Foundations of Clinical Research. Applications to Evidence-Based Practice

692 Pages·2020·47.914 MB·English

by  Leslie G. Portney

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Leslie G. Portney, DPT, PhD, FAPTA
Dean Emerita
MGH Institute of Health Professions
School of Health and Rehabilitation Sciences
Boston, Massachusetts
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Library of Congress Cataloging-in-Publication Data
Names: Portney, Leslie Gross, author.
Title: Foundations of clinical research : applications to evidence-based
practice / Leslie G. Portney.
Description: Fourth edition. | Philadelphia : F.A. Davis, [2020] | Includes
bibliographical references and index. | Summary: “The text of this
fourth edition has maintained its dual perspectives. It is designed for
those who do research to generate new knowledge and examine theories and
for those who want to be critical consumers of evidence to inform
clinical decision-making. All of us need to be able to critically
evaluate research reports to inform practice and further study. The
scope of the text allows it to fulfill different needs at different
times. This edition will continue to serve this wide audience. It
incorporates several important contemporary themes, including the
application of evidence-based practice (EBP) for clinical
decision-making, the imperative of interprofessional collaboration, the
implications of translational research, and the focus of healthcare on
quality of life, meaningful outcomes, and patient values. These
directions and priorities continue to define research philosophies that
guide the conduct of clinical inquiry, the types of questions that are
deemed important, and how they influence advances in measurement and
analysis techniques”— Provided by publisher.
Identifiers: LCCN 2019044025 (print) | LCCN 2019044026 (ebook) | ISBN
9780803661134 (hardcover) | ISBN 9780803661165 (ebook)
Subjects: MESH: Biomedical Research—methods | Evidence-Based
Practice—methods | Clinical Decision-Making—methods | Statistics as
Topic—methods | Research Design
Classification: LCC R853.C55 (print) | LCC R853.C55 (ebook) | NLM W 20.5
| DDC 610.72/4—dc23
LC record available at https://lccn.loc.gov/2019044025
LC ebook record available at https://lccn.loc.gov/2019044026
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To Hazel
. . . and joyful running hugs
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Preface
It is now more than 25 years since this text was first The popular features of the book have not changed,
published in 1993. It has become a classic reference, such as its accessible tone, use of extensive clinical exam-
cited extensively in literature from researchers around ples to illustrate applications of design and statistics, and
the globe. It is gratifying to see its sustained and wide- careful logic in explanation of complex concepts. It re-
spread use by clinicians, faculty, students, and re- mains a comprehensive text, meant to fulfill different
searchers in a variety of fields including rehabilitation, needs at different times, serving as both an introduction
medicine, public health, nursing, public policy, epi- and a reference as readers grow in their knowledge and
demiology, exercise physiology, and other disciplines experience. However, this fourth edition does represent
concerned with healthcare. This edition will continue a significant update in format, focus, and organization.
to serve this wide audience.
As I have approached each revision, I have been struck
by how much the framework for research continues to ■ New and Updated
evolve over time, and this edition is no different. It in-
corporates several important contemporary perspectives.
Perhaps most notable is the new design, intended to
First, regardless of the role we play in healthcare, be
make the text more reader-friendly and engaging with
it clinician, administrator, policy maker, or researcher,
full color. Tables and figures have a new look to facilitate
we all need to be critical consumers of evidence to in-
interpretation. Starting with the book’s title, the empha-
form decisions. This requires at least a basic understand-
sis on EBP is clear, including the Focus on Evidence and
ing of principles of measurement, design, and analysis to
Case in Point features in each chapter that illustrate the
enable reasonable and critical evaluation.
direct application of concepts in the literature. Addi-
Second, the concept of evidence-based practice
tional information is included in boxes to clarify content,
(EBP), although ubiquitous in healthcare discussion,
emphasize key points, offer interesting background and
must be understood in terms of its full purpose for clin-
historical context—and a few fun facts along the way.
ical decision-making, relying not only on literature but
Commentaries build on important concepts. Online re-
also on clinical expertise, patient values, and environ-
sources have also been expanded, with chapter supple-
mental conditions. These considerations have influenced
ments, access to data files, and materials for instructors
translational research and the need to address meaning-
and students.
ful outcomes.
Five new chapters have been included in this edition,
Third, today’s healthcare enterprise mandates atten-
focusing on translational research (Chapter 2), evidence-
tion to interprofessional collaboration. I have included
based practice (Chapter 5), health measurement scales
examples throughout the book to illustrate various con-
(Chapter 12), clinical trials (Chapter 14), and qualitative
cepts. These are drawn from many different fields, with
research (Chapter 21). All of these address content that
the intent that all examples have wide application. I be-
was covered in previous editions but with greater depth
lieve this is a distinct advantage of this book, to under-
and application.
score the importance of appreciating research within and
Many chapters also include new or updated content
beyond one’s own field.
to address current clinical research issues. For example,
And finally, the content will work for those who want
to plan or implement research. Detailed examples are in- • Examples provided throughout the book have been
cluded for those who want more depth, both in the text updated with more recent literature.
as well as in online supplements. • The stronger emphasis on EBP includes use of the
These directions and priorities continue to define re- PICO strategy to answer clinical questions as well as
search philosophies that guide the conduct of clinical in- to generate research questions. The EBP emphasis
quiry, the types of questions that are deemed important, reflects a comprehensive model that includes research
and how they influence advances in measurement and evidence, clinical expertise, patient values, and clinical
analysis techniques. circumstances.
v
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vi Preface
• The chapter on searching the literature has been ■ Organization
reorganized to provide stronger guidance in that
process.
Although the overall organization of the book remains
• The chapter on ethical principles has been
the same with content in five parts, some chapters have
updated to reflect recent changes in regulations
been rearranged to better reflect their role in the re-
and definitions.
search process. Each section focuses on a different phase
• Measurement chapters provide a stronger emphasis
of that process.
on relative and absolute reliability, a broader context
for construct validity, and extensive discussion on • Part 1- Foundations of Research and Evidence
measuring change. focuses on fundamental concepts that define
• The chapter on health measurement scales focuses the clinical research enterprise, such as types of
on construct validity and reliability for question- research, frameworks of health, translational
naires that are used to measure function, quality of research, application of theory, ethical principles,
life, and personal characteristics that impact health. how to frame clinical and research questions, litera-
It includes expanded coverage of Rasch analysis. ture search strategies, and the overarching structure
• Chapters on foundations of design cover contempo- of EBP for clinical decision-making. These chapters
rary issues related to clinical trials, such as transla- provide an overview of the research process that
tional research, efficacy and effectiveness, serves as a foundation for the rest of the text.
superiority and non-inferiority trials, pragmatic • Part 2- Concepts of Measurementincludes the
trials, N-of-1 trials, and implementation studies. basic premises of measurement for all types of data
• Content related to observational designs has collection, including validity and reliability. The
increased emphasis on use of these designs for chapter on surveys has been relocated to this section
diagnosis and prognosis, as well as for evaluating to emphasize their role as data collection tools
causal relationships. rather than a design. Fundamental concepts in the
• Qualitative research has a stronger presence design of health measurement scales are presented.
throughout the book, including a new chapter • Part 3- Designing Clinical Researchincludes con-
that also addresses mixed methods. Although this tent on descriptive, observational, and explanatory
coverage cannot substitute for a full reference on research, with extensive examples related to validity
this topic, this content should facilitate reading and and application of these designs. These include a
interpreting the literature, and will reinforce the rel- variety of clinical trial designs, single-subject designs,
evance of qualitative research in a variety of contexts. observational studies using cohort and case-control
• The International Classification of Functioning, designs, and descriptive and qualitative studies.
Disability and Health (ICF)is used as a framework • Part 4- Analyzing Datapresents the full scope of
for terminology as part of understanding patient- statistical content, starting with descriptive statistics
oriented research. and developing concepts through multivariate
• Statistics chapters have added coverage of validity procedures. Parametric and nonparametric statistics
measures and regression and multivariate meth- are described for comparative, predictive, and
ods, such as exploratory and confirmatory factor descriptive studies, validity and reliability, studies
analysis and structural equation modeling. Data of diagnostic tests, and epidemiology related to
files for all examples presented in tables can be risk assessments. Information is presented from an
accessed online. intuitive perspective without emphasis on mathemat-
• Information on power, effect size, and sample size ical derivation or statistical notation. These chapters
calculations are integrated with statistical proce- include a review of output using SPSS to illustrate
dures and illustrated in detail in chapter supple- how statistical results can be interpreted. Data files
ments. Some analyses are done using SPSS and can also be accessed online for all analyses.
others using G*Power. • Part 5- Putting It All Togetherfocuses on the aspects
• The chapters on critical appraisal and dissemination of research that incorporate all the principles of de-
include information on use of reporting guidelines sign and analysis that have been covered in preceding
for different types of studies. chapters. Chapter 35 describes guidelines for devel-
• The chapter on systematic reviews has been oping a research proposal. Chapter 36 addresses the
expanded to include more detail related to process of critical appraisal for several types of stud-
meta-analyses and now includes information ies. Chapter 37 provides an overview of methods
on scoping reviews. for conducting and evaluating systematic reviews,
6113_FM_i-xxiv 05/12/19 1:31 pm Page vii
Preface vii
meta-analyses, and scoping reviews. Finally, Chapter 38 No matter how many years I have engaged in re-
presents a format for preparing and disseminating search, I continue to learn about advances in design and
research through publications and presentations. analysis that set new directions and priorities for clini-
cal inquiry. This edition is intended to strengthen our
Appendix Aincludes reference tables for major statis-
collective commitment to new knowledge, discovery
tical tests and additional tables are provided in Appendix
and innovation, and implementation of evidence, as we
A Online. Appendix Bincludes an algorithm for choos-
strive to provide the most effective care. The process
ing a statistical procedure based on design and measure-
remains a challenge to all of us engaged in healthcare,
ment considerations. Appendix Ccovers content related
and this text will always be a work in progress. I look
to management of quantitative data, including data trans-
forward to continuing to hear from colleagues across
formation. The glossary, also available online, provides
disciplines regarding how to make this volume most
a handy reference to define terms, symbols, and common
useful.
abbreviations.
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Contributors
Jessica Bell, MS K. Douglas Gross, MPT, DPT, ScD,
Director of Library and Instructional Design FAAOMPT, CPed
MGH Institute of Health Professions Associate Professor, Department of Physical Therapy
Boston, Massachusetts MGH Institute of Health Professions
School of Health and Rehabilitation Sciences
Marianne Beninato, DPT, PhD Boston, Massachusetts
Professor Emerita
MGH Institute of Health Professions Catherine Lysack, PhD, OT(C)
Boston, Massachusetts Associate Professor, Occupational Therapy
and Gerontology
Peter S. Cahn, PhD Eugene Applebaum College of Pharmacy and Health
Associate Provost for Academic Affairs Sciences
MGH Institute of Health Professions Wayne State University
Boston, Massachusetts Detroit, Michigan
Heather Fritz, PhD, MSOT David A. Scalzitti, PT, PhD
Assistant Professor, Occupational Therapy Program Assistant Professor, Physical Therapy and Health
and Institute of Gerontology Care Sciences
Eugene Applebaum College of Pharmacy and Health School of Medicine and Health Sciences
Sciences The George Washington University
Wayne State University Washington, DC
Detroit, Michigan
ix
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Reviewers
Jason Browning, OTR/L Joanne Gallagher Worthley, EdD, OTR/L, CAPS
Assistant Professor Professor
Occupational Therapy Occupational Therapy
Jefferson College of Health Sciences Worcester State University
Roanoke, Virginia Worcester, Massachusetts
Stephen Chao, PT, DPT, CSCS Sean F. Griech, PT, DPT, OCS, COMT
Clinical Assistant Professor Assistant Professor
Physical Therapy Department Doctor of Physical Therapy Program
School of Health Technology and Management DeSales University
State University of New York at Stony Brook Center Valley, Pennsylvania
Southampton, New York
Emmanuel B. John, PT, DPT, PhD, MBA
Jennifer B. Christy, PT, PhD Associate Professor & Chair
Associate Professor Department of Physical Therapy
Physical Therapy Chapman University
The University of Alabama at Birmingham Irvine, California
Birmingham, Alabama
Steven G. Lesh, PhD, PT, SCS, ATC
Peter C. Douris, PT, EdD, DPT, OCS Chair and Professor of Physical Therapy
Professor Southwest Baptist University
Physical Therapy Bolivar, Missouri
New York Institute of Technology
Old Westbury, New York Jean MacLachlan, PhD, OTR/L
Associate Professor
Winnie Dunn, PhD, OTR, FAOTA Occupational Therapy
Distinguished Professor Salem State University
Occupational Therapy Salem, Massachusetts
University of Missouri
Columbia, Missouri Dennis McCarthy, PhD, OTR/L, FAOTA
Associate Professor
Ashraf Elazzazi, PT, PhD Occupational Therapy
Associate Professor Nova Southeastern University
Chair, Department of Physical Therapy Tampa, Florida
Utica College
Utica, New York Raymond F. McKenna, PT, PhD, CSCS
Clinical Associate Professor
Simon French, BAppSc, MPH, PhD Physical Therapy
Associate Professor Stony Brook University
School of Rehabilitation Therapy Stony Brook, New York
Queen’s University
Kingston, Ontario, Canada
xi
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xii Reviewers
Saurabh Mehta, PT, PhD Eric Schussler, PhD, PT, ATC
Assistant Professor Assistant Professor
School of Physical Therapy School of Physical Therapy and Athletic Training
Marshall University Old Dominion University
Huntington, West Virginia Norfolk, Virginia
Tamara Mills, PhD, OTR/L, ATP Mary P. Shotwell, PhD, OT/L, FAOTA
Assistant Professor Professor
School of Occupational Therapy School of Occupational Therapy
Brenau University Brenau University
Norcross, California Gainesville, Georgia
Pablo Mleziva, PT, OCS, CSCS Matt S. Stock, PhD
Physical Therapist-Assistant Professor Assistant Professor
Physical Therapy Department of Health Professions
Loma Linda University University of Central Florida
Loma Linda, California Orlando, Florida
Shannon Petersen, PT Ying-Chih Wang, PhD
Associate Professor Associate Professor
Physical Therapy Occupational Science & Technology
Des Moines University University of Wisconsin – Milwaukee
Des Moines, Iowa Milwaukee, Wisconsin
Lenny Ramsey, PhD Jennifer B. Wasserman, DPT, MS, PhD
Assistant Professor Associate Professor
Physical Therapy Physical Therapy
Carroll University Franklin Pierce University
Waukesha, Wisconsin Manchester, New Hampshire
David A. Scalzitti, PT, PhD Mark Patrick Wilhelm, PT, DPT
Assistant Professor, Physical Therapy and Health Care Assistant Professor
Sciences Physical Therapy
School of Medicine and Health Sciences Walsh University
The George Washington University North Canton, Ohio
Washington, DC
6113_FM_i-xxiv 05/12/19 1:31 pm Page xiii
Acknowledgments
Taking on this edition as a solo effort has been an in- I’d also like to acknowledge my dear friend and writing
teresting challenge. I am so appreciative for the sup- partner Mary Watkins, who retired several years ago and
port of many friends and colleagues who have helped is happily ensconced in New Hampshire with her family.
me through it. I am indebted to those who contributed Our collaboration was a special part of our lives for more
to writing chapters—Jessica Bell, Marianne Beninato, than 20 years, and I sorely missed her this time around.
Peter Cahn, Heather Fritz, Doug Gross, Cathy Lysack, Over the past 50 years of my professional life, of which
and David Scalzitti. I am thankful to John Wong for 45 were spent in academia, I have had the good fortune
many hours of invaluable guidance on statistics. I am to work with thousands of students, faculty colleagues,
also grateful to those who took the time to review sec- and teachers who have inspired and challenged me. Too
tions of the book and provide valuable feedback— numerous to mention by name, I thank them all for their
Marjorie Nicholas, Winnie Dunn, Lisa Connor and friendship, mentorship, and dedication. I am also grateful
Saurabh Mehta among others. I also want to acknowl- to many people, some I know and many I have never met,
edge the long-time support of colleagues at the MGH from all over the world, who over the years have taken
Institute of Health Professions, especially Alex John- the time to stop me at conferences, or to call or send
son, Mary Ellen Ferolito, Denis Stratford and the en- emails, telling me how much they appreciated the book—
tire IT team, as well as the graduate assistants Jasmin and pointing out the mistakes and where information
Torres and Baothy Huynh, who will both be awesome needed updating. Those comments have been immensely
Doctors of Occupational Therapy. I reserve a special rewarding and incredibly helpful. I hope these colleagues
note of deep appreciation for David Scalzitti who has are pleased with the revisions they have inspired.
reviewed every page of this book and who freely of- And of course, there is no way a project like this gets
fered his advice along the way, in every instance pro- done without support and sacrifice from those closest
viding important comments to make things more to me. My parents, who were so proud of this work,
accurate, complete, and useful. Many of his “pearls” have both passed away since the last edition was pub-
are included in the text. Additionally, I’d like to thank lished, but they continue to inspire me every day.
David for creating the online Test Bank questions and My husband Skip has given up so much of our together
PowerPoint slides. Always a wonderful colleague, he time, particularly once we were both supposed to be
has become a good friend. retired. He gave me the space and time I needed, and
The F. A. Davis team was remarkably patient with now I have a lot of dishes to wash to make up my share!
me over many years, providing incredible support. Spe- My children, Devon and Jay and Lindsay and Dan, are
cial thanks to Jennifer Pine, Senior Sponsoring Editor, always there to prop me up with love and understand-
who held my hand and tolerated my missing every ing, and Lindsay was even able to help me through
deadline. Megan Suermann, Content Project Manager, some of the more complex statistics—very cool. And
lived through every word, figure, and table, and all their finally, little Hazel, the ultimate joy that only a grand-
changes. Thank you to everyone who helped with every parent can understand. Her smile and hugs have lifted
aspect of the project—to Cassie Carey, Senior Produc- me up through many tough days. She confirms for me
tion Editor at Graphic World Publishing Services; Kate every day that the future is bright. What they say is so
Margeson, Illustration Coordinator; Nichole Liccio, true—if I’d known grandchildren were so much fun,
Melissa Duffield, and especially to Margaret Biblis, I would have had them first! With no pun intended, the
Editor-in-Chief, who said “yes” so quickly. next chapter awaits!
xiii