Measuring Health-Related Quality of Life in Neurology Clinical ...

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

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2.5

-3 -2 -1 0 1 2 3

Theta

Info

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tio

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-3 -2 -1 0 1 2 3

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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