Session Information
Session Type: Abstract Submissions (ACR)
Background/Purpose: Patient self-report has become prominent in the assessment of rheumatic and musculoskeletal diseases (RMD). In a workshop on priorities in PROs in 2009 it was proposed to generate a catalogue with the available instruments. The main objective of this project was to develop a structured design for this catalogue freely available online which would include a comprehensive database of validated instruments.
Methods: The first work package was aimed to develop the toolbox with the participation of 15 methodological collaborators to decide the toolbox´s aims and scope, and basic requirements. In a second work package, the aim was to feed the toolbox using systematic reviews of the literature by target disease with a common strategy to identify validation of PRO. The systematic literature review for systematic lupus erythematous (SLE) is described here as a methodological example. A search strategy was run in MEDLINE, EMBASE, and the Cochrane Library. Validation studies, cohort studies, reviews and meta-analyses were included. Selection criteria were: a) studies on adult patients with SLE defined by the ACR criteria, and b) validation studies of PRO. A reviewer screened title/abstracts and the relevant information was collected using a standardized data collection form based on the COSMIN checklist. The third work package was to provide educational support.
Results: Two hundred thirty six instruments have been identified of which 106 are generic and the remaining ones RMD specific. In particular for SLE, from 704 initial studies captured, we identified 18 articles meeting the predefined criteria. Studies addressed validation of 10 PRO, 1 to evaluate disease activity, 1 to evaluate lupus symptoms, 3 to evaluate quality of life, 2 to evaluate damage and 3 to evaluate patient’s needs, health and family functioning. Internal consistency was studied in all, with Cronbach’s α ranging from 0.71 to 0.96. Validity, examined by means of convergence with other instruments, was generally similar between tools. Responsiveness was tested in SLAQ, SLEQoL, with a standardized response mean (SRM) ranging from 0.12 to 0.44. Interpretability was only tested in SLE QoL, Lupus QoL and Lupus PRO with similar floor/ceiling effect (Table). Information about construct being measured, recommendations for use, translated versions and validation have been uploaded in the catalogue website. A list and definition for each validation aspect was included for educational support
Table: Summary of the results of the validity of the 10 SLE patient report outcomes
|
SLE PROs |
Domain |
# Items & Range |
Measurement Properties |
|||
|
ReliabilityCronbach |
Validity |
Responsiveness |
Interpretability |
|||
|
SLAQ (Systemic Lupus Activity Questionnaire) |
Disease Activity |
Items: 3 Range: 0-44 |
α = 0.87 |
+++ |
SRM: 0.12 |
— |
|
SSC (SLE Symptom Checklist questionnaire) |
Lupus Symptoms |
Items: 38 Range: 0-152 |
α = 0.89
|
+++ |
++ |
— |
|
SLEQOL (SLE-specific quality-of-life instrument) |
Quality of Life |
Items: 49 Range: 40-280 |
α = 0.95
|
+++ |
SRM: 0.44 Effect Size: 0.33 |
Floor:14.9% Ceiling:<2.6% |
|
LupusQoL (Lupus quality of life) |
Quality of Life |
Items: 34 Range: 0-100 |
α = 0.88-0.96 |
+++ |
— |
Floor:<10.8% Ceiling: 4-21% |
|
SLENQ (SLE needs questionnaire) |
Needs |
Items: 97 Range: – |
α = 0.77
|
+++ |
— |
— |
|
LDIQ (Lupus Damage Index Questionnaire) |
Damage |
Items: 56 Range: 0-22 |
α = 0.72 |
+++ |
— |
+ |
|
L-QoL (Lupus quality of life) |
Quality of Life |
Items: 25 Range: 0-22 |
α = 0.92
|
+++ |
— |
— |
|
BILD (Brief Index of Lupus Damage) |
Damage |
Items: 28 Range: 0-33 |
— |
+++ |
— |
— |
|
LupusPRO (Lupus Patient-Reported Outcome) |
Health outcome |
Items: 44 Range: 0-100 |
α = 0.72-0.94 |
+++ |
++ |
Floor:22.3% Ceiling:1.2%
|
|
SLE-FAMILY |
Family functioning |
Items: 6 Range: 1-7 |
α = 0.71
|
+++ |
— |
—
|
SRM: Standardized Response Mean
Conclusion: A freely accessible toolbox for PROs has been designed. This toolbox employs scientific measurement properties and provides PROs with advice on their application in a user-friendly manner. This toolbox is an on-going process led by rheumatologists, related professionals, and patients that should help to better understand and use PRO in RMD.
Disclosure:
I. Castrejón,
None;
L. Carmona,
None;
R. Christensen,
Abbott, Axellus A/S, Bristol-Myers Squibb, Cambridge Weight Plan, Norpharma, Pfizer, and Roche,
5,
Axellus A/S, Cambridge Weight Plan, Mundipharma, and Roche,
2,
Abbott, Axellus, Bayer HealthCare Pharmaceuticals, Biogen Idec, Bristol-Myers Squibb, Cambridge Weight Plan, Ipsen, Laboratoires Expanscience, MSD, Mundipharma, Norpharma, Pfizer, Roche, and Wyeth,
8;
T. Uhlig,
None;
B. Prodinger,
None;
F. Guillemin,
None;
M. Scholte-Voshaar,
None;
L. Gossec,
None.
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