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
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•
•
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
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•
•
•
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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