Transcript Slide 1

Other databases in
the United States
1. QUEST-RA
2. National Data Bank for
the rheumatic diseases
3. RAPID Scores
[email protected]
Quantitative Patient Questionnaire Monitoring
in Standard Clinical Care of Patients with
Rheumatoid Arthritis (QUEST-RA)
• 100 RA patients from site over 2-6 months
• Patient questionnaire: 4 pages HAQ + HAQ
II,MDHAQ items; MDHAQ
• MD questionnaire: 3 pages SPERA Standard Protocol to Evaluate RA: clinical
features, medications, 42 joint count
4 major goals:
• Experience with questionnaires in
standard care
• Database for patient scores, DAS, work
status, etc. in different sites and countries
• Compare therapies atdifferent sites
• Paient self-report RADAI joint count vs
physician/assessor joint count
Quantitative Patient Questionnaire Monitoring
in Standard Clinical Care of Patients with
Rheumatoid Arthritis (QUEST-RA)
Possible advantages over existing
databases:
• All RA patients over a given period –
not only those taking anti-TNF, etc.
• Predicition of going on to anti-TNF?
• Enhance clinical rheumatology as a
quantitaive science
Please contact TP if interested.
[email protected]
Thank you
The National Data Bank for
Rheumatic Diseases (NDB)
 Founded 1998
 Goal: a generalizable, comprehensive, valid,
reliable, believable national databank for research
and teaching
 Surveys at 6 month intervals
 Mailed surveys, Internet, Telephone interviewing
 Follow-up medical records, MD and patient contact
 RA 77%, also OA, SLE, fibromyalgia, etc.
 Programmers, research analysts, verifiers, QC
staff, callers, records department, mortality
staff, designers, administrators
NDB participation by year
1999.1
1999.2
2000.1
2000.2
2001.1
2001.2
2002.1
2002.2
2003.1
2003.2
2004.1
2004.2
2005.1
2005.2
7,712
11,986
11,515
10,937
11,158
12,979
13,090
13,562
13,435
13,353
13,231
12,395
12,961
11,450
0
5,000
10,000
Completed questionnaires
15,000
Treatment in NDB
At Last Assessment
(NDB – 2005)
Variable
N
Leflunomide (%)
Auranofin (%)
Azathioprine (%)
Sulfasalazine (%)
Cyclosporin (%)
Cyclophosphamide (%)
Injectable Gold (%)
Minocycline (%)
Penicillamine (%)
Hydroxychloroquine (%)
Etanercept (%)
Adalimumab (%)
Anakinra (%)
Infliximab (%)
Methotrexate (%)
No DMARD/biologic (%)
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
22495
Mean
15.0
0.3
2.3
5.4
0.3
0.1
1.0
1.4
0.2
17.3
12.4
5.2
0.7
27.5
49.8
16.4
Sum
3378
59
509
1209
74
26
230
313
33
3897
2796
1165
158
6186
11206
3692
Variables
 Demographics (full)
 Treatments (all)
 Adverse events
 cardio-vascular, GI,
immune disorders,
 Infection, cancer, etc
 Hospitalization
 Work
 Costs, cost-utility
 SF-36, HAQ family
 Pain, fatigue, sleep,
satisfaction, anxiety,
depression, global,
RADAI,
 Utilities:
EuroQol,HUI, Sf6D
EuroQol
NDB Data Processing Hardware
 T1 -> Cisco Router
 Firewall (Sonicwall)
 WWW (HTTP)
 VPN
 E-mail
 Network printers (4)
 High speed scanner
 Digital scanner
 4 Servers (hardware)








Web Server
NT2 (E-mail)
NT1 (SQL Database)
FS-ARC (On-line SQL DB
for WebQuest)
Workstations (27)
VPN workstations (5)
Tape back-up
Disk-based back-up
(SQL)
The Report Project
NDB Privacy and
Confidentiality Policy
 General
 Protection of participant identifying
information (PII) in computer databases
 Faxed questionnaires
 Hard Copy questionnaires
 Web-based data entry
 SSL Encryption
HIPPA Compliance
 As a covered entity under HIPAA, NDB has met the
timelines for implementation of the initial HIPAA
standards (privacy and security) that are applicable to
our business. IRB approval of this implementation is
available upon request.
 The NDB has processes and procedures in place as
they relate to the protection of data, as well as patient
information.
Data Bank Questions/Projects
 Costs of illness, C/E, C/U
 Rates/predictors of outcomes: mortality,
joint replacement, work disability, ADR
 Effectiveness of therapies
 Measurement of severity, development
of clinical and research instruments
 Psychosocial issue & predictors
 Statistical techniques
Complexities in assessment of
patients with rheumatic diseases:
1.
No single “gold standard” (eg, blood pressure,
cholesterol) for clinical trials or standard care:
therefore, indices of 3-7 measures.
2.
Laboratory tests limited in both diagnosis and
treatment - primary criteria are clinical.
3.
Patient questionnaires to assess physical
function, pain, global status, often best
quantitative measures.
Indices to assess RA
ACR
# Tender joints
# Swollen joints
MD global
ESR or CRP
Patient function
Patient pain
Patient global
√
√
√
√
√
√
√
DAS28 SDAI
√
√
√
--√
√
√
√
√
--√
CDAI
√
√
√
---√
PAS/
RAPID
----√
√
√
RAPID (Routine Assessment of
Patient Index Data) Measures
Index:
RAPID RAPID RAPID
2
3
4
PTJC
RAPID
4
MDGL
RAPID
4
MDJC
RAPID
5
Physical
Function
√
√
√
√
√
Pain
√
√
√
√
√
√
√
√
√
√
Patient Global
Estimate
√
√
Patient Joint
Count (RADAI)
√
√
MD/Assessor
Joint Count
MD/Assessor
Global Estimate
√
√
√
3. Please place a check (√) in the appropriate spot to indicate the amount of pain you
are having today in each of the joint areas listed below:
None
Mild Moderate Severe
None
Mild Moderate Severe
a.LEFT FINGERS
b.LEFT WRIST
c.LEFT ELBOW
d.LEFT SHOULDER
e.LEFT HIP
f.LEFT KNEE
g.LEFT ANKLE
h.LEFT TOES
i.RIGHT FINGERS
j.RIGHT WRIST
k.RIGHT ELBOW
l.RIGHT SHOULDER
m.RIGHT HIP
n.RIGHT KNEE
o.RIGHT ANKLE
p.RIGHT TOES
q.NECK
r.BACK
RADAI vs Core Data Set measures (n=274)
RADAI
Swollen 28
Tender 28
MD Global VAS
ESR
CRP
FN MDHAQ
Pt Global VAS
Pain VAS
RADAI
SJC 28
TJC 28
ESR
--0.42
0.55
0.52
0.13*
0.08***
0.68
0.69
0.71
0.42
--0.55
0.74
0.23
0.18**
0.47
0.36
0.39
0.55
0.55
--0.57
0.32
0.21
0.52
0.53
0.56
0.13*
0.23
0.32
0.26
--0.50
0.25
0.21
0.21
Adjusted for age, disease duration, education and center, All p<0.0001,
except *p=0.035, **p=0.003, ***p>0.05
RADAI self-report Jt Count vs MD TJtC
RADAI
score
(0-48)
0-5
5-9
10-19
20-48
Total
MD tender joint count (0-28)
0-2
91
(88%)
39
(63%)
31
(48%)
12
(27%)
173
(63%)
3-5
8 (8%)
14
(23%)
16
(25%)
1 (2%)
39
(14%)
6-11
12+
3 (3%)
2 (2%)
6 (10%)
3 (5%)
14
(22%)
16
(36%)
39
(14%)
3 (5%)
15
(34%)
23
(8%)
Total
104
(38%)
62
(23%)
64
(23%)
44
(16%)
274
Spearman Correlation Coefficients in
274 Patients with RA – All p<0.001
Measure
CDAI
RADAI 3
RAPID 4 RADAI
RAPID 4 MD SJC
RAPID 4 MD TJC
RAPID 4 MD S&T
DAS vs
0.84
0.66
0.65
0.72
0.73
0.73
CDAI vs
--0.74
0.75
0.83
0.81
0.83
CDAI by RAPID4 with RADAI Joint
• CCC=0.558
• Line of
perfect
concordance
• Actual
Concordance
DAS28 Categories
<2.6
= Remission
2.6-3.19
= Low DAS
3.2-5.1
= Moderate DAS
>5.1
= High DAS
DAS28 and RAPID RA Categories
DAS Categories
<2.6
=
Remission
2.6-3.19 =
Low DAS
3.2-5.1 =
Moderate DAS
>5.1
=
High DAS
Proposed RAPID Categories
< 1.0
=
Near Remission
1.01-2
=
Low Severity
2.01-4.0 =
Moderate Severity
>4.0
=
High Severity
DAS28 compared to RAPID 3 scores in
274 patients at 3 sites
RAPID 3 Scores
DAS28
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
0-2.6=Remission
50 (50%)
23 (23%)
18 (18%)
2.6-3.19=Low
DAS
10 (27%)
8 (23%)
15 (41%)
4 (11%)
37 (14%)
3.2-5.1=Moderate
DAS
7 (8%)
16 (18%)
26 (30%)
38 (44%)
87 (32%)
>5.1=High DAS
1 (2%)
1 (2%)
11 (22%)
36 (73%)
49 (18%)
68 (25%)
48 (18%)
70 (26%)
88 (32%)
274
Total
4.110=High
severity
Total
10 (10%) 101 (37%)
DAS28 compared to RAPID 4 MDCT
scores in 274 patients at 3 sites
RAPID 4 MDCT Scores
DAS28
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.110=High
severity
Total
0-2.6=Remission
48 (48%)
25 (25%)
22 (22%)
6 (6%)
101 (37%)
2.6-3.19=Low
DAS
10 (27%)
9 (24%)
15 (41%)
3 (8%)
37 (14%)
3.2-5.1=Moderate
DAS
7 (8%)
15 (17%)
34 (39%)
31 (36%)
87 (32%)
>5.1=High DAS
0 (0%)
2 (4%)
9 (18%)
38 (78%)
49 (18%)
65 (24%)
51 (19%)
80 (29%)
78 (28%)
274
Total
CDAI compared to RAPID 3 scores in
274 patients at 3 sites
RAPID 3 Scores
CDAI
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.1-10=
High
severity
Total
0-3.3=Remission
39 (70%)
13 (23%)
4 (7%)
0 (0%)
56 (20%)
3.4-11.0=Low
activity
23 (25%)
24 (26%)
31 (33%)
15 (16%)
93 (34%)
11.1-26.0=
Moderate activity
5 (5%)
11 (12%)
29 (31%)
50 (53%)
95 (35%)
>26=High activity
1 (3%)
0 (0%)
6 (20%)
23 (77%)
30 (11%)
68 (25%)
48 (18%)
70 (26%)
88 (32%)
274
Total
DAS VS RAPID IN ABATACEPT TRIALS--AIM
DAS28
RAPID2
RAPID3
RAPID4-MD RAPID4-JC
RAPID5
0%
-10%
Mean % Change
-20%
-21%
-25%
-30%
-28%
-27%
-30%
-32%
-40%
-43%
-47%
-50%
-54%
-56%
-60%
-61%
-70%
-52%
Control
Abatacept
Mean Time to Score
120
100
Seconds
80
60
40
20
0
28 Joint
Count
Rheum #1
Rheum #2
Rheum #3
Mean of Rheum #1 #2 #3
84
113
71
90
DAS 28 – HAQ FN MDHAQ
enter
+ PN, GL FN + PN,
numbers
VAS
GL VAS
12.9
16.8
14.6
14.6
41.5
42.2
41.9
6.4
8.5
7.5
7.5
RAPID 3 RAPID
RAPID
RAPID2 = FN, PN, 4MD=RA 4JC =
GL
PID 3+MD RAPID 3
4.3
4.4
4
4.3
Format
9.2
12.1
9.1
9.6
11.8
16.1
12
12.2
19
22.8
15.3
19
RAPID 5
19.4
27.3
17.5
19.4
Saving time and improving
care with a multidimensional
health assessment
questionnaire: 10 practical
considerations
T Pincus, Y Yazici, M Bergman
J Rheumatol 33:448-454, 2006
Question for Rheumatologists
For patients with RA under your care (not including
patients in clinical trials), how often do you perform
formal tender and swollen joint counts?
Never
13%
1–24% of visits
25–49% of visits
50–74% of visits
75–99% of visits
Always
32%
11%
14%
16%
14%
Pincus and Segurado
Ann Rheum Dis 2006
Multi-Dimensional Health Assessment Questionnaire (R771-NP2)
This questionnaire includes information not available from blood tests, X-rays, or any source other
than you. Please try to answer each question, even if you do not think it is related to you at this time.
Try to complete as much as you can yourself, but if you need help, please ask. There are no right or
FOR OFFICE
wrong answers. Please answer exactly as you think or feel. Thank you.
USE ONLY
MDHAQ
Page 1
1. Please check (√) the ONE best answer for your abilities at this time:
Without
With
With
OVER THE LAST WEEK, were you able to:
ANY
SOME
MUCH
Difficulty
Difficulty
Difficulty
a. Dress yourself, including tying shoelaces and
doing buttons?
_____0
_____1
_____2
b. Get in and out of bed?
_____0
_____1
_____2
c. Lift a full cup or glass to your mouth?
_____0
_____1
_____2
d. Walk outdoors on flat ground?
_____0
_____1
_____2
e. Wash and dry your entire body?
_____0
_____1
_____2
f. Bend down to pick up clothing from the floor?
_____0
_____1
_____2
g. Turn regular faucets on and off?
_____0
_____1
_____2
h. Get in and out of a car, bus, train, or airplane?
_____0
_____1
_____2
i. Walk two miles or three kilometers, if you wish?
_____0
_____1
_____2
j. Participate in recreational activities and sports
_____0
_____1
_____2
as you would like, if you wish?
k. Get a good night’s sleep?
_____0
_____1.1 _____2.2
l. Deal with feelings of anxiety or being nervous?
_____0
_____1.1 _____2.2
m.Deal with feelings of depression or feeling blue?
_____0
_____1.1 _____2.2
UNABLE
To Do
_____3
_____3
_____3
_____3
_____3
_____3
_____3
_____3
_____3
_____3.3
_____3.3
_____3.3
NO                      PAIN AS BAD AS
PAIN 0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10 IT COULD BE
3. Please place a check (√) in the appropriate spot to indicate the amount of pain you
are having today in each of the joint areas listed below:
None
Mild Moderate Severe
None
Mild Moderate Severe
i.RIGHT FINGERS
j.RIGHT WRIST
k.RIGHT ELBOW
l.RIGHT SHOULDER
m.RIGHT HIP
n.RIGHT KNEE
o.RIGHT ANKLE
p.RIGHT TOES
q.NECK
r.BACK
2
4. Considering all the ways in which illness and health conditions may affect you at this
time, please indicate below how you are doing:
VERY                     
WELL 0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10
For Office Use Only: RAPID 3
Please turn to the other side
RAPID 3 (0-10)
RAPID 4
VERY
POORLY
16=5.3
17=5.7
18=6.0
19=6.3
20=6.7
21=7.0
22=7.3
23=7.7
24=8.0
25=8.3
26=8.7
27=9.0
28=9.3
29=9.7
30=10
2.PN (0-10)
4.PTGL (0-10)
RAPID 3 (0-30)
3.a-pPTJT(0-10)
1=0.2
25=5.2
2=0.4
26=5.4
3=0.6
27=5.6
4=0.8
28=5.8
5=1.0
29=6.0
6=1.3
30=6.3
7=1.5
31=6.4
8=1.7
32=6.7
9=1.9
33=6.9
10=2.1 34=7.1
11=2.3 35=7.3
12=2.5 36=7.5
13=2.7 37=7.7
14=2.9 38=7.9
15=3.1 39=8.1
16=3.3 40=8.3
17=3.5 41=8.5
18=3.8 42=8.8
19=4.0 43=9.0
20=4.2 44=9.2
21=4.4 45=9.4
22=4.6 46=9.6
23=4.8 47=9.8
24=5.0 48=10
?
?
?
?
RAPID 4 (0-40)
RAPID 4 (0-10)
NR: 1=0.3, 2=0.7, 3=1.0 LS: 4=1.3, 5=1.7, 6=2.0
NR: 1=0.3, 2=0.5, 3=0.8, 4=1.0
LS: 5=1.3, 6=1.5, 7=1.8, 8=2.0
MS: 7=2.3, 8=2.7, 9=3.0, 10=3.3, 11=3.7, 12=4.0
MS: 9=2.3, 10=2.5, 11=2.8, 12=3.0, 13=3.3, 14=3.5, 15=3.8, 16=4.0
HS: 13=4.3, 14=4.7, 15=5.0, 16=5.3, 17=5.7,18=6.0,
HS: 17=4.3, 18=4.5, 19=4.8, 20=5.0, 21=5.3, 22=5.5, 23=5.8, 24=6.0,
19=6.3, 20=6.7, 21=7.0, 22=7.3, 23=7.7, 24=8.0,
25=6.3, 26=6.5, 27=6.8, 28=7.0, 29=7.3, 30=7.5, 31=7.8, 32=8.0,
25=8.3, 26=8.7, 27=9.0, 28=9.3, 29=9.7, 30=10.0
33=8.3, 34=8.5, 35=8.7, 36=9.0, 37=9.3, 38=9.5, 39=9.8, 40=10.0
NR: 1=0.2, 2=0.4, 3=0.6, 4=0.8 5=1.0 LS: 6=1.2, 7=1.4, 8=1.6, 9=1.8, 10=2.0,
RAPID 5
MS:11=2.2, 12=2.4, 13=2.6, 14=2.8, 15=3.0, 16=3.2, 17=3.4, 18=3.6, 19=3.8, 20=4.0
(0-10)
HS: 21=4.2, 22=4.4, 23=4.6, 24=4.8, 25=5.0, 26=5.2, 27=5.4, 28=5.6, 29=5.8, 30=6.0, 31=6.2, 32=6.4, 33=6.6, 34=6.8, 35=7.0,
36=7.2, 37=7.4, 38=7.6, 39=7.8, 40=8.0, 41=8.2, 42=8.4, 43=8.6, 44=8.8, 45=9.0, 46=9.2, 47=9.4, 48=9.6, 49=9.8, 50=10.0
Copyright: Health Report Services, Telephone 615-936-2151, E-mail [email protected]
1=0.3
2=0.7
3=1.0
4=1.3
5=1.7
6=2.0
7=2.3
8=2.7
9=3.0
10=3.3
11=3.7
12=4.0
13=4.3
14=4.7
15=5.0
_____3
2. How much pain have you had because of your condition OVER THE PAST WEEK? Please
indicate below how severe your pain has been:
a.LEFT FINGERS
b.LEFT WRIST
c.LEFT ELBOW
d.LEFT SHOULDER
e.LEFT HIP
f.LEFT KNEE
g.LEFT ANKLE
h.LEFT TOES
1.a-j FN (0-10)
\ \\
MDGLOBAL(0-10))
RAPID 5 (0-50)
35
Median number of seconds to score various RA measures
120
100
Seconds
80
60
40
20
0
Rheum #1
Rheum #2
Rheum #3
Mean
28 JT
CT
DAS28
84
113
71
90
12.9
16.8
14.6
14.6
HAQ MDHAQ
RAPID
FN +
+ PN, RAPID2 RAPID 3
4MD
PN, GL
GL
41.5
42.2
41.9
6.4
8.5
7.5
7.5
4.3
4.4
4
4.3
9.2
12.1
9.1
9.6
11.8
16.1
12
12.2
RAPID
RAPID 5
4JC
19
22.8
15.3
19
19.4
27.3
17.5
19.4
3. Please place a check (√) in the appropriate spot to indicate the amount of pain you
are having today in each of the joint areas listed below:
None
Mild Moderate Severe
None
Mild Moderate Severe
a.LEFT FINGERS
b.LEFT WRIST
c.LEFT ELBOW
d.LEFT SHOULDER
e.LEFT HIP
f.LEFT KNEE
g.LEFT ANKLE
h.LEFT TOES
i.RIGHT FINGERS
j.RIGHT WRIST
k.RIGHT ELBOW
l.RIGHT SHOULDER
m.RIGHT HIP
n.RIGHT KNEE
o.RIGHT ANKLE
p.RIGHT TOES
q.NECK
r.BACK
Methods
• A cross-sectional database of 100
consecutive patients with RA was
established at 3 sites:
New York –Yazici, Philadelphia –
Bergman, Nashville –Pincus.
• The rheumatologists completed a
28 joint count.
• Patients completed an expanded
health assessment questionnaire
(HAQ), including a self-report
RADAI joint count.
Indices to assess RA
ACR
DAS28 SDAI
CDAI
# Tender joints
√
√
√
√
# Swollen joints
√
√
√
√
MD global
√
-
√
√
ESR or CRP
√
√
√
--
Patient function
√
--
--
--
Patient pain
√
--
--
--
Patient global
√
√
√
√
Indices to assess RA + RAPID=
Routine Assessment of Patient Index Data
# Tender joints
√
√
√
√
PAS/
RAPID
--
# Swollen joints
√
√
√
√
--
MD global
√
-
√
√
--
ESR or CRP
√
√
√
--
--
Patient function
√
--
--
--
√
Patient pain
√
--
--
--
√
Patient global
√
√
√
√
√
ACR
DAS28 SDAI
CDAI
RAPID (Routine Assessment of
Patient Index Data) Measures
Index:
RAPID 3
RAPID 4
PT JC
RAPID 4 MD
JC
Physical
Function
√
√
√
Pain
√
√
√
Patient Global
Estimate
√
√
√
Patient Joint
Count (RADAI)
MD/Assessor
Joint Count
MD/Assessor
Global Estimate
√
√
DAS28 and proposed
RAPID Categories
DAS28 Categories
<2.6
=
Remission
2.6-3.19 =
Low DAS
3.2-5.1 =
Moderate DAS
>5.1
=
High DAS
Proposed RAPID Categories
< 1.0
=
Near Remission
1.01-2
=
Low Severity
2.01-4.0 =
Moderate Severity
>4.0
=
High Severity
DAS28 compared to RAPID 4 MDS&T
scores in 274 patients at 3 sites
RAPID 4 MDS&T Scores
DAS28
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.110=High
severity
Total
0-2.6=Remission
53 (52%)
28 (28%)
17 (17%)
3 (3%)
101 (37%)
2.6-3.19=Low
DAS
10 (27%)
13 (35%)
13 (35%)
1 (3%)
37 (14%)
3.2-5.1=Moderate
DAS
6 (7%)
18 (21%)
41 (47%)
22 (25%)
87 (32%)
>5.1=High DAS
0 (0%)
2 (4%)
11 (22%)
36 (73%)
49 (18%)
69 (25%)
61 (22%)
82 (30%)
62 (23%)
274
Total
DAS28 compared to RAPID 3 scores in
274 patients at 3 sites
RAPID 3 Scores
DAS28
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
0-2.6=Remission
50 (50%)
23 (23%)
18 (18%)
2.6-3.19=Low
DAS
10 (27%)
8 (23%)
15 (41%)
4 (11%)
37 (14%)
3.2-5.1=Moderate
DAS
7 (8%)
16 (18%)
26 (30%)
38 (44%)
87 (32%)
>5.1=High DAS
1 (2%)
1 (2%)
11 (22%)
36 (73%)
49 (18%)
68 (25%)
48 (18%)
70 (26%)
88 (32%)
274
Total
4.110=High
severity
Total
10 (10%) 101 (37%)
CDAI compared to RAPID 4 MDS&T
scores in 274 patients at 3 sites
RAPID 4 MDS&T Scores
CDAI
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.1-10=
High
severity
Total
0-3.3=Remission
43 (77%)
12 (21%)
1 (2%)
0 (0%)
56 (20%)
3.4-11.0=Low
activity
23 (25%)
36 (39%)
31 (33%)
3 (3%)
93 (34%)
11.1-26.0=
Moderate activity
3 (3%)
12 (13%)
45 (47%)
35 (37%)
95 (35%)
>26=High activity
0 (0%)
1 (3%)
5 (17%)
24 (80%)
30 (11%)
69 (25%)
61 (22%)
82 (30%)
62 (23%)
274
Total
CDAI compared to RAPID 3 scores in
274 patients at 3 sites
RAPID 3 Scores
CDAI
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.1-10=
High
severity
Total
0-3.3=Remission
39 (70%)
13 (23%)
4 (7%)
0 (0%)
56 (20%)
3.4-11.0=Low
activity
23 (25%)
24 (26%)
31 (33%)
15 (16%)
93 (34%)
11.1-26.0=
Moderate activity
5 (5%)
11 (12%)
29 (31%)
50 (53%)
95 (35%)
>26=High activity
1 (3%)
0 (0%)
6 (20%)
23 (77%)
30 (11%)
68 (25%)
48 (18%)
70 (26%)
88 (32%)
274
Total
RAPID 4 RADAI compared to RAPID 4
MDS&T scores in 274 patients at 3 sites
RAPID 4 MDS&T Scores
RAPID 4
RADAI
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.1-10=
High
severity
Total
0-1.0=Near
remission
64 (96%)
3 (5%)
0 (0%)
0 (0%)
67 (24%)
1.1-2.0=Low
severity
5 (9%)
46 (84%)
4 (7%)
0 (0%)
55 (20%)
2.1-4.0=
Moderate severity
0 (0%)
12 (16%)
61 (84%)
0 (0%)
73 (27%)
4.1-10=High
severity
0 (0%)
0 (0%)
17 (22%)
62 (78%)
79 (29%)
69 (25%)
61 (22%)
82 (30%)
62 (23%)
274
Total
RAPID 3 compared to RAPID 4 RADAI
scores in 274 patients at 3 sites
RAPID 4 RADAI Scores
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.1-10=
High
severity
Total
0-1.0=Near
remission
65 (96%)
3 (4%)
0 (0%)
0 (0%)
68 (25%)
1.1-2.0=Low
severity
2 (4%)
43 (90%)
3 (6%)
0 (0%)
48 (18%)
2.1-4.0=
Moderate severity
0 (0%)
9 (13%)
57 (81%)
4 (6%)
70 (26%)
4.1-10=High
severity
0 (0%)
0 (0%)
13 (15%)
75 (85%)
88 (32%)
67 (24%)
55 (20%)
73 (27%)
79 (29%)
274
RAPID 3
Total
RAPID 3 compared to RAPID 4 MDS&T
scores in 274 patients at 3 sites
RAPID 4 MDS&T Scores
0-1.0=Near
remission
1.1-2.0=
Low
Severity
2.1-4.0=
Moderate
severity
4.1-10=
High
severity
Total
0-1.0=Near
remission
65 (96%)
3 (4%)
0 (0%)
0 (0%)
68 (25%)
1.1-2.0=Low
severity
4 (8%)
43 (90%)
1 (2%)
0 (0%)
48 (18%)
2.1-4.0=
Moderate severity
0 (0%)
15 (21%)
54 (77%)
1 (1%)
70 (26%)
4.1-10=High
severity
0 (0%)
0 (0%)
27 (31%)
61 (69%)
88 (32%)
69 (25%)
61 (22%)
82 (30%)
62 (23%)
274
RAPID 3
Total
Criteria for clinical measure
• Clinical trials -
– Validity – does it measure what is
supposed to be measured?
– Reliability – is it reproducible?
• Clinical care – also consider
– Feasibility – can it be performed?
– Acceptability – will clinicians
assess it?
Patient questionnaires for clinical
research and improved standard
patient care: is it better to have 80%
of the information in 100% of
patients or 100% of the information
in 5% of patients?
T Pincus, F Wolfe
J Rheumatol 32:575-577, 2005.
Conclusions
•A self-report RADAI joint count is confirmed
to give information similar to a tender joint
count performed by an assessor.
•Self-report joint counts might be considered
as a routine procedure for standard care of
patients with rheumatic diseases.
•A self-report RADAI joint count might be
included in clinical trial protocols to assess
longitudinal performance to distinguish
between active and control treatements.
Conclusions
•We hope to collaborate with 30
rheumatologists to perform comparisons
of self-report joint counts and
MD/assessor joint counts in 100 RA
patients in their clinical care.
•If interested, please contact:
[email protected]
Continuous quality improvement
based on MDHAQ indices
1. MDHAQ for feasibility
2. Easy scoring
3. Flowsheets–lab and drugs
4. Index or indices
5. Categories of severity
6. Continuous quality improvement
for treatment decisions
Patients seen for standard
rheumatoid arthritis care
have significantly better
articular, radiographic,
laboratory, and functional
status in 2000 than in 1985
T Pincus, T Sokka, H Kautiainen
Arthritis Rheum 52:1009-1019, 2005
Cross-Sectional Data in Patients With RA:
Cohort #2 in 1985 and Cohort #4 in 2000:
Swollen Joint Count Scores
1985
2000
20
Swollen Joint Count 28
Swollen Joint Count 28
20
16
12
8
4
0
16
12
8
4
0
0
5
10
15
Disease Duration (Years)
20
0
5
10
15
Disease Duration (Years)
Pincus, Sokka, Kautiainen, Arth Rheum 52:1009, 2005
20
1985
30
RF-
25
RF+
20
15
10
5
0
0
5
10
Disease duration
15
2000
Larson score for hands, % of max
Larson score for hands, % of max
Cross-Sectional Data in RA Patients:
Cohort #2- 1985 and Cohort #4-2000:
Larsen X-Ray score,% of maximum
30
25
20
RF+
15
10
RF
55
positive
RF-
0
00
0
5
10
Disease duration
Pincus, Sokka, Kautiainen, Arth Rheum 52:1009, 2005
15
Cross-Sectional Data in Patients With RA:
Cohort #2 in 1985 and Cohort #4 in 2000:
Multidimensional Health Assessment Questionnaire (MDHAQ)
scores
2000
2.0
2.0
1.5
1.5
MHAQ
MHAQ
1985
1.0
1.0
0.5
0.5
0.0
0.0
0
5
10
15
Disease Duration (Years)
20
0
5
10
15
20
Disease Duration (Years)
Pincus, Sokka, Kautiainen, Arth Rheum 52:1009, 2005
The treatment of
rheumatoid arthritis:
getting better
all the time?
RF van Vollenhoven, L Klareskog
Arthritis Rheumatism 52:991-994, 2005
Six-year report of the STURE
registry for biologicals in
rheumatology: satisfactory
overall results, but plenty of
room of improvement
RF van Vollenhoven, C Cullinane,
J Bratt, L Klareskog
Arthritis Rheumatism 52:S135, 2005
Are patient questionnaires more
“scientific” than lab test and x-rays in RA?



Physical function scores on the HAQ or MDHAQ,
not a joint count, lab test or X-ray, is far and away
the most significant predictor in RA of functional
status, work disability, costs, joint replacement
surgery, and death
A physician may treat fever without a
temperature, tachycardia without a pulse, diabetic
coma without a glucose, but why
Treating RA with primary attention to laboratory
tests and radiographs without a physical function
score may be analogous to treating hypertension
according to heart sounds or renal failure
according to a urinalysis – it’s relevant but a not
the most effective prognostic marker
Focus on function – a modern patient centered approach in rheumatology
-
-
-
Rheumatology measures for clinical
research versus standard care
Why focus on patient-reported outcome
(PRO) measures?
Routine Assessment of Patient Index Data
(RAPID) scores on an MDHAQ to quantify RA
severity without formal joint counts
Remodeling the biomedical model to include
a biopsychosocial model
Is it better to have 80% of the
information in 100% of
patients or 100% of the
information in 5% of patients?
T Pincus, F Wolfe
J Rheumatol 32:575-577, 2005.
Some limitations of patient self-report
questionnaires
1. Need for translation –language issues
2. Cultural and linguistic issues
3. Possibility of “gaming” by patient, health
professional to provide desired responses
4. Not specific to any disease
Focus on function – a modern patient centered approach in rheumatology
-
-
-
Rheumatology measures for clinical
research versus standard care
Why focus on patient-reported outcome
(PRO) measures?
Routine Assessment of Patient Index Data
(RAPID) scores on an MDHAQ to quantify RA
severity without formal joint counts
Remodeling the biomedical model to include
a biopsychosocial model
The need for a new medical model:
a challenge for biomedicine
“Medicine’s unrest derives from a
growing awareness among many
physicians of the contradiction between
the excellence of their biomedical
background on the one hand and the
weakness of their qualifications in certain
attributes essential for good patient care
on the other.”
- George L. Engel
Science 196:134, 1977
Some Assumptions of a
Biomedical Model
 Reductionism – single cause,
single cure for each disease
 Mind-body dualism – “mental” vs
“somatic" as separate entities
 Diagnosis based mostly on “tests”
 High tech lab, X-ray data superior
to patient data to assess & predict
 Outcomes depend more on MDs,
drugs, than on patients
Editorial: Challenges to the
biomedical model: are actions of
patients almost always as
important as actions of health
professionals in long-term
outcomes of chronic diseases?
T Pincus
Advances in Mind-Body Medicine
16:276-294, 2000
9-10 Year Survival According to
Quantitative Markers in Three Chronic
Diseases
Rheumatoid Arthritis Activities of Daily Living
A
Rheumatoid Arthritis -
B
>90%
81–90%
80
% Active “With Ease”
60
40
71–80%
Survival (%)
Survival (%)
100
>12 Years
80
9–12 Years
60
8 Years
40
20
70%
20
Formal Education Level
100
(Data from Pincus et al, 1987)
(Data from Pincus et al, 1987)
20
60
80
100
100
Stage I
80
Stage II
Stage III All Stages,
All Causes
Stage IV
60
40
0
Months
Hodgkin’s Disease Anatomic Stage
C
Survival (%)
40
20
D
0
2
4
6
8
10
Years
40
60
80
100
Months
Coronary Artery Disease # of Involved Vessels
80
60
40
20
(Data from Kaplan, 1972)
20
100
Survival (%)
0
1 Artery
2 Arteries
3 Arteries
LCA
(Data from Proudfit et al, 1978)
0
2
4
6
8
10 Years
Survival in rheumatoid arthritis 1973-1982
Pincus et al. Arthritis Rheum. 1984;27:864. J Rheumatol 1987;14:240
Severe functional declines,
work disability, and increased
mortality in seventy-five
rheumatoid arthritis patients
studied over nine years
T Pincus, LF Callahan, WG Sale,
AL Brooks, LE Payne, WK Vaughn
Arthritis Rheum 27:864-872, 1984
Rheumatoid Arthritis over 9 years – changes
in functional status in activities of daily
living and morning stiffness 1973-1982
Activities of daily living
1973
100
1982
Morning Stiffness
1973
0
90
30
80
60
70
90
60
120
50
150
40
180
30
210
20
240
10
270
0
300
% No Difficulty
1982
Minutes
Pincus et al. Arthritis Rheum. 1984;27:864; J Rheumatol. 1992;19:1051
Taking mortality in
rheumatoid arthritis
seriously – predictive
markers, socioeconomic
status and comorbidity
T Pincus , LF Callahan
J Rheumatology 13:841-845 1986
Formal education (socioeconomic
status) and health –
Possible
explanations
Limited education
Possible solutions
More education
Limited Resources Money, Medicaid
Limited access to
“health care”
Psychosocioeconomic problems
Increase access to
medical services
Change how
“system” works
16
14
12
10
8
6
4
2
0
Year
98
19
94
19
90
19
86
19
82
19
78
19
74
19
70
19
19
66
Health
Education
Defense
62
19
Percentage of GDP
U.S. Expenditures as a Percentage of
GDP, 1962-1998
“A Biopsychosocial Model”
GL Engel, Science 196:129, 1977
• “Non -reductionism” or “wholism” - multiple
causes, approaches to “control” vs “cure”treat whole patient - not just “broken part”
• Mind-body connections in all aspects of care
• Patient data more “scientific” than lab, X-ray
• Outcomes depend as much on patients as on
professionals
• Medical care system limited to improve health
Does this model provide an extraordinary
opportunity for leadership by rheumatologists,
rather than trying to fit a “biomedical model” ?
Do We Need Two
Complementary Models?
• Bio-medical
model in acute
diseases, and in
acute events
within chronic
diseases for
short term care
• Bio-psychosocial model
in chronic
diseases, to
address long
term patient care
needs and
outcomes
Rudolph Virchow
•“Omnis cellula ex
cellula
•Founder of cellular
pathology
•Named:
• leukocyte,
• leukemia,
• pulmonary
embolism,
•amyloid,
•trichinosis
“The improvement of medicine
would eventually prolong human
life, but improvement of social
conditions could achieve this
result now more rapidly and
more successfully.”
Rudolf Virchow 1848
“Medicine is a social science, and
politics nothing else but medicine
on a large scale.”
“The physicians are the natural
attorneys of the poor, and the
social problems should largely be
solved by them.”
Rudolf Virchow, 1848
The Sickness Unto Death
A Christian Psychological Exposition for
Upbuilding and Awakening
Soren Kierkegaard, 1849
This concept, the sickness unto death,
must, however, be understood in a
particular way… we use the expression
“fatal sickness” as synonymous with the
sickness unto death.
The Varieties of Religious Experience
William James,1902,“The Sick Soul”
“antagonism may naturally arise
between the healthy-minded way of
viewing life and the way that takes all
this experience of evil as something
essential...Evil is a disease; and worry
over disease is itself an additional form
of disease…to the healthy-minded way,
on the other hand, the way of the sick
soul seems diseased.”
Johns Hopkins (1795-1873)
The indigent sick of this city and its
environs, without regard to sex, age or
color, who may require surgical or
medical treatment, and the poor of this
city and State, of all races, who are
stricken down by any casualty, shall be
received into the Hospital, without
charge, for such periods of time and
under such regulations as you may
prescribe.
Letter to the first Trustees of the
Johns Hopkins Hospital, March 1873
Focus on function – a modern patient centered approach in rheumatology
-
-
-
Measures in clinical rheumatology
Why focus on patient-reported outcome
(PRO) measures?
A continuous quality improvement strategy
using patient questionnaires in the
infrastructure of patient care
Remodeling the biomedical model to include
a biopsychosocial model
“The proper study of mankind is
man….”
Alexander Pope, 1733
“The proper study of mankind is
man….”
Alexander Pope, 1733
….and woman
Theodore Pincus, 2006
[email protected]
Thank you for your
attention and interest!
Tack so mycket
Multidimensional Health Assessment Questionnaire
AT THIS MOMENT, are you able to:
Dress yourself, including tying shoelaces and
doing buttons?
Get in and out of bed?
Lift a full cup or glass to your mouth?
Walk outdoors on flat ground?
Wash and dry your entire body?
Bend down to pick up clothing from the floor?
Turn regular faucets on and off?
Get in and out of a car, bus, train or airplane?
Walk 2 miles or 3 kilometers?
Participate in sports and games as you would like?
Get a good night’s sleep?
Deal with feelings of anxiety or being nervous?
Deal with feelings of depression or feeling blue?
Without ANY
Difficulty
With SOME
Difficulty
With MUCH
Difficult
UNABLE
To Do
----------------------------------------
----------------------------------------
----------------------------------------
----------------------------------------
How much pain have you had because of your condition IN THE PAST WEEK?
Place a mark on the line below to indicate how severe your pain has been:
NO PAIN
                    
0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10
PAIN AS BAD AS
IT COULD BE
Considering all the ways in which your illness and and health conditions
may affect you at this time, place a mark to show how you are doing:
VERY
WELL
                    
0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10
VERY
POORLY
A Multinational Cross-Sectional
Database To Assess Clinical
Status Of Patients With
Rheumatoid Arthritis (QUESTRA)
Sokka, Mäkinen, Hetland,
Verstappen, Toloza, Herborn,
Naranjo, Gossoc, Bresnihan,
Cazzato, Baecklund,
Sierakowski, Tunc, Skakic,
Pincus
for the Quest-RA Group
An international effort:
• To enroll 100 consecutive
patients with RA
• >10 countries
• >3 sites/country
• to establish a collaborative cross
sectional study of RA
Primary Objective
• To provide experience with
measurement tools that can be
used in routine clinical care, to
improve patient care
Methods of data collection
• 100 consecutive patients with RA in
each clinic to complete 4-page a
patient questionnaire
• The patients to be assessed
according to a standard protocol
to evaluate RA:
– a review of clinical features
– comorbidities
– all disease-modifying antirheumatic drugs (DMARDs) used
– joint count
• The patients completed an
expanded self-report health
assessment questionnaire (HAQ)
with
• visual analog scales (VAS) for pain,
global status, and fatigue
• a self-report joint count on RADAI
• life-style choices such as smoking
and physical exercise
• work status
• Data collection started in January
2005
Study status 14-August-2006
# sites
# patients in
the database
Denmark
3
301
Finland
3
304
France
4
389
Germany
3
226
Ireland
3
225
Italy
4
336
Netherlands
3
317
Poland
7
638
Spain
3
301
Sweden
3
244
UK
3
114
Turkey
3
300
Serbia
1
100
USA
3
295
Argentina
2
246
Total = 15
48
4336
Country
New Quest-RA Countries collecting data:
Estonia
Greece
Hungary
Latvia
Lithuania
Macedonia
New countries interested; ACR 2006
• Russia
• Australia
• Canada
• Latin America
Patients;
Demographic variables
Demographic Variables
Age, current, years
Age at 1st symptoms, years
Female
Education, years
Mean (SD) or
percentage
57.0 (13.8)
45.4 (15.0)
78.0%
10.6 (3.9)
Caucasian
88.0%
Currently working full time
20.7%
Patients;
Disease characteristics
Disease Characteristics
Mean (SD) or
percentage
Disease duration, years
from 1st symptoms
11.5 (10.0)
RF positive, ever
72.5%
Erosions, ever
59.4%
Extra-articular disease*
24.5%
Time to diagnosis, months
from 1st symptoms
22.0 (0.8)
*nodules/pulm fibrosis/pericarditis/Felty/vasculitis/scleritis
Patients;
Clinical measures
Swollen Joint count 28
Tender Joint count 28
DAS28 0-10
MD global VAS 0-10
Mean (SD)
4.2 (5.4)
6.0 (7.2)
4.1 (1.7)
2.8 (2.4)
Questionnaire measures
HAQ 0-3
Pain VAS 0-10
1.0 (0.8)
4.1 (2.7)
Clinical measures
Morning stiffness, minutes
Laboratory
ESR
51.3 (69.2)
27.7 (23.4)
Comparison of clinical measures in
Western Europe vs. Other Countries
N
SJC28
TJC28
MDGlobal VAS
ESR
DAS28
HAQ
PAIN VAS
Fatigue VAS
Morning stiffness
W.Europe and USA
2964
Other Countries
1194
3.3
4.4
2.3
6.5
9.9
4.0
24.2
3.7
0.9
3.7
36.0
5.1
1.3
5.0
4.0
45.4
5.1
64.9
Mean values shown; Student’s t-test p<0.001 for all comparisons
The first DMARD in 1970’s to 2000’s in Europe;
The raise of methotrexate
70
60
IM Gold
MTX
% of patients
50
40
30
20
10
SSZ
Traditional
DMARDs
HCQ
0
1970
1980
1990
2000
Next slide will show
• Selected DMARDs ever used by >4000
RA patients from the 15 countires
• In each column
• yellow indicates the highest
• blue the lowest
percentage among the countries
DMARD ever:
Pred
MTX
HCQ
SSZ
LEF
Any Biol
Denmark
43%
86%
39%
64%
11%
23%
Finland
74%
85%
74%
84%
21%
17%
France
83%
87%
55%
49%
42%
53%
Germany
54%
80%
30%
36%
25%
29%
Ireland
71%
92%
15%
33%
24%
41%
Italy
69%
78%
42%
14%
31%
26%
Netherlands
26%
81%
28%
35%
6%
19%
Poland
69%
79%
34%
60%
18%
8%
Spain
67%
85%
43%
29%
34%
27%
Sweden
66%
81%
34%
62%
9%
31%
UK
51%
75%
39%
46%
4%
16%
Turkey
69%
81%
27%
61%
22%
7%
Serbia
88%
69%
55%
17%
7%
2%
USA
77%
85%
49%
12%
19%
33%
Argentina
83%
68%
49%
6%
16%
3%
Total; n=4157
66%
81%
41%
43%
21%
23%
DMARDs: Discussion
• Large differences are seen in the use
of biologics and other DMARDs among
countries.
• Reasons for this observation may
include:
– patients who participated in RCTs were
not excluded
– local and national traditions to use
DMARDs
– differences in national guidelines to
use/limit biologics
– genetic/behavioral factors of patient
populations
– marketing
DOES POOR FUNCTIONAL CAPACITY
PREVENT PATIENTS WITH RHEUMATOD
ARTHRITIS FROM PHYSICAL EXERCISES?
Finland
Netherlands
Ireland
Sweden
Germany
Serbia
Denmark
UK
USA
Spain
Poland
France
Turkey
Italy
HAQ <1
HAQ 1-3
Argentina
0
10
20
30
40
50
60
70
80
90
Exercise once weekly or more, percentage of patients
100
Physical Exercises:
Discussion
• A low proportion of RA patients exercise in many
countries.
• However, poor functional status does not
necessarily prevent RA patients from physical
exercises.
• Importance of regular physical exercises should be
emphasized in RA patients
– rather than neglected or even forbidden
- its effects on fitness, metabolic status,
and longevity need more attention in
patients with RA.
• These data may serve as a basis for health
educators to improve patterns of exercise habits in
patients with RA in different countries.
Clinical status of RA in relation
to macro economic variables in
15 countries
Next slide:
Relationship between Gross Domestic
Product (GDP), expressed as parity
purchasing power, and the overall
clinical status on the Mean Outcome
Index for Rheumatoid Arthritis (MOIRA) in 15 QUEST-RA countries. The
area of the disc reflects the amount
of the total national health
expenditure (TNEH) per capita in
each country.
60
50
MOI-RA
40
30
20
10
r = -0.73
0
0
5
10
15
20
25
30
35
40
45
50
Gross Domestic Product per capita, 1000$
Discussion; macro economic
variables and RA disease activity
• Macro-economic variables that characterize
a nation are important predictors of health
outcomes.
• Gross domestic product (GDP) is a predictor
of overall mortality, infant mortality, and life
expectancy.
• Concerning outcomes of specific diseases, a
5-years survival of cancer was associated
with GDP in an analysis of data from 22
European countries.
• Macro-economic variables appear important
also concerning RA clinical disease activity.
Conclusions
• This international multi-center cross sectional
database will provide a general overview of
clinical status and treatments of patients
with RA in standard clinical care in 2005-06
although data may not be representative for all
included countries due to few sites.
• The QUEST-RA program should further enhance
introduction of quantitative assessment into
standard care of patients with rheumatic
diseases, including those who are not treated
with biological agents and not included in
databases that involve patient selection.
Thanks to the Quest-RA Group:
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
Denmark: K Hørslev-Petersen, M Hetland, TM Hansen;
Finland: H Makinen, K Immonen, S Forsberg, J Lähteenmäki, R
Luukkainen;
France: M Dougados, L Gossec, JF Maillefert, B Combe, J Sibilia;
Germany: R Rau, G Herborn, R Alten, C Pohl, G Burmester;
Ireland: B Bresnihan, P Minnock, E Murphy, C Sheehy, J Devlin, S Alraqi;
Italy: M Cutolo, M Cazzato, GF Ferraccioli, F Salaffi, A Stancati;
The Netherlands: S Verstappen, M Huisman, M Hoekstra;
Poland: S Sierakowski, S Sadkiewicz, M Majdan, D Zarowny-Wierzbinska,
W Romanowski, D Kapolka, W Tlustochowicz;
Spain: M Belmonte, J Calvo-Alen, A Naranjo;
Sweden: E Baecklund, AC Holmqvist, R Oding;
UK: P Taylor, C McClinton, K Dolan, E Choy, S Kelly, A Woolf, G Chorghade;
Turkey: F Gogus, S Celik, R Tunc;
Serbia: V Skakic, A Dimic, J Nedovic, A Stankovic;
USA: T Pincus, M Bergman, Y Yazici;
Argentina: S Toloza
Abbott
DATABASE SPECIALIST: Christopher Swearingen
DATA ENTRY: Melissa Gibson, Gina Sung, Kalevi Koskinen, Joni
Saalamo
Ted Pincus
An index of the three core data set patient
questionnaire measures distinguishes efficacy
of active treatment from the of placebo as
effectively as the American College of
Rheumatology 20% response criteria (ACR20) or
the disease activity score (DAS) in a rheumatoid
arthritis clinical trial.
T Pincus, V Strand, G Koch, I Amara, B Crawford,
F Wolfe, S Cohen, D Felson
Arthritis Rheum 48:625-630, 2003
A proposed continuous quality
improvement program to improve care
of patients with RA without formal
joint counts based on MDHAQ indices
1. MDHAQ for feasibility
2. Easy scoring
3. Flow sheets–lab and drugs
4. Index or indices
5. Categories of severity
6. Continuous quality improvement
for treatment decisions
Focus on function – a modern patient centered approach in rheumatology
-
Rheumatology measures for clinical
research versus standard care
Why focus on patient-reported outcome
(PRO) measures?
A RAPID index for RA severity based on
MDHAQ, without joint counts
Remodeling the biomedical model to include
a biopsychosocial model
Median number of seconds to score various RA measures
120
100
Seconds
80
60
40
20
0
Rheum #1
Rheum #2
Rheum #3
Mean
28 JT
CT
DAS28
84
113
71
90
12.9
16.8
14.6
14.6
HAQ MDHAQ
RAPID
FN +
+ PN, RAPID2 RAPID 3
4MD
PN, GL
GL
41.5
42.2
41.9
6.4
8.5
7.5
7.5
4.3
4.4
4
4.3
9.2
12.1
9.1
9.6
11.8
16.1
12
12.2
RAPID
RAPID 5
4JC
19
22.8
15.3
19
19.4
27.3
17.5
19.4
Continuous quality improvement
based on MDHAQ indices
1. MDHAQ for feasibility
2. Easy scoring
3. Flowsheets–lab and drugs
4. Index or indices
5. Categories of severity
6. Continuous quality improvement
for treatment decisions
How much pain have you had because of your condition OVER THE PAST WEEK?
Please indicate below how severe your pain has been:
1.
NO
PAIN AS BAD AS
IT COULD BE
PAIN
2.
NO
PAIN
                   
PAIN AS BAD AS
IT COULD BE
3.
NO
PAIN
                    
0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10
PAIN AS BAD AS
IT COULD BE
4.
NO
PAIN
                    
P
PAIN AS BAD AS
IT COULD BE
5.
NO
PAIN
6.
NO
PAIN
7.
NO
PAIN
8.
9.
10.
  
0
  
  
2
  
◊
      
4
  
◊
6
   
8
◊
10
      
                 
0 .15 .30 .45 .61.76 .91 1.1 1.2 1.4 1.5 1.6 1.8 1.9 2.1 2.3 2.4 2.6 2.7 2.9 3.0
VERY
WELL
                  
NO
PAIN

VERY
WELL

0 0.2 0.3 0.5 0.6 0.8 0.9 1.1 1.2 1.4 1.5 1.6 1.8 1.9 2.1 2.3 2.4 2.6 2.7 2.9 3.0
0
0
1
1
2
2
3
3
4
4
5
5
6
6
7
7
8
8
9
9
10
10
PAIN AS BAD AS
IT COULD BE
PAIN AS BAD AS
IT COULD BE
PAIN AS BAD AS
IT COULD BE
VERY
POORLY
PAIN AS BAD AS
IT COULD BE
VERY
POORLY
Types of measures to assess rheumatoid arthritis:
1.
2.
3.
4.
Joint count
a. Swelling, tenderness or pain on motion
b. Limited motion or deformity
Radiographs scores
a. Erosion
b. Joint space narrowing
Laboratory tests
a. Erythrocyte Sedimentation Rate (ESR)
b. C-Reactive Protein (CRP)
Patient questionnaire
a. Physical function
b. Pain
c. Psychological distress d. Fatigue
5.
Global measures
a. Physician/assessor
b. Patient
A Routine Assessment of Patient Data
(RAPID) score based on the MDHAQ
indices
1. MDHAQ for feasibility
2. Easy scoring
3. Flowsheets–lab and drugs
4. Index or indices
5. Categories of severity
6. Continuous quality improvement
for treatment decisions
Multi-Dimensional Health Assessment Questionnaire (R771-NP2)
This questionnaire includes information not available from blood tests, X-rays, or any source other
than you. Please try to answer each question, even if you do not think it is related to you at this time.
Try to complete as much as you can yourself, but if you need help, please ask. There are no right or
FOR OFFICE
wrong answers. Please answer exactly as you think or feel. Thank you.
USE ONLY
MDHAQ
Page 1
1. Please check (√) the ONE best answer for your abilities at this time:
Without
With
With
OVER THE LAST WEEK, were you able to:
ANY
SOME
MUCH
Difficulty
Difficulty
Difficulty
a. Dress yourself, including tying shoelaces and
doing buttons?
_____0
_____1
_____2
b. Get in and out of bed?
_____0
_____1
_____2
c. Lift a full cup or glass to your mouth?
_____0
_____1
_____2
d. Walk outdoors on flat ground?
_____0
_____1
_____2
e. Wash and dry your entire body?
_____0
_____1
_____2
f. Bend down to pick up clothing from the floor?
_____0
_____1
_____2
g. Turn regular faucets on and off?
_____0
_____1
_____2
h. Get in and out of a car, bus, train, or airplane?
_____0
_____1
_____2
i. Walk two miles or three kilometers, if you wish?
_____0
_____1
_____2
j. Participate in recreational activities and sports
_____0
_____1
_____2
as you would like, if you wish?
k. Get a good night’s sleep?
_____0
_____1.1 _____2.2
l. Deal with feelings of anxiety or being nervous?
_____0
_____1.1 _____2.2
m.Deal with feelings of depression or feeling blue?
_____0
_____1.1 _____2.2
UNABLE
To Do
_____3
_____3
_____3
_____3
_____3
_____3
_____3
_____3
_____3
_____3.3
_____3.3
_____3.3
NO                      PAIN AS BAD AS
PAIN 0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10 IT COULD BE
3. Please place a check (√) in the appropriate spot to indicate the amount of pain you
are having today in each of the joint areas listed below:
None
Mild Moderate Severe
None
Mild Moderate Severe
i.RIGHT FINGERS
j.RIGHT WRIST
k.RIGHT ELBOW
l.RIGHT SHOULDER
m.RIGHT HIP
n.RIGHT KNEE
o.RIGHT ANKLE
p.RIGHT TOES
q.NECK
r.BACK
2
4. Considering all the ways in which illness and health conditions may affect you at this
time, please indicate below how you are doing:
VERY                     
WELL 0 0.5 1.0 1.5 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10
For Office Use Only: RAPID 3
Please turn to the other side
RAPID 3 (0-10)
RAPID 4
VERY
POORLY
16=5.3
17=5.7
18=6.0
19=6.3
20=6.7
21=7.0
22=7.3
23=7.7
24=8.0
25=8.3
26=8.7
27=9.0
28=9.3
29=9.7
30=10
2.PN (0-10)
4.PTGL (0-10)
RAPID 3 (0-30)
3.a-pPTJT(0-10)
1=0.2
25=5.2
2=0.4
26=5.4
3=0.6
27=5.6
4=0.8
28=5.8
5=1.0
29=6.0
6=1.3
30=6.3
7=1.5
31=6.4
8=1.7
32=6.7
9=1.9
33=6.9
10=2.1 34=7.1
11=2.3 35=7.3
12=2.5 36=7.5
13=2.7 37=7.7
14=2.9 38=7.9
15=3.1 39=8.1
16=3.3 40=8.3
17=3.5 41=8.5
18=3.8 42=8.8
19=4.0 43=9.0
20=4.2 44=9.2
21=4.4 45=9.4
22=4.6 46=9.6
23=4.8 47=9.8
24=5.0 48=10
?
?
?
?
RAPID 4 (0-40)
RAPID 4 (0-10)
NR: 1=0.3, 2=0.7, 3=1.0 LS: 4=1.3, 5=1.7, 6=2.0
NR: 1=0.3, 2=0.5, 3=0.8, 4=1.0
LS: 5=1.3, 6=1.5, 7=1.8, 8=2.0
MS: 7=2.3, 8=2.7, 9=3.0, 10=3.3, 11=3.7, 12=4.0
MS: 9=2.3, 10=2.5, 11=2.8, 12=3.0, 13=3.3, 14=3.5, 15=3.8, 16=4.0
HS: 13=4.3, 14=4.7, 15=5.0, 16=5.3, 17=5.7,18=6.0,
HS: 17=4.3, 18=4.5, 19=4.8, 20=5.0, 21=5.3, 22=5.5, 23=5.8, 24=6.0,
19=6.3, 20=6.7, 21=7.0, 22=7.3, 23=7.7, 24=8.0,
25=6.3, 26=6.5, 27=6.8, 28=7.0, 29=7.3, 30=7.5, 31=7.8, 32=8.0,
25=8.3, 26=8.7, 27=9.0, 28=9.3, 29=9.7, 30=10.0
33=8.3, 34=8.5, 35=8.7, 36=9.0, 37=9.3, 38=9.5, 39=9.8, 40=10.0
NR: 1=0.2, 2=0.4, 3=0.6, 4=0.8 5=1.0 LS: 6=1.2, 7=1.4, 8=1.6, 9=1.8, 10=2.0,
RAPID 5
MS:11=2.2, 12=2.4, 13=2.6, 14=2.8, 15=3.0, 16=3.2, 17=3.4, 18=3.6, 19=3.8, 20=4.0
(0-10)
HS: 21=4.2, 22=4.4, 23=4.6, 24=4.8, 25=5.0, 26=5.2, 27=5.4, 28=5.6, 29=5.8, 30=6.0, 31=6.2, 32=6.4, 33=6.6, 34=6.8, 35=7.0,
36=7.2, 37=7.4, 38=7.6, 39=7.8, 40=8.0, 41=8.2, 42=8.4, 43=8.6, 44=8.8, 45=9.0, 46=9.2, 47=9.4, 48=9.6, 49=9.8, 50=10.0
Copyright: Health Report Services, Telephone 615-936-2151, E-mail [email protected]
1=0.3
2=0.7
3=1.0
4=1.3
5=1.7
6=2.0
7=2.3
8=2.7
9=3.0
10=3.3
11=3.7
12=4.0
13=4.3
14=4.7
15=5.0
_____3
2. How much pain have you had because of your condition OVER THE PAST WEEK? Please
indicate below how severe your pain has been:
a.LEFT FINGERS
b.LEFT WRIST
c.LEFT ELBOW
d.LEFT SHOULDER
e.LEFT HIP
f.LEFT KNEE
g.LEFT ANKLE
h.LEFT TOES
1.a-j FN (0-10)
\ \\
MDGLOBAL(0-10))
RAPID 5 (0-50)
Continuous quality improvement
based on MDHAQ indices
1. MDHAQ for feasibility
2. Easy scoring
3. Flowsheets–lab and drugs
4. Index or indices
5. Categories of severity
6. Continuous quality improvement
for treatment decisions
Median number of seconds to…
Perform 28 joint count 90
Enter DAS at web site 14.6
Score standard HAQ
Score MDHAQ
Score RAPID 2
Score RAPID 3
Score RAPID 4MDGL
Score RAPID 4PTJC
Score RAPID 5
41.9
7.5
4.3
9.6
12.2
19.0
19.4
Continuous quality improvement
based on MDHAQ indices
1. MDHAQ for feasibility
2. Easy scoring
3. Flowsheets–lab and drugs
4. Index or indices
5. Categories of severity
6. Continuous quality improvement
for treatment decisions
Patient Self-Report Questionnaire Scores in
the Assessment of RA
1. Significant correlation with joint counts, ESR,
X-ray scores, physical measures
2. More reproducible than traditional joint
counts, ESR, X-ray scores
3. As informative as the ACR-20, -50, -70, or
DAS in clinical trials
4. Predicts work disability, costs, joint
replacement, and premature death better than
traditional joint counts, radiographs, and
laboratory tests
DAS = Disease Activity Score.
mHAQ: Correlation With Various Measures of
Clinical Status in 259 Patients With RA
Variable*
Correlation Coefficient
Joint count score
0.60
Radiographic score
0.31
ESR
0.24
Grip strength
–0.53
Walk time
0.44
ARA class
0.60
Patient global
0.74
Age
0.23
Duration of disease
0.28
Formal education level
–0.24
*P < 0.001 for all variables versus mean scores for 8 activities of daily living on the mHAQ.
ARA = American Rheumatism Association; mHAQ = Modified Health Assessment Questionnaire.
Pincus T, et al. Ann Intern Med. 1989;110:259–266.
Patient Self-Report Questionnaire Scores in
the Assessment of RA
1. Significant correlation with joint counts, ESR,
X-ray scores, physical measures
2. More reproducible than traditional joint
counts, X-ray scores, ESR
3. As informative as the ACR-20, -50, -70, or
DAS in clinical trials
4. Predicts work disability, costs, joint
replacement and premature death better than
traditional joint counts, radiographs, and
laboratory tests
Patient Self-Report Questionnaire Scores in
the Assessment of RA
1. Significant correlation with joint counts, ESR,
X-ray scores, physical measures
2. More reproducible than traditional joint
counts, ESR, X-ray scores
3. As informative as the ACR-20, -50, -70, or
DAS in clinical trials
4. Predicts work disability, costs, joint
replacement and premature death better than
traditional joint counts, radiographs, and
laboratory tests
Patient Self-Report Questionnaire Scores in
the Assessment of RA
1. Significant correlation with joint counts, ESR,
X-ray scores, physical measures
2. More reproducible and less likely to improve
with placebo than traditional joint counts,
ESR, X-ray scores, physical measures
3. As informative as the ACR-20, -50, -70, or
DAS in clinical trials
4. Predicts work disability, costs, joint
replacement and premature death better than
traditional joint counts, radiographs, and
laboratory tests
Attributed Causes of Death in 2,262 RA
Patients in 13 Series from Diverse Locales
Compared to General Population
Attributed Cause of Death
Deaths
Cardiovascular disease
Cancer
Infection
Renal disease
Pulmonary disease
RA
GI disease
CNS disease
Accidents
Miscellaneous
Unknown
% of RA Deaths
% of 1977 US
42.1
14.1
9.4
7.8
7.2
5.3
4.2
4.2
1.0
6.4
0.6
41.0
20.4
1.0
1.1
3.9
--2.4
9.6
5.4
15.2
---
Pincus T, Callahan LF. J Rheumatol. 1986;13:841.
Atherosclerosis--an
inflammatory disease
R Ross
N Engl J Med 1999; 340(2):115-26.
Quantitative Monitoring of RA
Over 720 Days: MHAQ, Pain
10
Visual Analog 5
Pain Scale 0
2
MHAQ 1
Difficulty Score 0
Salsalate
Zero Order Aspirin
Fenoprofen
Piroxicam
Methotrexate
Auranofin
Injectable Gold
Prednisone
9/87
1/88
5/88
Pincus T. Arthritis Care Res. 1996;9:339.
9/88
1/89
5/89 8/89
Quantitative Monitoring of a Patient
With SLE over 180 days: ESR, anti-DNA,
CH50
Creat
Clear
(mL/min)
LE
Prep
CH50
100
140
50
90
0
40
-
+ +
-
-
-
-
200
100
% DNA
50 Bound
100
0
Prednison
e
(mg/day)
ESR
(mm/hr
)
0
50
0
20
40
60
80
100 120
140
160
180 Days
Pincus, Schur, Rose, Talal, Decker. New Engl J Med. 1969;281:701.
RADAI self-report Jt Count vs MD TJtC
RADAI
score
(0-48)
0-5
5-9
10-19
20-48
Total
MD tender joint count (0-28)
0-2
91
(88%)
39
(63%)
31
(48%)
12
(27%)
173
(63%)
3-5
8 (8%)
14
(23%)
16
(25%)
1 (2%)
39
(14%)
6-11
12+
3 (3%)
2 (2%)
6 (10%)
3 (5%)
14
(22%)
16
(36%)
39
(14%)
3 (5%)
15
(34%)
23
(8%)
Total
104
(38%)
62
(23%)
64
(23%)
44
(16%)
274
Criteria for clinical measure
• Clinical trials -
– Validity – does it measure what is
supposed to be measured?
– Reliability – is it reproducible?
• Clinical care – also consider
– Feasibility – can it be performed?
– Acceptability – will clinicians
assess it?
DAS VS RAPID IN ABATACEPT TRIALS--AIM
DAS28
RAPID2
RAPID3
RAPID4-MD RAPID4-JC
RAPID5
0%
-10%
Mean % Change
-20%
-21%
-25%
-30%
-28%
-27%
-30%
-32%
-40%
-43%
-47%
-50%
-54%
-56%
-60%
-61%
-70%
-52%
Control
Abatacept
5-Year Survival in 206 Patients
With RA: Cohort #2 – 19851990
Rheumatoid Factor
MHAQ Score
80
80
Survival (%)
100
Survival (%)
100
60
60
40
Absent (29)
Present
20
(175)
0.00 (12)
0.01–0.99 (91)
1.00–1.99 (86)
>2.00 (21)
40
20
0
0
0
12
24
36
48
Months After Baseline
Arthritis Care Res 10:381,1997
60
0
12
24
36
48
Months After Baseline
60
A HAQ or MDHAQ Patient Questionnaire not a
Lab test or X-ray – is Best Predictor in RA of…
 Functional status (Pincus et al. Arthritis Rheum. 1984,
Wolfe et al. J Rheumatol. 1991)
 Work disability (Borg et al. J Rheumatol 1991, Callahan
et al. J Clin Epidemiol. 1992, Wolfe and Hawley. J
Rheumatol. 1998, Fex et al. J Rheumatol 1998, Sokka et
al. J Rheumatol 1999, Barrett et al. Rheumatology 2000, )
 Costs (Lubeck et al. Arthritis Rheum. 1986)
 Joint replacement surgery (Wolfe and Zwillich. Arthritis
Rheum. 1998)
 Death (Pincus et al. Arthritis Rheum. 1984, Ann Intern
Med.1994, Wolfe et al. J Rheumatol 1988, Leigh&Fries J
Rheumatol 1991, Wolfe et al. Arthritis Rheum. 1994,
Callahan et al. Arthrits Care Res 1996, 1997, Soderlin et
al. J Rheumatol 1998, Maiden et al. Ann Rheum Dis 1999,
Sokka et al. Ann Rheum Dis 2004)
Fax Server
 Accepts faxed forms electronically
 8 Fax lines capability
 Converts faxes to electronic Teleform
data
 Verified, validated and converted to
SQL by same method as paper forms
Rheumatic Disease Data Banking
 Data Collection Methods






Scanning (Teleform)
Fax Server
Web entry
Interactive PDF
Telephone interview with patient
Telephone contact with physicians (rare)