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Neuro -QOL: Health-Related Quality of Life Measures for Neurology Research and Practice . Claudia Scala Moy, PhD Office of Clinical Research National Institute of Neurological Disorders and Stroke. Disclosures. No financial disclosures
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Neuro-QOL: Health-Related Quality of Life Measures for Neurology Research and Practice Claudia Scala Moy, PhD Office of Clinical Research National Institute of Neurological Disorders and Stroke
Disclosures • No financial disclosures • This work represents a collaboration between NINDS and Northwestern University • Funded by NINDS contract HHSN 2652004236-01C • Thanks to David Cella, Cindy Nowinski, Richard Gershon, David Victorson, Vitali Ustsinovich
Need for a New Tool in Neurological Disorders • Many Disorders • Multitude of generic and targeted measurement instruments • Many not validated for use in clinical trials • Unresponsive to differences that exist across different conditions or treatments • Limited by floor and ceiling effects • Respondent burden • Research cost • Lack of consensus about best measurement approaches
Objectives of NINDS Quality of Life Initiative • Develop psychometrically robust instrument that is accepted by neurology clinical trials community • Foster inclusion of QOL measures in clinical research
Purpose of contract • Develop a core set of questions that cut across chronic neurological disorders • Develop supplemental questions that address additional concerns of specific diseases, subgroups of patients
Neuro-QOL: Special Features • Target neurological conditions • Adult and pediatric disorders • Inclusion of ethnic minorities • Field tested Spanish-language version • Assessment of acceptability to neurology research and clinical community • IRT-based approach/CAT • Available in the public domain
Selection of Target Conditions • Prevalence of the disease/disorder • Magnitude of the disease’s impact on the individual • The existence of promising current or new treatments on the horizon • Multiple domains affected • Chronic nature of the disease/possibility of seeing HRQL change
Adult Epilepsy Multiple Sclerosis Parkinson’s disease Stroke ALS Pediatric Epilepsy Muscular dystrophies Target Conditions
Neuro-QOL Collaborations • Spinal cord injury (SCI-QOL) – D. Tulsky • Traumatic brain injury (TBI-QOL) – D. Tulsky • Huntington’s Disease (HD-HRQL) – N. Carlozzi • Poly trauma (VA) - • PTSD (VA) – S. Luther
Adult Domain Framework for Item Banks and Scales Upper Extremity Function- Fine Motor, ADL-B PHYSICAL Function/Health Bowel Function -D Urinary/Bladder Function-D Symptoms Fatigue-B Lower Extremity Function- Mobility- B Sleep Disturbance-B Sexual Function-D MENTAL Emotional Health Depression-B Anxiety-B Stigma-B Applied Cognition-General Concerns-B Cognitive Health Positive Affect & Well-Being-B Applied Cognition-Executive Function-B Emotional & Beh. Dyscontrol-B End of Life Concerns-D Communication-S Satisfaction with Social Roles & Activities-B SOCIAL Ability to Participate in Social Roles & Activities-B B=Bank S=Scale D=Developed but not Tested
Pediatric Domain Framework for Item Banks and Scales Lower Extremity Function Mobility-S PHYSICAL Function/Health Upper Extremity Function Fine Motor, ADL-S Pain-B Symptoms Fatigue-B MENTAL Emotional Health Depression-B Anxiety-B Stigma-B Cognitive Health Applied Cognition-General Concerns-B Anger-B Social Relations- Interactions with Peers-B SOCIAL Social Relations-Interactions with Adults-U B=Bank S= Scale U=Domain Being Identified
Lower Extremity Function – Mobility 60 running up and down an incline? taking a 20-minute brisk walk, without stopping to rest? getting into and out of a kneeling position? Are you able to jump up and down? walking 45 minutes on an even surface? climbing stairs step over step without a handrail? going up and down three flights of stairs inside, using a handrail? walking on a slippery surface, outdoors? Are you able to get up off the floor from lying on your back without help? Are you able to go for a walk of at least 15 minutes? 55 How difficult is it for you to go for a walk of at least 15 minutes? standing up from a low, soft couch? crossing the road at a 4-lane traffic light with curbs? going up and down a flight of stairs inside, using a handrail? walking in a busy place without losing your balance? Are you able to run errands and shop? sitting down on a low, soft couch? getting into and out of a truck, bus, shuttle van, or sport utility vehicle? Are you able to step up and down curbs? T-scores walking on uneven surfaces? 50 opening a window above shoulder height, while standing? walking in a dark room without falling? standing up from an armless straight chair? using an escalator? Are you able to push open a heavy door? Are you able to get out of bed into a chair? moving from lying on your back to sitting on the side of the bed? Are you able to get in and out of a car? sitting down on an armless straight chair? Are you able to get on and off the toilet? 45 moving from sitting at the side of the bed to lying down on your back?
Applied Cognition - Executive Function 41 remembering a list of 4 or 5 errands without writing it down? keeping important personal papers such as bills, insurance documents and tax forms organized? reading a long book (over 100 pages) over a number of days? taking care of complicated tasks like managing a checking account or getting appliances fixed? 40 handling an unfamiliar problem? organizing what you want to say? checking the accuracy of financial documents? doing calculations in your head while shopping? planning for and completing regularly scheduled weekly tasks? 39 explaining how to do something involving several steps to another person? managing your time to do most of your daily activities? counting the correct amount of money when making purchases? have planning what to do in the day? 38 learning new tasks or instructions? carrying on a conversation with a familiar person in a noisy environment? planning an activity several days in advance? remembering where things were placed or put away? using a local street map to locate a new store or doctor's office? 37 T-Scores putting words together to form grammatically correct sentences? remembering to take medications at the appropriate time? planning for and keeping appointments that are not part of your weekly routine? carrying on a conversation with a small group of familiar people? 36 dialing familiar numbers such as a family member or doctor? reading and following complex instructions? composing a brief note or e-mail to someone? understanding pictures that explain how to assemble something? looking up a phone number or address in the phone book? 35 making yourself understood to other people during ordinary conversations? understanding familiar people during ordinary conversations?
Advantages of Short-Forms Developed from Item Banks • Select a set of items that are matched to the severity level of the target population. • All scales built from the same item bank are linked on a common metric.
Validation phase • Multiple disease groups • Ethnic minorities • Geographically diverse populations • Quality of proxy reporting • Evaluation of modes of administration • Approaches to missing data • Longitudinal study issues (response to change, practice effects, etc.)
Neuro-QOL Testing Highlights • WAVE Ia: Online clinical testing in adults (n=500) and children (n=100) • WAVE Ib: Online English and Spanish general population calibration testing for adults (n= 3000) and children (n=1500) • WAVE II: Clinical validation testing of IRT-calibrated short forms
Clinical Validation of Short Forms Purpose: to evaluate the reliability, validity and responsiveness of Neuro-QOL short forms and scales in clinical neurology populations • Administered Neuro-QOL Short Forms and clinical validation measures (both cross-disease and disease-specific), physician ratings and chart review at baseline and at a 180-day follow up (to assess responsiveness). • Test-retest reliability of the Neuro-QOL Short Forms was evaluated at 7 days.
Neuro-QOL Wave 2 Testing Sites *Dartmouth-Hitchcock Medical Center Cleveland Clinic * • Chicago sites : * • NMFF • NorthShore University HealthSystem • NU Parkinson’s Disease Center • Rehabilitation Institute of Chicago • University of Chicago • Children’s Memorial Hospital *University of Pennsylvania * University of California at Davis *University of Texas HSC at San Antonio University of Puerto Rico*
Clinical Validation Results • High internal consistency and reliability • Moderate to high correlations with generic and disease-specific measures • Responsiveness comparable with legacy instruments • Neuro-QOL short forms successfully discriminate between patients grouped by disease severity or other clinical factor.
Future of Neuro-QOL • Create a publicly available, adaptable and sustainable system allowing clinical researchers access to a common item repository and computerized adaptive testing (“CAT”)
Demonstration of Neuro-QOL CATs in: Upper Extremity Function – Fine Motor, ADL Applied Cognition - Executive FunctionDepression
Time 1 Scores Time 1
