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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.

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Presentation on theme: "Neuro-QOL: Health-Related Quality of Life Measures for Neurology Research and Practice Claudia Scala Moy, PhD Office of Clinical Research National Institute."— Presentation transcript:

1 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

2 Disclosures No financial disclosures This work represents a collaboration between NINDS and Northwestern University Funded by NINDS contract HHSN C Thanks to David Cella, Cindy Nowinski, Richard Gershon, David Victorson, Vitali Ustsinovich

3 Neuro-QOL Team

4 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

5 NINDS Phase III Stroke Trials Secondary Prevention of Small Subcortical Stroke No QOL outcome measure Field Administration of Stroke Therapy – Magnesium Rankin (1 o ), Barthel, NIHSS, SIS (2 o ) 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 DepressionSSQOL Warfarin-Aspirin Symptomatic Intracranial Disease Study No QOL outcome measure Carotid Occlusion Surgery StudyRankin, NIHSS, mBI, SSQOL Warfarin vs Aspirin in Reduced Cardiac Ejection Fraction No QOL outcome measure Albumin in Acute StrokeEuroQOL

6 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

7 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

8 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

9 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

10 Target Conditions Adult – Epilepsy – Multiple Sclerosis – Parkinson’s disease – Stroke – ALS Pediatric – Epilepsy – Muscular dystrophies

11 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

12 PHYSICAL Adult Domain Framework for Item Banks and Scales Symptoms Function/Health Fatigue-B Sleep Disturbance-B Lower Extremity Function- Mobility- B MENTAL Cognitive Health Emotional Health SOCIAL Ability to Participate in Social Roles & Activities-B Satisfaction with Social Roles & Activities-B Bowel Function -D Upper Extremity Function- Fine Motor, ADL-B Sexual Function-D Depression-B Anxiety-B Stigma-B Positive Affect & Well-Being-B Emotional & Beh. Dyscontrol-B End of Life Concerns-D Communication-S Applied Cognition-General Concerns-B Applied Cognition-Executive Function-B B=Bank S=Scale D=Developed but not Tested Urinary/Bladder Function-D

13 Pediatric Domain Framework for Item Banks and Scales PHYSICAL Symptoms Function/Health Pain-B Fatigue-B Lower Extremity Function Mobility-S Upper Extremity Function Fine Motor, ADL-S MENTAL Cognitive Health Emotional Health Depression-B Anxiety-B Stigma-B Anger-B Applied Cognition-General Concerns-B B=Bank S= Scale U=Domain Being Identified SOCIAL Social Relations-Interactions with Adults-U Social Relations- Interactions with Peers-B

14 Adult Banks and Scales Domain Format # Items # Items in Short Form AnxietyBank218 DepressionBank248 FatigueBank198 Upper Extremity Function - Fine Motor, ADLBank208 Lower Extremity Function – MobilityBank198 Applied Cognition- Executive FunctionBank138 Applied Cognition- General ConcernsBank188 Emotional and Behavioral DyscontrolBank188 Positive Affect and Well-BeingBank239 Sleep DisturbanceBank8NA Ability to Participate in Social Roles and Activities Bank458 Satisfaction with Social Roles and ActivitiesBank458 StigmaBank248 CommunicationScale5NA

15 Pediatric Banks and Scales Domain Format # of Items # Items in Short Form AnxietyBank198 DepressionBank178 AngerBank8NA FatigueBank138 Upper Extremity Function - Fine Motor, ADLScale20NA Lower Extremity Function –MobilityScale20NA Applied Cognition- General ConcernsBank148 Social Relations - Interaction with PeersBank168 Social Relations - Interaction with AdultsTBD9NA StigmaBank188 PainBank10NA

16 Lower Extremity Function – Mobility T-scores 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? 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? 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? 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?

17 Applied Cognition - Executive Function T-Scores 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? 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? 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? 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 to someone? understanding pictures that explain how to assemble something? 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?

18 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.

19 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.)

20 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

21 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.

22 Neuro-QOL Wave 2 Testing Sites Chicago sites : * NMFF NMFF NorthShore University HealthSystem NorthShore University HealthSystem NU Parkinson’s Disease Center NU Parkinson’s Disease Center Rehabilitation Institute of Chicago Rehabilitation Institute of Chicago University of Chicago University of Chicago Children’s Memorial Hospital Children’s Memorial Hospital * University of California at Davis University of Puerto Rico * * University of Texas HSC at San Antonio * Dartmouth-Hitchcock Medical Center Medical Center Cleveland Clinic Cleveland Clinic * * University of Pennsylvania Pennsylvania

23 Clinical Validation Sample Number completing assessment Baseline7-day180-day Multiple Sclerosis Parkinson’s disease Adult Epilepsy Stroke Stroke Proxies ALS Pediatric Epilepsy Pediatric Epilepsy Proxies Muscular Dystrophy5148 Muscular Dystrophy Proxies5148 Total:

24 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.

25 Overlap between Neuro-QOL, PROMIS and AM-PAC Domain Neuro-QOLPROMISAM-PAC Ability to Participate in Social Roles & Activities XX-- Satisfaction with Social Roles & Activities XX-- Lower Extremity Function - Mobility XXX Upper Extremity Function – Fine motor, ADL XXX Depression XX-- Anxiety XX-- Positive Affect and Well-being XX-- FatigueXX-- Sleep DisturbanceXX-- Pain XX-- Stigma X -- Applied Cognition – Executive Function X -- Applied Cognition – General ConcernsX -- X Emotional & Behavioral DyscontrolX --

26 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”)

27 Demonstration of Neuro-QOL CATs in: Upper Extremity Function – Fine Motor, ADL Applied Cognition - Executive Function Depression

28 Time 1

29 Neuro-QOL Physical Function Upper Extremity CAT

30 Item 1

31 Item 2

32 Item 3

33 Item 4

34 Final Score after 4 items T=25.0 (SE=2.0)

35 Neuro-QOL Applied Cognition Executive Function CAT

36 Item 1

37 Item 2

38 Item 3

39 Item 4

40 Final Score after 4 items T=26.0 (SE=2.5)

41 Neuro-QOL Depression CAT

42 Item 1

43 Item 2

44 Item 3

45 Item 4

46 Final Score after 4 items T=59.9 (SE=1.8)

47 Time 1 Scores Time 1

48 Time 2

49 Neuro-QOL Physical Function Upper Extremity CAT

50 Item 1

51 Item 2

52 Item 3

53 Item 4

54 Item 5

55 Item 6

56 Final Score after 6 items T=41.8 (SE=2.6)

57 Neuro-QOL Applied Cognition Executive Function CAT

58 Item 1

59 Item 2

60 Item 3

61 Item 4

62 Item 5

63 Final Score after 5 items T=42.7 (SE=2.6)

64 Neuro-QOL Depression CAT

65 Item 1

66 Item 2

67 Item 3

68 Item 4

69 Final Score after 4 items T=51.8 (SE=1.8)

70 Time 1 and 2 Scores Time 1Time 2


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