Background
Patient-reported outcome measures (PROMs) are used to measure how patients themselves
experience their health and health related quality of life (HRQoL) (Sveriges kommuner och
regioner 2023). One of the most commonly used PROMs is EQ-5D, which is a generic
instrument used to measure, value and compare health across symptoms and diagnoses
(Devlin and Brooks 2017). When choosing a PROM to follow-up care from the patient
perspective and to assess HRQoL in patients with rheumatic disease, it is important to
know that the instrument is valid and responsive. An instrument with low validity and
responsiveness might not capture and describe patients' health and changes in their
health accurately. The aim of the study is therefore to investigate the validity and
responsiveness of EQ-5D-3L. The research questions are: 1. What is the construct validity
of EQ-5D-3L in patients with rheumatic disease? 2. What is the responsiveness of EQ-5D-3L
in patients with rheumatic disease?
Methodology
Study design and data collection
This study will assess the construct validity and responsiveness of EQ-5D-3L among
patients with rheumatoid arthritis (RA), polyarthritis, psoriatic arthritis (PsA), and
ankylosing spondylitis (AS) based on retrospective registry data. The design of the study
will follow the established guidelines from COSMIN on how to assess validity and
responsiveness of PROMs (Mokkink, Prinsen et al. 2019).
Construct validity refers to the degree to which an instrument measures the constructs it
intends to measure (Fayers and Machin 2007). Construct validity will be assessed in two
ways, convergent validity and known-groups 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). Known-groups validity refers to how well the
instrument can find differences between groups known to differ. Responsiveness refers to
the ability of the instrument to capture change over time in the construct that is
measured (Mokkink, Terwee et al. 2010).
Historical registry data from the Swedish Rheumatology Register (SRQ) will be used to
assess the construct validity and responsiveness of EQ-5D-3L and EQ VAS. Data on EQ-5D-3L
has been collected since 2008 (Ernstsson, Janssen et al. 2020) and the study will include
data from 2008 until the time of data extraction. The analyses will be conducted
independently for the different patient groups.
Ethical approval has been granted for the project (2023-04394-01).
Outcome measures
EQ-5D-3L and EQ VAS (see text about intervention). DAS28, DAPSA, BASDAI, ASDAS. The
Health Assessment Questionnaire Disability Index (HAQ-DI), the Bath Ankylosing
Spondylitis Functional Index (BASFI) VAS questions (pain, fatigue, and global score)
Convergent validity EQ-5D-3L
Convergent validity will be assessed by testing hypotheses regarding the expected
direction and magnitude of correlation between the EQ-5D-3L and the other outcome
measures (see hypotheses in document Study Protocol and Statistical Analysis Plan).
Constructs that are considered to be related are expected to have at least a moderate
correlation and constructs that are considered to be similar are expected to have a
strong correlation (Prinsen, Mokkink et al. 2018).
Known-groups validity EQ-5D-3L
In the assessment of known-groups validity, patients will be divided into groups for
which there is an expected difference in HRQoL (see hypotheses in document Study Protocol
and Statistical Analysis Plan). The groups represent patients with different levels of
disease activity or functional ability.
Responsiveness EQ-5D-3L
Responsiveness will be assessed in two ways. One way is by assessing the relationship
between individual changes in EQ-5D-3L index value and dimensions over time with changes
in other outcome measures over the same time period (see hypotheses in document Study
Protocol and Statistical Analysis Plan). The second way is to assess whether EQ-5D-3L can
discriminate between patients who have improved over time and those that have not, based
on changes in disease activity or functional ability. The relationship between changes in
the EQ-5D-3L and changes in other outcome measures will be assessed by analysing the
correlation between the changes in the variables between the two first measurements of
each individual during the first year after diagnosis (see hypotheses in document Study
Protocol and Statistical Analysis Plan).
To assess whether EQ-5D-3L can discriminate between patients who have improved and those
that have not, the area under the receiver operating curve ROC curve (AUC) will be
calculated. Patients will be considered to have improved based on results from the
measures of disease activity (DAS28, DAPSA, ASDAS or BASDAI) and functional ability
(BASFI and HAQ-DI) using criteria for what is defined as a response from previous studies
(see hypotheses in document Study Protocol and Statistical Analysis Plan).
Convergent validity EQ VAS
Convergent validity of EQ VAS will be assessed with the same analyses used for EQ-5D-3L
(see the section about convergent validity EQ-5D-3L and hypotheses in document Study
Protocol and Statistical Analysis Plan).
Known-groups validity EQ VAS
Known-groups validity of EQ VAS will be assessed for the same groups as the EQ-5D-3L
index (see section about Known-groups validity EQ-5D-3L and hypotheses in document Study
Protocol and Statistical Analysis Plan).
Responsiveness EQ VAS
Responsiveness of EQ VAS will be assessed with the same analyses used for EQ-5D-3L (see
the section about responsiveness EQ-5D-3L and hypotheses in document Study Protocol and
Statistical Analysis Plan).