real datasets This book is primarily aimed at applied statisticians and graduate students of statistics and biostatistics. It can also serve as an introductory reference for methodological researchers interested in the main extensions of classical survival analysis.
This book is an accessible, practical and comprehensive guide for researchers from multiple disciplines including biomedical, epidemiology, engineering and the social sciences. Written for accessibility, this book will appeal to students and researchers who want to understand the basics of survival and event history analysis and apply these methods without getting entangled in mathematical and theoretical technicalities. Inside, readers are offered a blueprint for their entire research project from data preparation to model selection and diagnostics. Engaging, easy to read, functional and packed with enlightening examples, ‘hands-on’ exercises, conversations with key scholars and resources for both students and instructors, this text allows researchers to quickly master advanced statistical techniques. It is written from the perspective of the ‘user’, making it suitable as both a self-learning tool and graduate-level textbook. Also included are up-to-date innovations in the field, including advancements in the assessment of model fit, unobserved heterogeneity, recurrent events and multilevel event history models. Practical instructions are also included for using the statistical programs of R, STATA and SPSS, enabling readers to replicate the examples described in the text.
Interval-Censored Time-to-Event Data: Methods and Applications collects the most recent techniques, models, and computational tools for interval-censored time-to-event data. Top biostatisticians from academia, biopharmaceutical industries, and government agencies discuss how these advances are impacting clinical trials and biomedical research. Divided into three parts, the book begins with an overview of interval-censored data modeling, including nonparametric estimation, survival functions, regression analysis, multivariate data analysis, competing risks analysis, and other models for interval-censored data. The next part presents interval-censored methods for current status data, Bayesian semiparametric regression analysis of interval-censored data with monotone splines, Bayesian inferential models for interval-censored data, an estimator for identifying causal effect of treatment, and consistent variance estimation for interval-censored data. In the final part, the contributors use Monte Carlo simulation to assess biases in progression-free survival analysis as well as correct bias in interval-censored time-to-event applications. They also present adaptive decision making methods to optimize the rapid treatment of stroke, explore practical issues in using weighted logrank tests, and describe how to use two R packages. A practical guide for biomedical researchers, clinicians, biostatisticians, and graduate students in biostatistics, this volume covers the latest developments in the analysis and modeling of interval-censored time-to-event data. It shows how up-to-date statistical methods are used in biopharmaceutical and public health applications.
Making complex methods more accessible to applied researchers without an advanced mathematical background, the authors present the essence of new techniques available, as well as classical techniques, and apply them to data. Practical suggestions for implementing the various methods are set off in a series of practical notes at the end of each section, while technical details of the derivation of the techniques are sketched in the technical notes. This book will thus be useful for investigators who need to analyse censored or truncated life time data, and as a textbook for a graduate course in survival analysis, the only prerequisite being a standard course in statistical methodology.