Neuro-QOL: Health-Related Quality of Life Measures for Neurology
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 265200423601C • Thanks to David Cella, Cindy Nowinski, Richard Gershon, David Victorson, Vitali Ustsinovich Neuro-QOL Team 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 NINDS Phase III Stroke Trials Secondary Prevention of Small Subcortical Stroke No QOL outcome measure Field Administration of Stroke Therapy – Magnesium Rankin (1o), Barthel, NIHSS, SIS (2o) Efficacy of a Family Telephone Intervention for Stroke Craig Handicap Assessment and Reporting, Satisfaction with Life Scale Carotid Revascularization Endarterectomy vs Stenting Trial SF36, Frenchay Activity Index Treatment for Post Stroke Depression SSQOL Warfarin-Aspirin Symptomatic Intracranial Disease Study No QOL outcome measure Carotid Occlusion Surgery Study Rankin, NIHSS, mBI, SSQOL Warfarin vs Aspirin in Reduced Cardiac Ejection Fraction No QOL outcome measure Albumin in Acute Stroke EuroQOL 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 Target Conditions • Adult – Epilepsy – Multiple Sclerosis – Parkinson’s disease – Stroke – ALS • Pediatric – Epilepsy – Muscular dystrophies 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 FunctionFine Motor, ADL-B PHYSICAL Function/Health Bowel Function -D Urinary/Bladder Function-D Symptoms MENTAL Cognitive Health Fatigue-B Sleep Disturbance-B Emotional Health Applied Cognition-General Concerns-B Applied Cognition-Executive Function-B Communication-S Lower Extremity FunctionMobility- B Sexual Function-D Depression-B Anxiety-B Stigma-B Positive Affect & Well-Being-B Emotional & Beh. Dyscontrol-B End of Life Concerns-D 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 PHYSICAL Function/Health Pain-B Lower Extremity Function Mobility-S Upper Extremity Function Fine Motor, ADL-S Symptoms Fatigue-B MENTAL Emotional Health Depression-B Anxiety-B Stigma-B Cognitive Health SOCIAL Applied Cognition-General Concerns-B Anger-B Social Relations- Interactions with Peers-B Social Relations-Interactions with Adults-U B=Bank S= Scale U=Domain Being Identified Adult Banks and Scales Domain Anxiety Depression Fatigue Upper Extremity Function - Fine Motor, ADL Lower Extremity Function – Mobility Applied Cognition- Executive Function Applied Cognition- General Concerns Emotional and Behavioral Dyscontrol Positive Affect and Well-Being Sleep Disturbance Ability to Participate in Social Roles and Activities Satisfaction with Social Roles and Activities Stigma Communication Format Bank Bank Bank Bank Bank Bank Bank Bank Bank Bank Bank Bank Bank Scale # Items # Items in Short Form 21 8 24 8 19 8 20 8 19 8 13 8 18 8 18 8 23 9 8 NA 45 8 45 24 5 8 8 NA Pediatric Banks and Scales Domain Format # of # Items in Short Items Form 19 8 Anxiety Bank Depression Bank 17 8 Anger Fatigue Bank Bank 8 13 NA 8 Upper Extremity Function - Fine Motor, ADL Scale 20 NA Lower Extremity Function –Mobility Scale 20 NA Applied Cognition- General Concerns Bank 14 8 Social Relations - Interaction with Peers Bank 16 8 Social Relations - Interaction with Adults Stigma TBD Bank 9 18 NA 8 Pain Bank 10 NA 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? 55 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? 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? T-scores 50 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? walking on uneven surfaces? 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? 45 sitting down on an armless straight chair? Are you able to get on and off the toilet? moving from sitting at the side of the bed to lying down on your back? Applied Cognition - Executive Function 41 40 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? handling an unfamiliar problem? organizing what you want to say? checking the accuracy of financial documents? doing calculations in your head while shopping? 39 38 planning for and completing regularly scheduled weekly tasks? 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? learning new tasks or instructions? carrying on a conversation with a familiar person in a noisy environment? T-Scores planning an activity several days in advance? 37 36 remembering where things were placed or put away? using a local street map to locate a new store or doctor's office? 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? 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? 35 looking up a phone number or address in the phone book? 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 IRTcalibrated 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 Cleveland Clinic Chicago sites : * University of California at Davis * * •NMFF •NorthShore University HealthSystem •NU Parkinson’s Disease Center •Rehabilitation Institute of Chicago •University of Chicago •Children’s Memorial Hospital Medical Center *University of Pennsylvania *University of Texas HSC at San Antonio University of Puerto Rico * Clinical Validation Sample Number completing assessment Baseline 7-day 180-day Multiple Sclerosis 161 125 132 Parkinson’s disease 120 116 108 Adult Epilepsy 119 119 109 Stroke 101 95 90 Stroke Proxies 84 78 73 ALS 80 77 59 Pediatric Epilepsy 62 60 56 Pediatric Epilepsy Proxies 62 60 56 Muscular Dystrophy 51 48 48 Muscular Dystrophy Proxies 51 48 48 Total: 891 826 779 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. Overlap between Neuro-QOL, PROMIS and AM-PAC Domain Neuro-QOL PROMIS AM-PAC Ability to Participate in Social Roles & Activities X X -- Satisfaction with Social Roles & Activities X X -- Lower Extremity Function - Mobility X X X Upper Extremity Function – Fine motor, ADL X X X Depression X X -- Anxiety X X -- Positive Affect and Well-being X X -- Fatigue X X -- Sleep Disturbance X X -- Pain X X -- Stigma X -- -- Applied Cognition – Executive Function X -- -- Applied Cognition – General Concerns X -- X Emotional & Behavioral Dyscontrol X -- -- 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 Function Depression Time 1 Neuro-QOL Physical Function Upper Extremity CAT Item 1 Item 2 Item 3 Item 4 Final Score after 4 items T=25.0 (SE=2.0) Neuro-QOL Applied Cognition Executive Function CAT Item 1 Item 2 Item 3 Item 4 Final Score after 4 items T=26.0 (SE=2.5) Neuro-QOL Depression CAT Item 1 Item 2 Item 3 Item 4 Final Score after 4 items T=59.9 (SE=1.8) Time 1 Scores 65 60 55 50 45 PF Exec 40 Dep 35 30 25 20 1 Time 1 Time 2 Neuro-QOL Physical Function Upper Extremity CAT Item 1 Item 2 Item 3 Item 4 Item 5 Item 6 Final Score after 6 items T=41.8 (SE=2.6) Neuro-QOL Applied Cognition Executive Function CAT Item 1 Item 2 Item 3 Item 4 Item 5 Final Score after 5 items T=42.7 (SE=2.6) Neuro-QOL Depression CAT Item 1 Item 2 Item 3 Item 4 Final Score after 4 items T=51.8 (SE=1.8) Time 1 and 2 Scores 65 60 55 50 45 PF Exec 40 Dep 35 30 25 20 1 Time 1 2 Time 2
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