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Interval-Censored Time-to-Event Data: Methods and Applications PDF

426 Pages·2012·3.11 MB·English
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I Statistics n t e r v Interval-Censored Time-to-Event Data: Methods and Applications a collects the most recent techniques, models, and computational tools for l - interval-censored time-to-event data. Top biostatisticians from academia, C Interval-Censored biopharmaceutical industries, and government agencies discuss how e these advances are impacting clinical trials and biomedical research. n s Time-to-Event Data Divided into three parts, the book begins with an overview of interval- o censored data modeling, including nonparametric estimation, survival r e functions, regression analysis, multivariate data analysis, competing d risks analysis, and other models for interval-censored data. The next part Methods and Applications T presents interval-censored methods for current status data, Bayesian i semiparametric regression analysis of interval-censored data with m monotone splines, Bayesian inferential models for interval-censored data, e an estimator for identifying causal effect of treatment, and consistent - t variance estimation for interval-censored data. In the final part, the o contributors use Monte Carlo simulation to assess biases in progression- - E free survival analysis as well as correct bias in interval-censored time-to- v event applications. They also present adaptive decision-making methods e to optimize the rapid treatment of stroke, explore practical issues in using n weighted logrank tests, and describe how to use two R packages. t D A practical guide for biomedical researchers, clinicians, biostatisticians, a and graduate students in biostatistics, this volume covers the latest t developments in the analysis and modeling of interval-censored time- a to-event data. It shows how up-to-date statistical methods are used in biopharmaceutical and public health applications. Edited by Ding-Geng (Din) Chen P C e S h a u Jianguo Sun e c n n e Karl E. Peace K14515 K14515_Cover.indd 1 6/1/12 3:07 PM Interval-Censored Time-to-Event Data Methods and Applications K14515_FM.indd 1 5/31/12 9:53 AM Editor-in-Chief Shein-Chung Chow, Ph.D. Professor Department of Biostatistics and Bioinformatics Duke University School of Medicine Durham, North Carolina Series Editors Byron Jones Jen-pei Liu Biometrical Fellow Professor Statistical Methodology Division of Biometry Department of Agronomy Integrated Information Sciences National Taiwan University Novartis Pharma AG Taipei, Taiwan Basel, Switzerland Karl E. Peace Bruce W. Turnbull Georgia Cancer Coalition Professor Distinguished Cancer Scholar School of Operations Research Senior Research Scientist and and Industrial Engineering Professor of Biostatistics Cornell University Jiann-Ping Hsu College of Public Health Ithaca, New York Georgia Southern University Statesboro, Georgia K14515_FM.indd 2 5/31/12 9:53 AM Adaptive Design Methods in Clinical Trial Methodology Clinical Trials, Second Edition Karl E. Peace and Ding-Geng (Din) Chen Shein-Chung Chow and Mark Chang Computational Methods in Biomedical Adaptive Design Theory and Research Implementation Using SAS and R Ravindra Khattree and Dayanand N. Naik Mark Chang Computational Pharmacokinetics Advanced Bayesian Methods for Medical Anders Källén Test Accuracy Confidence Intervals for Proportions and Lyle D. Broemeling Related Measures of Effect Size Advances in Clinical Trial Biostatistics Robert G. Newcombe Nancy L. Geller Controversial Statistical Issues in Basic Statistics and Pharmaceutical Clinical Trials Statistical Applications, Second Edition Shein-Chung Chow James E. De Muth Data and Safety Monitoring Committees Bayesian Adaptive Methods for in Clinical Trials Clinical Trials Jay Herson Scott M. Berry, Bradley P. Carlin, Design and Analysis of Animal Studies in J. Jack Lee, and Peter Muller Pharmaceutical Development Bayesian Analysis Made Simple: An Excel Shein-Chung Chow and Jen-pei Liu GUI for WinBUGS Design and Analysis of Bioavailability and Phil Woodward Bioequivalence Studies, Third Edition Bayesian Methods for Measures of Shein-Chung Chow and Jen-pei Liu Agreement Design and Analysis of Bridging Studies Lyle D. Broemeling Jen-pei Liu, Shein-Chung Chow, Bayesian Missing Data Problems: EM, and Chin-Fu Hsiao Data Augmentation and Noniterative Design and Analysis of Clinical Trials with Computation Time-to-Event Endpoints Ming T. Tan, Guo-Liang Tian, Karl E. Peace and Kai Wang Ng Design and Analysis of Non-Inferiority Bayesian Modeling in Bioinformatics Trials Dipak K. Dey, Samiran Ghosh, Mark D. Rothmann, Brian L. Wiens, and Bani K. Mallick and Ivan S. F. Chan Biostatistics: A Computing Approach Difference Equations with Public Health Stewart J. Anderson Applications Causal Analysis in Biomedicine and Lemuel A. Moyé and Asha Seth Kapadia Epidemiology: Based on Minimal DNA Methylation Microarrays: Sufficient Causation Experimental Design and Statistical Mikel Aickin Analysis Clinical Trial Data Analysis using R Sun-Chong Wang and Arturas Petronis Ding-Geng (Din) Chen and Karl E. Peace K14515_FM.indd 3 5/31/12 9:53 AM DNA Microarrays and Related Genomics Monte Carlo Simulation for the Techniques: Design, Analysis, and Pharmaceutical Industry: Concepts, Interpretation of Experiments Algorithms, and Case Studies David B. Allison, Grier P. Page, Mark Chang T. Mark Beasley, and Jode W. Edwards Multiple Testing Problems in Dose Finding by the Continual Pharmaceutical Statistics Reassessment Method Alex Dmitrienko, Ajit C. Tamhane, Ying Kuen Cheung and Frank Bretz Elementary Bayesian Biostatistics Randomized Clinical Trials of Lemuel A. Moyé Nonpharmacological Treatments Isabelle Boutron, Philippe Ravaud, and Frailty Models in Survival Analysis David Moher Andreas Wienke Sample Size Calculations in Clinical Generalized Linear Models: A Bayesian Research, Second Edition Perspective Shein-Chung Chow, Jun Shao Dipak K. Dey, Sujit K. Ghosh, and Hansheng Wang and Bani K. Mallick Statistical Design and Analysis of Handbook of Regression and Modeling: Stability Studies Applications for the Clinical and Shein-Chung Chow Pharmaceutical Industries Daryl S. Paulson Statistical Evaluation of Diagnostic Performance: Topics in ROC Analysis Interval-Censored Time-to-Event Data: Kelly H. Zou, Aiyi Liu, Andriy Bandos, Methods and Applications Lucila Ohno-Machado, and Howard Rockette Ding-Geng (Din) Chen, Jianguo Sun, and Karl E. Peace Statistical Methods for Clinical Trials Mark X. Norleans Joint Models for Longitudinal and Time- to-Event Data: With Applications in R Statistics in Drug Research: Dimitris Rizopoulos Methodologies and Recent Developments Measures of Interobserver Agreement Shein-Chung Chow and Jun Shao and Reliability, Second Edition Mohamed M. Shoukri Statistics in the Pharmaceutical Industry, Third Edition Medical Biostatistics, Third Edition Ralph Buncher and Jia-Yeong Tsay A. Indrayan Translational Medicine: Strategies and Meta-Analysis in Medicine and Health Statistical Methods Policy Dennis Cosmatos and Shein-Chung Chow Dalene Stangl and Donald A. Berry K14515_FM.indd 4 5/31/12 9:53 AM Interval-Censored Time-to-Event Data Methods and Applications Edited by Ding-Geng (Din) Chen Jianguo Sun Karl E. Peace K14515_FM.indd 5 5/31/12 9:53 AM CRC Press Taylor & Francis Group 6000 Broken Sound Parkway NW, Suite 300 Boca Raton, FL 33487-2742 © 2013 by Taylor & Francis Group, LLC CRC Press is an imprint of Taylor & Francis Group, an Informa business No claim to original U.S. Government works Version Date: 20120525 International Standard Book Number-13: 978-1-4665-0428-8 (eBook - PDF) This book contains information obtained from authentic and highly regarded sources. Reasonable efforts have been made to publish reliable data and information, but the author and publisher cannot assume responsibility for the validity of all materials or the consequences of their use. The authors and publishers have attempted to trace the copyright holders of all material reproduced in this publication and apologize to copyright holders if permission to publish in this form has not been obtained. If any copyright material has not been acknowledged please write and let us know so we may rectify in any future reprint. Except as permitted under U.S. Copyright Law, no part of this book may be reprinted, reproduced, transmit- ted, or utilized in any form by any electronic, mechanical, or other means, now known or hereafter invented, including photocopying, microfilming, and recording, or in any information storage or retrieval system, without written permission from the publishers. For permission to photocopy or use material electronically from this work, please access www.copyright. com (http://www.copyright.com/) or contact the Copyright Clearance Center, Inc. (CCC), 222 Rosewood Drive, Danvers, MA 01923, 978-750-8400. CCC is a not-for-profit organization that provides licenses and registration for a variety of users. For organizations that have been granted a photocopy license by the CCC, a separate system of payment has been arranged. Trademark Notice: Product or corporate names may be trademarks or registered trademarks, and are used only for identification and explanation without intent to infringe. Visit the Taylor & Francis Web site at http://www.taylorandfrancis.com and the CRC Press Web site at http://www.crcpress.com To my parents and parents-in-law, who value higher education and hard work, and to my wife Ke, my son John D. Chen, and my daughter Jenny K. Chen, for their love and support. Ding-Geng (Din) Chen TomywifeXianghuanLi,andmysonsRyanandNicholasSun,fortheirlove, patience, and support. Jianguo Sun TothememoryofmylatemotherElsieMaeCloudPeace,mylatewifeJiann- Ping Hsu, and to my son Christopher K. Peace, daughter-in-law Ashley Hop- kins Peace, and grandchildren Camden and Henry. Karl E. Peace TThhiiss ppaaggee iinntteennttiioonnaallllyy lleefftt bbllaannkk Contents List of Figures xvii List of Tables xix Preface xxi List of Contributors xxv I Introduction and Overview 1 1 Overview of Recent Developments for Interval-Censored Data 3 Jianguo Sun and Junlong Li 1.1 Introduction . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 1.2 More Introduction . . . . . . . . . . . . . . . . . . . . . . . . 6 1.3 Nonparametric Estimation . . . . . . . . . . . . . . . . . . . 9 1.4 Nonparametric Comparison of Survival Functions . . . . . . 12 1.5 Regression Analysis . . . . . . . . . . . . . . . . . . . . . . . 14 1.6 Analysis of Multivariate or Clustered Interval-Censored Data 17 1.7 Analysis of Competing Risks Interval-Censored Data . . . . 19 1.8 Other Topics and Concluding Remarks . . . . . . . . . . . . 20 Bibliography . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22 2 A Review of Various Models for Interval-Censored Data 29 Qiqing Yu and Yuting Hsu 2.1 Introduction . . . . . . . . . . . . . . . . . . . . . . . . . . . 29 ix

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