Prostate Cancer Screening

Transcription

1 Prostate Cancer Screening

2 Current Clinical Urology Eric A. Klein, MD, SERIES EDITOR Prostate Cancer Screening, Second Edition, edited by Donna P. Ankerst, Catherine M. Tangen, Ian M. Thompson, 2009 Urologic Robotic Surgery, edited by Jeffrey A. Stock, Michael P. Esposito, and Vincent Lanteri, 2008 Genitourinary Pain and Inflammation: Diagnosis and Management, edited by Jeanette M. Potts, 2008 Prostate Biopsy: Indications, Techniques, and Complications, edited by J. Stephen Jones, 2008 Female Urology: A Practical Clinical Guide, edited by Howard Goldman and Sandip P. Vasavada, 2007 Urinary Stone Disease: The Practical Guide to Medical and Surgical Management, edited Marshall L. Stoller and Maxwell V. Meng, 2007 PeyronieŠs Disease: A Guide to Clinical Management, edited by Laurence A. Levine, 2006 Male Sexual Function: A Guide to Clinical Management, Second Edition, edited by John J. Mulcahy, 2006 Advanced Endourology: The Complete Clinical Guide, edited by Stephen Y. Nakada and Margaret S. Pearle, 2005 Oral Pharmacotherapy of Male Sexual Dysfunction: A Guide to Clinical Management, edited by Gregory A. Broderick, 2005 Urological Emergencies: A Practical Guide, edited by Hunter Wessells and Jack W. McAninch, 2005 Management of Prostate Cancer, Second Edition, edited by Eric A. Klein, 2004 Essential Urology: A Guide to Clinical Practice, edited by Jeannette M. Potts, 2004 Management of Benign Prostatic Hypertrophy, edited by Kevin T. McVary, 2004 Pediatric Urology, edited by John P. Gearhart, 2003 Laparoscopic Urologic Oncology, edited by Jeffrey A. Cadeddu, 2004 Essential Urologic Laparoscopy: The Complete Clinical Guide, edited by Stephen Y. Nakada, 2003 Urologic Prostheses: The Complete Practical Guide to Devices, Their Implantation, and Patient Follow-Up, edited by Culley C. Carson, III, 2002 Male Sexual Function: A Guide to Clinical Management, edited by John J. Mulcahy, 2001 Prostate Cancer Screening, edited by Ian M. Thompson, Martin I. Resnick, and Eric A. Klein, 2001 Bladder Cancer: Current Diagnosis and Treatment, edited by Michael J. Droller, 2001

3 Prostate Cancer Screening Second Edition Edited by Donna P. Ankerst, Ph.D. Technical University, Garching, Germany and University of Texas Health Science Center at San Antonio, San Antonio, TX, USA Catherine M. Tangen, DrPh Fred Hutchinson Cancer Research Center, Seattle, WA, USA Ian M. Thompson Jr, MD University of Texas Health Science Center at San Antonio, San Antonio, TX, USA

4 Editors Donna P. Ankerst Professor, Department of Mathematics Technical University, Garching, Germany Associate Professor, Departments of Urology, and Epidemiology/Biostatistics University of Texas Health Science Center at San Antonio San Antonio, TX, USA Catherine M. Tangen Member, Southwest Oncology Group Statistical Center Fred Hutchinson Cancer Research Center Seattle, WA, USA Ian M. Thompson Professor and Chair, Department of Urology University of Texas Health Science Center at San Antonio San Antonio, TX, USA Series Editor Eric A. Klein Professor of Surgery Cleveland Clinic Lerner College of Medicine Head, Section of Urologic Oncology Glickman Urological and Kidney Institute Cleveland, OH, USA ISBN e-isbn DOI / Library of Congress Control Number: c Humana Press, a part of Springer Science+Business Media, LLC 2009 All rights reserved. This work may not be translated or copied in whole or in part without the written permission of the publisher (Humana Press, c/o Springer Science+Business Media, LLC, 233 Spring Street, New York, NY 10013, USA), except for brief excerpts in connection with reviews or scholarly analysis. Use in connection with any form of information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed is forbidden. The use in this publication of trade names, trademarks, service marks, and similar terms, even if they are not identified as such, is not to be taken as an expression of opinion as to whether or not they are subject to proprietary rights. While the advice and information in this book are believed to be true and accurate at the date of going to press, neither the authors nor the editors nor the publisher can accept any legal responsi-bility for any errors or omissions that may be made. The publisher makes no warranty, express or implied, with respect to the material contained herein. Printed on acid-free paper springer.com

5 Preface We are entering a remarkable time in medicine. In the USA and other countries, preventive medicine and public health efforts have successfully increased life expectancy substantially; concurrently, the demographics of the post-world War II population tells us that the over-50 population will dramatically increase over the next two decades. As prostate cancer is distinctly age-related, we can anticipate a tsunami-like increase in the numbers of patients with this disease. How will we respond to this challenge over the next two decades? Between the early 1900s and the mid-1980s, about the only strategy against this disease was a digital rectal examination (DRE) with the goal of early detection and treatment; if the disease was detected late, hormonal therapy was the mainstay of therapy. Unfortunately, systematic analyses of DRE as a screening tool found that the majority of cases detected were incurable by the time the tumor was palpable. In the case of hormonal therapy for advanced disease, 60 years after its discovery, average survival remains only about 2 3 years. With the discovery of PSA, a remarkable marker of prostate cancer risk, and with a national enthusiastic embrace of this test, the entire approach to this disease changed. The number of diagnosed prostate cancers more than doubled and the majority of tumors detected were organ-confined and probably cured. A man s lifetime risk of prostate cancer diagnosis more than doubled from 8% in the early 1980s to almost 18% today. When PSA first began to be used for screening in the early to mid-1980s, screening was a simple matter for the patient and doctor: If his PSA was above 4.0 ng/ml, it was abnormal and a biopsy was recommended. If it was below 4.0 ng/ml, it was normal and he was reassured that all was well. We now know that this concept is not correct and that evaluating a man s risk of prostate cancer is considerably more complex. PSA is not abnormal or normal but reflects a range of risk with each increase in level associated with an increased level of risk. We know that a PSA value in one person with few other risk factors of prostate cancer means something completely different than the same PSA value in another man who has other risk factors that increase his risk of cancer. Clinicians can no longer say your PSA is normal but instead must understand how to integrate other measures of risk as well as understand when to request other screening tests. They must also understand how to explain these risks to their patients who have been accustomed to 20 years of normal/abnormal readings on their PSA slips. Concurrent with our understanding of this has been the explosion of new biomarkers and biomeasures of prostate cancer. We discriminate between the two terms, understanding that a biomarker may be the measured value of a substance in a bodily fluid or other biologic sample while a biomeasure could include body mass index, number of affected male relatives with prostate cancer, or other observed and quantifiable values. Biomarker and biomeasure discovery has rapidly emerged as one of the primary focus areas in prostate cancer screening, with the hope of significantly improving benefits of v

6 vi Preface screening through more accurate diagnosis. The translational endeavors, however, bring significant technologic, statistical methodologic, and clinical challenges. This book incorporates a series of thoughtful and cutting-edge works from the world s experts in prostate cancer screening, ranging from the current status quo of prostate cancer screening across the globe, consensus on optimal utilization of the traditional PSA and DRE tests, cutting-edge research in new biomarkers, biomeasures and extended risk algorithms for prostate cancer, and last but not least, coverage of large ongoing international prevention and screening trials that aim to reduce prostate cancer mortality. The information will be helpful not only to the clinician who is faced with explaining risk to the patient but also to the researcher who is developing new biomarkers, to the public health and policy decision-maker who is determining how screening should be implemented, as well as to current and future members of the biomarker industry who seek methods to better develop and support markers and measures of prostate cancer. We are indebted to our colleagues around the world who have contributed their time to this wonderful text. These are the scientists who have made and will continue to make discoveries that will impact the lives of hundreds of millions of men worldwide as they face the most common cancer in men cancer of the prostate.

7 Contents Preface... Contributors... ColorPlates... v xi xv PART I: TRENDS IN PROSTATE CANCER SCREENING 1 Overview of US Prostate Cancer Trends in the Era of PSA Screening Ruth Etzioni, Roman Gulati, and Angela Mariotto 2 Trends in Prostate Cancer Screening: Overview of the UK Richard J. Bryant and Freddie C. Hamdy 3 Trends in Prostate Cancer Screening: Canada Robert K. Nam and Laurence H. Klotz 4 Trends in Prostate Cancer Screening Overview of Europe Sanjai K. Addla and Bob Djavan PART II: PROSTATE-SPECIFIC ANTIGEN 5 Evolution of Prostate-Specific Antigen for Screening Javier Hernandez and Edith Canby-Hagino 6 The Performance Characteristics of Prostate-Specific Antigen for Prostate Cancer Screening Ian M. Thompson, Jr. and Donna P. Ankerst 7 The Performance of PSA for Predicting Prostate Cancer After a Prior Negative ProstateBiopsy Catherine M. Tangen 8 Subfractions and Derivatives of Total Prostate-Specific Antigen in the Early Detection of Prostate Cancer Alexander Haese and Alan W. Partin 9 PSA Velocity at Presentation as a Predictor of Prostate Cancer Aggressiveness Jason A. Efstathiou and Anthony V. D Amico PART III: CANCER RISK ASSESSMENT FOR PROSTATE 10 Nomograms for Prostate Cancer Shahrokh F. Shariat and Michael W. Kattan vii

8 viii Contents 11 Decision Aid Criteria and Artificial Neural Networks for Optimizing Prostate Cancer Risk Prediction Felix K.-H. Chun and Pierre I. Karakiewicz 12 Development of the Prostate Cancer Prevention Trial Prostate Cancer RiskCalculator Donna P. Ankerst and Ian M. Thompson 13 Integration of Risk Assessment in Prostate Cancer Screening Kadee E Thompson, Amanda Reed, and Dipen J Parekh 14 Family History of Prostate Cancer During Rapidly Increasing Incidence Kari Hemminki and Justo Lorenzo Bermejo PART IV: RECENT BIOMARKERS FOR PROSTATE CANCER 15 PSA Isoforms: [ 2]proPSA Significant Adjunct to Free PSA Jeffrey E. Tam 16 PCA Jack Groskopf, Jack Schalken, and Harry Rittenhouse 17 Transcriptional Profiling of Prostate Cancer: Biomarker Identification andclinicalapplications Nigel Clegg and Peter S. Nelson 18 Biomarkers for Prostate Cancer Detection: Family-Based Linkage Analysis andcase ControlAssociationStudies Joke Beuten and Teresa L Johnson-Pais 19 GSTP1 Hypermethylation for Prostate Cancer Detection Rui Henrique and Carmen Jerónimo 20 EPCA and EPCA-2 as Potential Biomarkers for Prostate Cancer Detection Eddy S. Leman and Robert H. Getzenberg PART V: DESIGN AND METHODOLOGIC CONSIDERATIONS IN BIOMARKER STUDIES 21 Toward a Robust System for Biomarker Triage and Validation EDRN Experience Ziding Feng, Jacob Kagan and Sudhir Srivastava 22 Statistical Evaluation of Markers and Risk Tools for Prostate Cancer ClassificationandPrediction Yingye Zheng and Donna P. Ankerst 23 Pitfalls in Prostate Cancer Biomarker Evaluation Studies Donna P. Ankerst PART VI: BUILDING UPON RECENTLY COMPLETED AND ONGOING PROSPECTIVE TRIALS 24 Prostate-Specific Antigen and Its Role on the Prostate Cancer Prevention Trial Catherine M. Tangen and Phyllis J. Goodman

9 Contents ix 25 The Selenium and Vitamin E Cancer Prevention Trial Eric A. Klein 26 PLCO: A Randomized Controlled Screening Trial for Prostate, Lung, Colorectal, and Ovarian Cancer Amanda Black, Robert L. Grubb III, and E. David Crawford 27 The European Randomized Study of Screening for Prostate Cancer (ERSPC) Tineke Wolters and Fritz H. Schröder Index...387

10 Contributors SANJAI K. ADDLA, FRCS Department of Urology, University of Brussels, Erasme Hospital, University Clinics of Brussels, Brussels, Belgium DONNA P. ANKERST, PHD Department of Mathematics, Technical University, Garching, Germany, and Departments of Urology, and Epidemiology/Biostatistics, University of Texas Health Science Center at San Antonio, San Antonio, TX, USA JUSTO LORENZO BERMEJO, MD Division of Molecular Genetic Epidemiology, German Cancer Research Center, Heidelberg, Germany JOKE BEUTEN, PHD Department of Cellular and Structural Biology, The University of Texas Health Science Center, San Antonio, TX, USA AMANDA BLACK, PHD Early Detection Research Group, Division of Cancer Prevention and, the Cancer Prevention Fellowship Program, Office of Preventive Oncology, National Cancer Institute, Bethesda, MD, USA RICHARD J. BRYANT Lecturer in Urology, Nuffield Department of Surgery, Fellow of Balliol, University of Oxford, John Radcliffe Hospital, Oxford OX3 9DU, United Kingdom EDITH CANBY-HAGINO, MD, MS Wilford Hall Medical Center, Lackland AFB, San Antonio, TX, USA FELIX K.-H. CHUN, MD Department of Urology, University of Hamburg, Germany NIGEL CLEGG, PHD Divisions of Human Biology and Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, WA, USA E. DAVID CRAWFORD, MD Division of Urology, University of Colorado School of Medicine, Denver, CO, USA ANTHONY V. D AMICO, MD, PHD Department of Radiation Oncology, Brigham & Women s Hospital and Dana Farber Cancer Institute, Harvard Medical School, Boston, MA, USA BOB DJAVAN MD, PHD Department of Urology, University of Brussels, Erasme Hospital, University Clinics of Brussels, Brussels, Belgium JASON A. EFSTATHIOU, MD, PHD Department of Radiation Oncology, Brigham & Women s Hospital and Dana Farber Cancer Institute, Harvard Medical School, Boston, MA, USA RUTH ETZIONI, MD Fred Hutchinson Cancer Research Center, Seattle, WA, USA ZIDING FENG, PHD Fred Hutchinson Cancer Research Center, Seattle, WA, USA ROBERT H. GETZENBERG, PHD The Brady Urological Institute, Johns Hopkins Hospital, Baltimore, MD, USA PHYLLIS J. GOODMAN, MS Southwest Oncology Group Statistical Center, Fred Hutchinson Cancer Research Center, Seattle, WA, USA JACK GROSKOPF, PHD Gen-Probe Incorporated, San Diego, CA, USA ROBERT L. GRUBB III, MD Division of Urologic Surgery, Washington University, St. Louis, MO, USA ROMAN GULATI, MS Fred Hutchinson Cancer Research Center, Seattle, Washington, USA xi

11 xii Contributors ALEXANDER HAESE, MD, PHD Department of Urology, University Clinic Hamburg-Eppendorf, Hamburg, Germany FC HAMDY, MD Nuffield Department of Surgery, Fellow of Balliol, University of Oxford, John Radcliffe Hospital, Oxford OX3 9DU, United Kingdom KARI HEMMINKI, MD Division of Molecular Genetic Epidemiology, German Cancer Research Center, Heidelberg, Germany and Center for Family and Community Medicine, Karolinska Institute, Huddinge, Sweden RUI HENRIQUE, MD, PHD Department of Pathology, Portuguese Oncology Institute Porto, Portugal, and Department of Pathology and Molecular Immunology, Institute of Biomedical Sciences Abel Salazar (ICBAS), University of Porto, Portugal JAVIER HERNANDEZ, MD, MS Brooke Army Medical Center, Fort Sam Houston, TX, USA CARMEN JERóNIMO, PHD Department of Genetics, Portuguese Oncology Institute Porto, Portugal, Department of Pathology and Molecular Immunology, Institute of Biomedical Sciences Abel Salazar (ICBAS), University of Porto, Portugal, and Fernando Pessoa University School of Health Sciences, Porto, Portugal TERESA LJOHNSON-PAIS, PHD Department of Pediatrics, The University of Texas Health Science Center, San Antonio, TX, USA JACOB KAGAN, PHD Cancer Biomarkers Research Group, National Cancer Institute, USA PIERRE I. KARAKIEWICZ, MD Cancer Prognostics and Health Outcomes Unit, University of Montreal, Montreal, Quebec, Canada MICHAEL W. KATTAN, PHD Department of Quantitative Health Sciences, The Cleveland Clinic, Cleveland, OH, USA ERIC A. KLEIN, MD Cleveland Clinic Glickman Urological and Kidney Institute, Cleveland, OH, USA LAURENCE H. KLOTZ, MD Division of Urology, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada EDDY S. LEMAN, PHD The Brady Urological Institute, Johns Hopkins Hospital, Baltimore, MD, USA ANGELA MARIOTTO, PHD Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, MD, USA ROBERT K. NAM, MD Division of Urology, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada PETER S. NELSON, MD Divisions of Human Biology and Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, WA, USA DIPEN JPAREKH, MD Department of Urology, University of Texas at San Antonio, San Antonio, TX, USA ALAN W. PARTIN, MD PHD Department of Urology, Oncology, Johns Hopkins Medical Institutions, Baltimore, MD, USA AMANDA REED, MD Department of Urology, University of Texas at San Antonio, San Antonio, TX, USA HARRY RITTENHOUSE, PHD Gen-Probe Incorporated, San Diego, CA, USA JACK SCHALKEN, PHD Department of Urology, Radboud University Nijmegen Medical Centre, The Netherlands FRITZ H. SCHRÖDER, MD Department of Urology, Erasmus University and Academic Hospital, Rotterdam, The Netherlands

12 Contributors xiii SHAHROKH F. SHARIAT, MD Division of Urology, Memorial Sloan-Kettering Cancer Center, New York, NY, USA SUDHIR SRIVASTAVA, PHD, MPH, Cancer Biomarkers Research Group, National Cancer Institute, USA JEFFREY E. TAM, PHD Beckman Coulter Inc., Chaska, MN, USA CATHERINE M. TANGEN, DRPH Southwest Oncology Group Statistical Center, Fred Hutchinson Cancer Research Center, Seattle, WA, USA IAN M. THOMPSON, JR., MD Department of Urology, University of Texas Health Science Center at San Antonio, San Antonio, TX, USA KADEE THOMPSON, MD Department of Urology, University of Texas at San Antonio, San Antonio, TX, USA TINEKE WOLTERS, MD Department of Urology, Erasmus University and Academic Hospital, Rotterdam, The Netherlands YINGYE ZHENG, PHD Department of Biostatistics and Biomathematics, Fred Hutchinson Cancer Research Center, Seattle, WA, USA

13 Color Plates Color Plate 1 Depiction of isoforms of free PSA (Chapter 8, Fig. 1; see discussion on p. 87). Color Plate 2 Risk of prostate cancer by PSA for four groups of participants according to their DRE and family history status; + denotes abnormal for DRE and positive for family history, denotes normal for DRE and negative for family history. Assumes no prior negative biopsy (Chapter 12, Fig. 1; see discussion on p. 198). Color Plate 3 Risk of high-grade prostate cancer by PSA for four groups of caucasian participants according to DRE age and; + abnormal DRE, normal DRE. Assumes no prior negative biopsy. (Chapter 12, Fig. 2; see discussion on p. 199). Color Plate 4 Prostate-specific antigen levels achieving specified risks of prostate cancer adjusted for risk factors DRE and family history of prostate cancer (Chapter 12, Fig. 5; see discussion on p. 201). Color Plate 5 Release of prostate cells into the urethra following DRE (a) vs. release of PSA protein into the bloodstream (b) (Chapter 16, Fig. 1; see discussion on p. 235). Color Plate 6 ROC curves for serum PSA, PCA3 Score, and the logistic regression (LR) model incorporating serum PSA, PCA3 Score, diagnostic DRE result, and prostate gland volume (Chapter 16, Fig. 3; see discussion on p. 239). Color Plate 7 Mean PCA3 Scores for men with low-volume / low-grade PCa (<0.5 cc, Gleason 6) vs. significant PCa (>0.5 cc, Gleason 7). Error bars represent the standard error of the mean (Chapter 16, Fig. 4; see discussion on p. 239). Color Plate 8 Heat map ordered by blood draw date. PCA Late group and Control group had blood draw PCA Early group had blood draw date prior to 1995 (Chapter 21, Fig. 1; see discussion on p. 298). Color Plate 9 ROC curves and 95% confidence intervals for Marker 1 and PSA on discovery data (highly promising). The low AUC value for PSA is due to the fact that biopsy is often triggered by elevated PSA (Chapter 21, Fig. 2; see discussion on p. 301). Color Plate 10 ROC curves and 95% confidence intervals for Marker 1 and PSA on EDRN reference set data (not promising) (Chapter 21, Fig. 3; see discussion on p. 301). Color Plate 11 ROC curve of Marker 2 on EDRN reference set (not promising), performance drastically decreased on EDRN reference set. On discovery data both sensitivity and specificity were above 90% (Chapter 21, Fig. 4; see discussion on p. 301). xv

14 xvi Color Plates Color Plate 12 ROC curves and 95% confidence intervals for %[ 2]proPSA and PSA: Confirmed moderate performance for %[ 2]proPSA consistent to that observed in initial discovery studies (Chapter 21, Fig. 5; see discussion on p. 302). Color Plate 13 US map showing the PLCO screening center locations (Chapter 26, Fig. 2; see discussion on p. 363). PLCO screening centers: Alabama (University of Alabama, Birmingham); Michigan (Henry Ford Health System, Detroit); Colorado (University of Colorado, Denver); Hawaii (Pacific Health Research Institute, Honolulu); Wisconsin (Marshfield Clinic Research Foundation, Marshfield); Minnesota (University of Minnesota, Minneapolis); Pennsylvania (University of Pittsburgh, Pittsburgh); Utah (University of Utah, Salt Lake City); Idaho (University of Utah/Boise, Boise) (Utah Satellite Center); Missouri (Washington University, St. Louis); Washington DC (Georgetown University, Washington). Color Plate 14 Randomization procedures in ERSPC (Chapter 27, Fig. 1; see discussion on p. 376). Color Plate 15 Effect of different assumptions on the power of the ERSPC trial by follow-up year (Chapter 27, Fig. 2; see discussion on p. 383). (Source: H. de Koning (2002). International Journal of Cancer 98: )