Construct Validity and Responsiveness of EQ-5D-3L in Patients With Rheumatic Disease

NCT06568029 | Completed

• 1 other identifier: 2023-04394-01
• Study Type: observational
• Target: 77,651
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to investigate the construct validity (convergent and known-groups) and responsiveness of EQ-5D-3L in patients with rheumatoid arthritis, polyarthritis, psoriatic arthritis, and ankylosing spondylitis. The study is based on retrospective registry data from the Swedish Rheumatology Registry (SRQ).

Conditions

Rheumatoid Arthritis; Polyarthritis; Psoriatic Arthritis; Ankylosing Spondylitis; Rheumatic Diseases

Keywords

EQ-5D; Validity; Responsiveness; Psychometric; Patient-reported outcome; PROM; EQ VAS

Outcome Measures

Primary Outcomes (3)

• Convergent validity

  Convergent validity refers to how well the instrument under study (EQ-5D-3L and EQ VAS) correlates with other outcome measures (Fayers and Machin 2007). According to COSMIN guidelines, convergent validity should be assessed by formulating and testing hypotheses regarding the expected direction and magnitude of the correlation between the measurement being studied (EQ-5D-3L and EQ VAS) and other outcome measures measuring the same or similar constructs (Mokkink, Prinsen et al. 2019). The constructs measured by the other outcome measures should be clearly described (Mokkink, Prinsen et al. 2019). See hypotheses in document Study Protocol and Statistical Analysis Plan.

  One measurement per patient.The latest complete EQ-5D-3L and EQ VAS measurements per patient during the period 2008-2024 is used for the analysis.

• Known-groups validity

  Known-groups validity refers to how well the instrument can find differences between groups known to differ. Known-groups validity should be assessed by formulating and testing hypotheses regarding expected directions and magnitude of the differences between subgroups (Mokkink, Prinsen et al. 2019). See hypotheses in document Study Protocol and Statistical Analysis Plan.

  One measurement per patient. The latest complete EQ-5D-3L and EQ VAS measurements per patient during the period 2008-2024 is used for the analysis.

• Responsiveness

  Responsiveness refers to the ability of the instrument to capture change over time in the construct that is measured (Mokkink, Terwee et al. 2010). According to COSMIN guidelines, responsiveness can be assessed by comparing changes in EQ-5D-3L and EQ VAS, with changes in other outcome measures, similar to convergent validity explained above (Mokkink, Prinsen et al. 2019). The hypotheses should include the expected direction and magnitude of the correlations, and the constructs should be clearly described. Responsiveness can also be assessed by analysing weather EQ-5D-3L and EQ VAS can discriminate between patients who have improved and those that have not, based on changes in another outcome, such as changes in disease activity or functional ability (Mokkink, Prinsen et al. 2019). See hypotheses in document Study Protocol and Statistical Analysis Plan.

  One year. The two first EQ-5D-3L and EQ VAS measurements of patients with newly diagnosed disease (having the diagnosis for ≤12 months) during the first year for patients included during the period 2008-2024

Study Arms (4)

Patient with rheumatoid arthritis

18 years and older at the time of first included measurement. A diagnosis of RA. Complete registration of responses in the EQ-5D-3L descriptive system or EQ VAS on at least one time point in SRQ. At least one measurement with Disease Activity Score 28 (DAS28) reported in relation to the same visit as EQ-5D-3L.

Other: Questionnaire

Patient with polyarthritis

18 years and older at the time of first included measurement. A diagnosis of polyarthritis. Complete registration of responses in the EQ-5D-3L descriptive system or EQ VAS on at least one time point in SRQ. At least one measurement with DAS28 reported in relation to the same visit as EQ-5D-3L.

Other: Questionnaire

Patients with psoriatic arthritis

18 years and older at the time of first included measurement. A diagnosis of psoriatic arthritis. Complete registration of responses in the EQ-5D-3L descriptive system or EQ VAS on at least one time point in SRQ. At least one measurement with DAS28 or Disease Activity in Psoriatic Arthritis (DAPSA) reported in relation to the same visit as EQ-5D-3L.

Other: Questionnaire

Patient with ankylosing spondylitis

18 years and older at the time of first included measurement. A diagnosis of ankylosing spondylitis. Complete registration of responses in the EQ-5D-3L descriptive system or EQ VAS on at least one time point in SRQ. At least one measurement with the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) reported in relation to the same visit as EQ-5D-3L.

Other: Questionnaire

Interventions

Questionnaire OTHER

In the study, the validity and responsiveness of EQ-5D-3L will be assessed. The EQ-5D-3L measures HRQoL and consists of two parts. The first part contains five questions about mobility, daily activities, self-care, pain/discomfort, and anxiety/depression (EuroQoL 1990, Brooks 1996). Each question can be answered with no problem (1), some/moderate problem (2) unable to perform certain activities/having extreme problems (3). The answers can be summarized in an index value based on an existing preference-based value set. In this study, the EQ-5D-3L value set by Dolan (Dolan 1997) will be used for the main analyses and a Swedish experience-based value set in a sensitivity analysis (Burström, Sun et al. 2014). For the EQ-5D-3L index, 1 represents full health and 0 represents a value equal to being dead. EQ VAS measures the persons health today on a visual analogue scale (VAS) from 0 (worst imaginable health) to 100 (best imaginable health) (EuroQoL 1990, Brooks 1996).

Also known as: EQ-5D-3L

Patient with ankylosing spondylitis; Patient with polyarthritis; Patient with rheumatoid arthritis; Patients with psoriatic arthritis

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study is based on retrospective registry data from the Swedish Rheumatology Registry (SRQ) and includes patients with rheumatoid arthritis, polyarthritis, psoriatic arthritis, and ankylosing spondylitis. The analyses will be conducted independently for the different patient groups.

You may qualify if:

• years and older at the time of first included measurement
• A diagnosis of RA, polyarthritis, PsA, or AS
• Complete registration of responses in the EQ-5D-3L descriptive system or EQ VAS on at least one time point in SRQ
• For patients with RA: At least one measurement with Disease Activity Score 28 (DAS28) reported in relation to the same visit as EQ-5D-3L
• For patients with polyarthritis: At least one measurement with DAS28 reported in relation to the same visit as EQ-5D-3L
• For patients with PsA: At least one measurement with DAS28 or Disease Activity in Psoriatic Arthritis (DAPSA) reported in relation to the same visit as EQ-5D-3L
• For patients with AS: At least one measurement with the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) reported in relation to the same visit as EQ-5D-3L
• For the analyses of construct validity, the latest measurement will be used if the individual patients have multiple complete registrations with EQ-5D-3L and the other required measure. The hypotheses for responsiveness will be tested in patients with newly diagnosed disease (having the diagnosis for ≤12 months), as changes in disease activity are likely to be present in this group. For the analysis of responsiveness, the two first measurements during the first year will be used.

Sponsors & Collaborators

• Karolinska Institutet: lead

Study Sites (1)

It is an observational study based on the The Swedish Rheumatology Quality Register

Stockholm, Sweden

Location

Related Publications (26)

• Aletaha D, Smolen JS. The Simplified Disease Activity Index (SDAI) and Clinical Disease Activity Index (CDAI) to monitor patients in standard clinical care. Best Pract Res Clin Rheumatol. 2007 Aug;21(4):663-75. doi: 10.1016/j.berh.2007.02.004.

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• Boonen A, van der Heijde D, Landewe R, van Tubergen A, Mielants H, Dougados M, van der Linden S. How do the EQ-5D, SF-6D and the well-being rating scale compare in patients with ankylosing spondylitis? Ann Rheum Dis. 2007 Jun;66(6):771-7. doi: 10.1136/ard.2006.060384. Epub 2007 Jan 9.

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• Burstrom K, Sun S, Gerdtham UG, Henriksson M, Johannesson M, Levin LA, Zethraeus N. Swedish experience-based value sets for EQ-5D health states. Qual Life Res. 2014 Mar;23(2):431-42. doi: 10.1007/s11136-013-0496-4. Epub 2013 Aug 22.

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• Calin A, Nakache JP, Gueguen A, Zeidler H, Mielants H, Dougados M. Defining disease activity in ankylosing spondylitis: is a combination of variables (Bath Ankylosing Spondylitis Disease Activity Index) an appropriate instrument? Rheumatology (Oxford). 1999 Sep;38(9):878-82. doi: 10.1093/rheumatology/38.9.878.

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• Devlin NJ, Brooks R. EQ-5D and the EuroQol Group: Past, Present and Future. Appl Health Econ Health Policy. 2017 Apr;15(2):127-137. doi: 10.1007/s40258-017-0310-5.

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• Dolan P. Modeling valuations for EuroQol health states. Med Care. 1997 Nov;35(11):1095-108. doi: 10.1097/00005650-199711000-00002.

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• Ernstsson O, Janssen MF, Heintz E. Collection and use of EQ-5D for follow-up, decision-making, and quality improvement in health care - the case of the Swedish National Quality Registries. J Patient Rep Outcomes. 2020 Sep 16;4(1):78. doi: 10.1186/s41687-020-00231-8.

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• EuroQol Group. EuroQol--a new facility for the measurement of health-related quality of life. Health Policy. 1990 Dec;16(3):199-208. doi: 10.1016/0168-8510(90)90421-9.

  PMID: 10109801 BACKGROUND

• Fayers, P. M. and D. Machin (2007). Quality of life : the assessment, analysis and interpretation of patient-reported outcomes. Chichester, J. Wiley.

  BACKGROUND

• Fries JF, Spitz P, Kraines RG, Holman HR. Measurement of patient outcome in arthritis. Arthritis Rheum. 1980 Feb;23(2):137-45. doi: 10.1002/art.1780230202.

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• Fritz CO, Morris PE, Richler JJ. Effect size estimates: current use, calculations, and interpretation. J Exp Psychol Gen. 2012 Feb;141(1):2-18. doi: 10.1037/a0024338. Epub 2011 Aug 8.

  PMID: 21823805 BACKGROUND

• Lukas C, Landewe R, Sieper J, Dougados M, Davis J, Braun J, van der Linden S, van der Heijde D; Assessment of SpondyloArthritis international Society. Development of an ASAS-endorsed disease activity score (ASDAS) in patients with ankylosing spondylitis. Ann Rheum Dis. 2009 Jan;68(1):18-24. doi: 10.1136/ard.2008.094870. Epub 2008 Jul 14.

  PMID: 18625618 BACKGROUND

• Mokkink LB, de Vet HCW, Prinsen CAC, Patrick DL, Alonso J, Bouter LM, Terwee CB. COSMIN Risk of Bias checklist for systematic reviews of Patient-Reported Outcome Measures. Qual Life Res. 2018 May;27(5):1171-1179. doi: 10.1007/s11136-017-1765-4. Epub 2017 Dec 19.

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• Mokkink LB, Terwee CB, Patrick DL, Alonso J, Stratford PW, Knol DL, Bouter LM, de Vet HC. The COSMIN study reached international consensus on taxonomy, terminology, and definitions of measurement properties for health-related patient-reported outcomes. J Clin Epidemiol. 2010 Jul;63(7):737-45. doi: 10.1016/j.jclinepi.2010.02.006.

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• Nell-Duxneuner VP, Stamm TA, Machold KP, Pflugbeil S, Aletaha D, Smolen JS. Evaluation of the appropriateness of composite disease activity measures for assessment of psoriatic arthritis. Ann Rheum Dis. 2010 Mar;69(3):546-9. doi: 10.1136/ard.2009.117945. Epub 2009 Sep 17.

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• Prevoo ML, van 't Hof MA, Kuper HH, van Leeuwen MA, van de Putte LB, van Riel PL. Modified disease activity scores that include twenty-eight-joint counts. Development and validation in a prospective longitudinal study of patients with rheumatoid arthritis. Arthritis Rheum. 1995 Jan;38(1):44-8. doi: 10.1002/art.1780380107.

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• Prinsen CAC, Mokkink LB, Bouter LM, Alonso J, Patrick DL, de Vet HCW, Terwee CB. COSMIN guideline for systematic reviews of patient-reported outcome measures. Qual Life Res. 2018 May;27(5):1147-1157. doi: 10.1007/s11136-018-1798-3. Epub 2018 Feb 12.

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• Schoels M, Aletaha D, Funovits J, Kavanaugh A, Baker D, Smolen JS. Application of the DAREA/DAPSA score for assessment of disease activity in psoriatic arthritis. Ann Rheum Dis. 2010 Aug;69(8):1441-7. doi: 10.1136/ard.2009.122259. Epub 2010 Jun 4.

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• Terwee CB, Bot SD, de Boer MR, van der Windt DA, Knol DL, Dekker J, Bouter LM, de Vet HC. Quality criteria were proposed for measurement properties of health status questionnaires. J Clin Epidemiol. 2007 Jan;60(1):34-42. doi: 10.1016/j.jclinepi.2006.03.012. Epub 2006 Aug 24.

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• Terwee CB, Prinsen CAC, Chiarotto A, Westerman MJ, Patrick DL, Alonso J, Bouter LM, de Vet HCW, Mokkink LB. COSMIN methodology for evaluating the content validity of patient-reported outcome measures: a Delphi study. Qual Life Res. 2018 May;27(5):1159-1170. doi: 10.1007/s11136-018-1829-0. Epub 2018 Mar 17.

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Related Links

• National Ankylosing Spondylitis Society. (2004). "Ankylosing Spondylitis (Axial Spondyloarthritis) (AS) The Bath Indices."
• Lindqvist, U. (2022). "Axial spondylartrit (ankyloserande spondylit, pelvospondylit, Bechterews sjukdom)."
• Lindqvist, U. (2022). "Psoriasisartrit."
• Mokkink, L., C. Prinsen, D. Patrick, K. Alonso, L. Bouter, H. de Vet and C. Terwee. (2019). "COSMIN Study Design checklist for Patient-reported outcome measurement instruments."
• Svensk Reumatologis Kvalitetsregister (SRQ). "PER - Patientens Egen Registrering."
• Svensk Reumatologisk Förening. (2022). "Riktlinjer för läkemedelsbehandling vid axial spondylartrit och psoriasisartrit 2022"

MeSH Terms

Conditions

Arthritis, Rheumatoid; Arthritis; Arthritis, Psoriatic; Spondylitis, Ankylosing; Rheumatic Diseases

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Joint Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Spondylarthropathies; Spondylarthritis; Spondylitis; Spinal Diseases; Bone Diseases; Psoriasis; Skin Diseases, Papulosquamous; Skin Diseases; Axial Spondyloarthritis; Ankylosis

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Emelie Heintz, PhD

  Karolinska Institutet

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate professor

Study Record Dates

• First Submitted: August 19, 2024
• First Posted: August 23, 2024
• Study Start: August 16, 2024
• Primary Completion: September 30, 2024
• Study Completion: September 30, 2024
• Last Updated: March 6, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1)