Quality of Life Measurement in Neurodegenerative and Related Conditions

Quality of Life Measurement in Neurodegenerative and Related Conditions

Quality of Life Measurement in Neurodegenerative and Related Conditions Edited by Crispin Jenkinson University of Oxford Michele Peters University of Oxford Mark B. Bromberg University of Utah

cambridge university press Cambridge, New York, Melbourne, Madrid, Cape Town, Singapore, São Paulo, Delhi, Dubai, Tokyo, Mexico City Cambridge University Press The Edinburgh Building, Cambridge CB2 8RU, UK Published in the United States of America by Cambridge University Press, New York www.cambridge.org Information on this title: www.cambridge.org/9780521829014 C Cambridge University Press 2011 This publication is in copyright. Subject to statutory exception and to the provisions of relevant collective licensing agreements, no reproduction of any part may take place without the written permission of Cambridge University Press. First published 2011 Printed in the United Kingdom at the University Press, Cambridge A catalogue record for this publication is available from the British Library Library of Congress Cataloguing in Publication data Quality of life measurement in neurodegenerative and related conditions / edited by Crispin Jenkinson, Michele Peters, and Mark B. Bromberg. p. ; cm. Includes bibliographical references and index. ISBN 978-0-521-82901-4 (hardback) 1. Nervous system Degeneration Patients Rehabilitation. 2. Outcome assessment (Medical care) 3. Quality of life Measurement. I. Jenkinson, Crispin, 1962 II. Peters, Michele III. Bromberg, M. B. (Mark B.) [DNLM: 1. Nervous System Diseases. 2. Outcome Assessment (Health Care) 3. Quality of Life. WL 140 Q12 2011] RC365.Q35 2011 616.8 0471 dc22 ISBN 978-0-521-82901-4 Hardback Cambridge University Press has no responsibility for the persistence or accuracy of URLs for external or third-party internet websites referred to in this publication and does not guarantee that any content on such websites is, or will remain, accurate or appropriate. Every effort has been made in preparing this book to provide accurate and up-to-date information which is in accord with accepted standards and practice at the time of publication. Although case histories are drawn from actual cases, every effort has been made to disguise the identities of the individuals involved. Nevertheless, the authors, editors and publishers can make no warranties that the information contained herein is totally free from error, not least because clinical standards are constantly changing through research and regulation. The authors, editors and publishers therefore disclaim all liability for direct or consequential damages resulting from the use of material contained in this book. Readers are strongly advised to pay careful attention to information provided by the manufacturer of any drugs or equipment that they plan to use.

Contents List of contributors Preface page vii xi 1 Aspects of methodology relevant to patient-reported outcome measures (PROMs) 1 Crispin Jenkinson, Jill Dawson, and Christopher Morris 2 The development and validation of the Parkinson s Disease Questionnaire and related measures 10 Crispin Jenkinson and Ray Fitzpatrick 3 The Multiple Sclerosis Impact Scale (MSIS-29): Initial development, subsequent revision, lessons learned 24 Jeremy Hobart 4 Patient-reported outcome measurement in motor neuron disease/amyotrophic lateral sclerosis the ALSAQ-40 and ALSAQ-5 41 Crispin Jenkinson 5 Measuring quality of life in progressive supranuclear palsy: The PSP-QoL 52 Anette Schrag, Caroline Selai, Niall Quinn, Jeremy Hobart, Andrew J. Lees, Irene Litvan, Anthony Lang, James Bower, and David Burn v

vi Contents 6 Measuring quality of life in multiple system atrophy 60 Anette Schrag, Caroline Selai, Christopher Mathias, Philip Low, Jeremy Hobart, and Niall Quinn 7 Health-related quality of life in Huntington s disease 71 Noelle E. Carlozzi and Rebecca E. Ready 8 Measuring quality of life in dementia 82 Rebecca E. Ready 9 Condition-specific instruments to measure the quality of life (QoL) of children and adolescents with cerebral palsy (CP) 95 Elizabeth Waters, Amy Shelly, and Elise Davis 11 Translating patient-reported outcome measures (PROMs) for cross-cultural studies 139 Michele Peters 12 Rasch analysis 147 Jeremy Hobart and Stefan Cano 13 A method for imputing missing questionnaire data 165 Crispin Jenkinson, Robert Harris, and Ray Fitzpatrick 14 Individualized quality of life measurement in neurodegenerative disorders 174 Mark B. Bromberg Index 188 10 Outcome measures for informal carers of individuals with neurodegenerative conditions 114 Michele Peters

List of contributors James Bower, MD Associate Professor of Neurology, Mayo Clinic College of Medicine, Rochester, Minnesota, USA Mark B. Bromberg, MD, PhD Professor of Neurology, Department of Neurology, University of Utah, Salt Lake City, Utah, USA David Burn, MD Professor of Movement Disorder Neurology and Honorary Consultant Neurologist, Department of Neurology, The Royal Victoria Infirmary, Newcastle upon Tyne, UK Stefan Cano, PhD, CPsychol Associate Professor (Senior Lecturer) in Psychometrics, Peninsula College of Medicine and Dentistry, University of Plymouth, Tamar Science Park, Plymouth, UK Noelle E. Carlozzi, PhD Assistant Professor, Center for Rehabilitation Outcomes and Assessment Research, Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, Michigan, USA vii

viii List of contributors Elise Davis, PhD Research Fellow, McCaughey Centre, VicHealth Centre for the Promotion of Mental Health and Community Wellbeing, School of Population Health, University of Melbourne, Melbourne, Australia Jill Dawson, DPhil Senior Research Scientist, Health Services Research Unit, Department of Public Health, University of Oxford, Oxford, UK Ray Fitzpatrick, PhD, FMedSci Professor of Public Health and Primary Care and Head of the Department of Public Health, University of Oxford, Old Road Campus, Oxford, UK Robert Harris, MSc Research Officer, Health Services Research Unit, Department of Public Health, University of Oxford, Oxford, UK Jeremy Hobart, FRCP, PhD Associate Professor (Reader) in Clinical Neurosciences, Peninsula College of Medicine and Dentistry, University of Plymouth, Tamar Science Park, Plymouth, UK Crispin Jenkinson, DPhil Professor of Health Services Research and Director, Health Services Research Unit, Department of Public Health, University of Oxford, Oxford, UK Anthony Lang, FRCPC, MD Professor of Neurology and Director, Morton and Gloria Shulman Movement Disorder Centre, Toronto Western Hospital, Ontario, Canada Andrew J. Lees, MD, FRCP, FMedSci Professor of Neurology, Honorary Consultant Neurologist, and Director, Reta Lila Weston Institute of Neurological Studies, University College London, London, UK Irene Litvan, MD Raymond Lee Lebby Professor of Parkinson Disease Research, and Director, Division of Movement Disorders, University of Louisville, Kentucky, USA Philip Low, MD Professor of Neurology, Mayo Clinic College of Medicine, Rochester, Minnesota, USA Christopher Mathias, DPhil, DSc, FRCP Professor of Neurovascular Medicine, and Consultant Physician, Imperial College, London, and Institute of Neurology, University College London, London, UK Christopher Morris, DPhil Senior Research Fellow, Cerebra Research Unit and PenCLARHC, Peninsula College of Medicine and Dentistry, University of Exeter, Exeter, Devon, UK

List of contributors ix Michele Peters, PhD Senior Research Scientist, Health Services Research Unit, Department of Public Health, University of Oxford, Oxford, UK Niall Quinn, MD, FRCP Emeritus Professor of Clinical Neurology, and Honorary Consultant Neurologist, Institute of Neurology, University College London, London, UK Rebecca E. Ready, PhD Associate Professor, Department of Psychology, University of Massachusetts, Amherst, Massachusetts, USA Anette Schrag, MD Reader in Neurology, and Honorary Consultant Neurologist, Department of Clinical Neurosciences, Royal Free and University College Medical School, University College London, London, UK Caroline Selai, PhD, CPsychol Senior Lecturer in Clinical Neuroscience, Sobell Department for Motor Neuroscience and Movement Disorders, and Head of the Education Unit, Institute of Neurology, University College London, London, UK Amy Shelly, BSc Project Officer, Paediatric Integrated Cancer Service, Children s Cancer Centre, Royal Children s Hospital, Parkville, Victoria, Australia Elizabeth Waters, DPhil Jack Brockhoff Chair of Child Public Health, McCaughey Centre, VicHealth Centre for the Promotion of Mental Health and Community Wellbeing, School of Population Health, University of Melbourne, Melbourne, Australia

Preface Patient-reported outcome measures (PROMs) are now generally regarded as central to the evaluation of health and medical care. This represents a substantial change in opinion from that of just a few decades ago, when the systematic collection of patient reports of their health and quality of life was a relatively new area of research. Today, PROMs feature in routine data collection systems and are seen as important in the monitoring of health care provision. They are increasingly used as primary outcomes measures in trials. Thus, the measurement of quality of life, or what is sometimes referred to as health-related quality of life, has moved into the mainstream of evaluation. In part, this reflects the growth in patient-centered care, and the importance now placed on public and patient views of health care. Thus, health care must reflect the needs of patients, and assessment of services must reflect both consumer views and patient-reported outcomes. This text is evidence of this move toward patient-centered evaluation and documents the development and validation of quality-of-life measures for use in a wide variety of neurodegenerative, and related, conditions. This book is primarily concerned with the development, validation, and application of disease-specific measures. Such instruments are generally regarded as potentially more precise and sensitive to changes than more generic measures because they are intended to reflect the particular demands of specific conditions. The first chapter documents established methods for the development and validation of instruments xi

xii Preface and introduces readers unfamiliar with the area to the fundamental concepts of measurement in quality-of-life measurement. The following chapters document the current state of the art in measurement of Parkinson s disease, multiple sclerosis, motor neuron disease, progressive supranuclear palsy, multiple system atrophy, Huntington s disease, and dementia. The particular demands of measuring the health status of children are explored in the chapter on cerebral palsy. Although cerebral palsy is not a neurodegenerative disorder in itself, the impact of the condition on growth for children, and on aging for adults, often causes, over time, profound changes in functioning and health status. Consequently, the phenotype of cerebral palsy has been referred to as a progressive neuromuscular condition hence its inclusion here. The impact of serious illness can have substantial effects on family members and friends, and these issues are explored in a chapter on quality of life and carers. The final chapters of the book explore methodologic issues in translation and scoring of instruments, as well as the place of more individualized measures that are specifically designed to reflect the concerns of individual patients. This book is intended to give a clear idea of the range of instruments available, as well as the issues involved in their development, validation, and application. Graham HK. Painful hip dislocation in cerebral palsy. Lancet 2002; 359: 907 908.