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
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
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”)
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
Computerized Adaptive Tests
01
23
- 1
- 2
- 3
high depression
low depression
01
2
2. Question
12
3. Question
Questionnairewith a highprecision -AND awide range
“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
Target Disorders
• Adult conditions– Stroke– Multiple Sclerosis– Parkinson’s disease– Epilepsy– ALS
• Pediatric conditions– Epilepsy– Muscular dystrophies
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
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
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
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.
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
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
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
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?
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
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
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
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
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
Thank You!
www.NeuroQOL.org