measuring health-related quality of life in neurology clinical
TRANSCRIPT
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Measuring Health-Related Quality of Life in Neurology Clinical Research:
Neuro-QOL Item Banks and Disease Targeted Scales
David Cella, Claudia Scala-Moy, Deborah Miller,Amy Peterman, Richard Gershon,
David Victorson, Cindy Nowinski, Seung Choi, Jin-Shei Lai, Rita Bode, and Anthony Reder
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Neuro-QOL Primary Sites
●UNC –Charlotte●UNC –Charlotte
● Westat● Westat
●Cleveland Clinic ●Cleveland Clinic
●Boston U●Boston U
ENH/NU/RIC ♥ENH/NU/RIC ♥ ● NIHNIH● NIHNIH
U. Chicago U. Chicago
–NINDS (Project Officer: Claudia Scala-Moy, Ph.D)
–Principal Investigator: David Cella, PhD
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Objectives of Neuro-QOLRFP-NIH-NINDS-03-04
Develop a core set of QOL questions that cut across chronic neurologic disorders
Develop supplemental questions that address concerns specific to targeted diseases or subgroups of patients
Create a publicly available, adaptable and sustainable system allowing clinical researchers access to a common item repository and computerized adaptive testing (“CAT”)
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What's wrong with today's static measurements ?
01
23
- 1
- 2
- 3
Questionnairewith a widerange -but low precision
12 Questionnaire
with a highprecision -but small range
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Computerized Adaptive Tests
01
23
- 1
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high depression
low depression
01
2
2. Question
12
3. Question
Questionnairewith a highprecision -AND awide range
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“Item Bank”• A large collection of items measuring a single
domain
• Basis for tailored/adaptive testing– Items in the same bank are linked on a common
metric.– Items are selected to maximize precision and
retain clinical relevance
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Target Disorders
• Adult conditions– Stroke– Multiple Sclerosis– Parkinson’s disease– Epilepsy– ALS
• Pediatric conditions– Epilepsy– Muscular dystrophies
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Psycho-metricTesting
Item Bank(IRT-calibrated items)
0.0
0.5
1.0
1.5
2.0
2.5
-3 -2 -1 0 1 2 3
Theta
Info
rma
tio
n
0.0
0.2
0.4
0.6
0.8
1.0
-3 -2 -1 0 1 2 3
Theta
Pro
ba
bil
ity o
f R
esp
on
se
Short FormInstruments
CAT
Literature Review
Item Pool
Patient Focus Groups
Expert Input and Consensus
Existing Items
(PROMIS; AMPAC)
Questionnaireadministered to largerepresentative sample
SecondaryData Analysis
CognitiveTesting
TranslationExpertReview
Newly Written
Items
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Neuro-QOL Testing Samples
• WAVE Ia: Online clinical samples (511 adults; 59 children– Complete
• WAVE Ib: Online general population calibration testing for adults (3000 adults; 1500 children): English and Spanish – Complete
• WAVE II: Clinical validation testing of IRT-calibrated short forms (n=800 baseline; n=400 7 day; n=400 180 day) – In progress
• Today’s results are for WAVE I (a and b) calibration field testing
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Analysis Plan• If N > 500
– PROMIS analysis plan • Reeve et al, 2007; or
– Evaluation of unidimensionality• Descriptive statistics
• Factor analysis
– Estimation of Item Parameter using 2-PL IRT
• If N < 500 (Wave 1a Pediatric)– Descriptive statistics and Rasch analysis
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Wave 1b: Adult Item Bank Information FunctionsPositive Psychological Function
T-score
rel.=.90
rel.=.95
0
20
40
60
80
100
10 20 30 40 50 60 70 80 90
Depression
T-score
rel.=.90
rel.=.95
0
20
40
60
80
100
10 20 30 40 50 60 70 80 90
Anxiety
T-score
rel.=.90
rel.=.95
0
10
20
30
40
50
60
10 20 30 40 50 60 70 80 90
Perceived Cognitive Deficiency
T-score
rel.=.90rel.=.95
0
50
100
150
10 20 30 40 50 60 70 80 90
Applied Cognitive Function
T-score
rel.=.90
rel.=.95
0
20
40
60
80
100
120
140
10 20 30 40 50 60 70 80 90
Stigma
T-score
rel.=.90
rel.=.95
0
20
40
60
80
100
10 20 30 40 50 60 70 80 90
Mobility Ambulation
T-score
rel.=.90rel.=.95
0
50
100
150
10 20 30 40 50 60 70 80 90
Upper Extremity ADL
T-score
rel.=.90rel.=.950
100
200
300
400
500
600
10 20 30 40 50 60 70 80 90
Social Role Ability
T-score
rel.=.90rel.=.95
0
50
100
150
200
250
10 20 30 40 50 60 70 80 90
Social Role Satisfaction
T-score
rel.=.90rel.=.95
0
50
100
150
200
250
300
10 20 30 40 50 60 70 80 90
The shaded areas represent the effective measurement ranges where a reliability of roughly .95 is attainable.
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Reliability and Item Total Correlation Examples
Domain Alpha i-total r
Positive Psychological Function .98 .60 - .91
Depression .98 .64 - .90
Anxiety .97 .56 - .87
Perceived Cognitive Dysfunction .98 .57 - .85
Applied Cognitive Function .97 .54 - .78
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Depression – 24-item bank
Location
50
52
54
56
I felt I had no reason for living
I felt like I needed help for my depression
I felt hopeless
I felt that nothing was interesting
I felt unloved
I felt that I wanted to give up on everything
I felt that nothing could cheer me up
I felt that my life was empty
I had trouble enjoying things that I used to enjoy
I felt worthless
I felt helpless
I withdrew from other people
I felt that I had nothing to look forward to
I had trouble keeping my mind on what I was doing
I found that things in my life were overwhelming
I felt pessimistic
I felt depressed
I felt emotionally exhausted
I felt lonely
I felt that everything I did was an effort
I felt discouraged about the future
I felt unhappy
I was critical of myself for my mistakes
I felt sad
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2 4 6 8 10 12
0.0
0.2
0.4
0.6
0.8
1.0
Depression
Test Length
SE
T-score=30T-score=40T-score=50T-score=60T-score=70
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Mobility Ambulation – 31-item bank
Location
45
50
55
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?Hwalking 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?
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2 4 6 8 10 12
0.0
0.2
0.4
0.6
0.8
1.0
Mobility Ambulation
Test Length
SE
T-score=30T-score=40T-score=50T-score=60T-score=70
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PHYSICALFunction/Health Symptoms
Fatigue
Sleep
Mobility/Ambulation
Sexual
Bowel/Bladder
ADL’s/UE’s
MENTALEmotional Health Cognitive Health
Perceived
Applied
Depression
Stigma
Positive Psych Fn
Anxiety
Personality Change
End of Life Concerns
SOCIALRole Participation Role Satisfaction
B=BankT=Targeted ScaleTD=Targeted scale developed but not tested
Adult Domain Framework for Item Banks and Targeted Scales
B - includes communication problems
B
B
TD
TD
T
T
B
B
B
B
B
B B
T
TD
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PHYSICALFunction/Health Symptoms
Pain
Fatigue
Mobility/Ambulation
ADL’s/UE’s
MENTALEmotional Health Cognitive Health
Depression
Stigma
Anxiety
SOCIAL
B=BankT=Targeted Scale
Pediatric Domain Framework for Item Banks and Targeted Scales
B
B T
T
B
B
T
T
B
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Wave II Clinical Validation Testing
Assessment I (Baseline)
Assessment II (7 days)
Assessment III(180days)
Stroke 100 X X
Multiple Sclerosis 100 X X
Parkinson’s Disease 100 X X
Adult Epilepsy 100 X X
ALS 100 X X
Proxies matched to Stroke 100 X X
Pediatric Epilepsy 50 X XProxies matched to above Epilepsy 50 X X
Pediatric Muscular Dystrophy 50 X X
Proxies matched to above MD 50 X X
Maximum Subtotal 800
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Wave II Clinical Testing Sites•University of Texas•Dartmouth•Northwestern University•University of Chicago•Rehabilitation Institute of Chicago•Cleveland Clinic Foundation•University of Pennsylvania•NorthShore University HealthSystem•Children’s Memorial Hospital (Chicago)•University of California Davis •University of Puerto Rico
N = 150 of 800
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Thank You!
www.NeuroQOL.org