Article Text
Abstract
Objective The heterogeneous multisystem manifestations of SLE include fatigue, pain, depression, sleep disturbance and cognitive dysfunction, and underscore the importance of a multidimensional approach when assessing health-related quality of life. The US Food and Drug Administration has emphasised the importance of patient-reported outcomes (PROs) for approval of new medications and Outcome Measures in Rheumatology has mandated demonstration of appropriate measurement properties of selected PRO instruments.
Methods Published information regarding psychometric properties of the Medical Outcomes Survey Short Form 36 (SF-36), Lupus Quality of Life Questionnaire (LupusQoL) and Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F), and their suitability as end points in randomised controlled trials (RCTs) and longitudinal observational studies (LOS) were assessed. A search of English-language literature using MEDLINE and EMBASE identified studies related to development and validation of these instruments. Evidence addressed content validity, reliability (internal consistency and test-retest reliability), construct validity (convergent and divergent) and longitudinal responsiveness, including thresholds of meaning and discrimination.
Results All instruments demonstrated strong internal consistency, reliability and appropriate face/content validity, indicating items within each instrument that measure the intended concept. SF-36 and LupusQoL demonstrated test-retest reliability; although not published with FACIT-F in SLE supported by evidence from other rheumatic diseases. All instruments demonstrated convergent validity with other comparable PROs and responsivity to treatment.
Conclusion The measurement properties of PRO instruments with published data from RCTs including: SF-36, LupusQoL and FACIT-F indicate their value as secondary end points to support labelling claims in RCTs and LOS evaluating the efficacy of SLE treatments.
- systemic lupus erythematosus
- outcomes research
- patient perspective
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Footnotes
Contributors The work reviewed in this manuscript was conducted under the auspices of the SLE working group of OMERACT (VS, LSS, SM and ZT), with the SLE international experts (John Esdaile (Canada), Martin Aringer (Germany), Matthias Schneider (Germany), Anca Askenaze (USA), Rosalind Ramsey-Goldman (USA), George Karpouzas (USA), Alfred Kim (USA), Julian Thumboo (Singapore), Eric Morand (Australia), David Tunnicliffe (Australia), Roger Levy (Brazil/(USA), Edward Vital (UK), Ian Bruce (UK)), the Patient Research Partners (PRPs) (Kirsten Lerstrom (Denmark), Francesca Marchiori (Italy), Davide Mazzoni (Italy), Nelma Nimaut (Brazil), Eduardo Ferreira Borba Neto (Brazil), Izabel Oliveiras (Brazil), Amy Reynolds (Australia), Corry Ang (Australia), Adwoa Parker (UK and MRC), Karina Svalya (Arthritis Research Canada), Debra Hurst (Canada), Rowena Rodriguez (Canada), Kelsey Schmitt (USA), Lucretia Taper (USA), Janeen Mays (USA)) and SLE sponsor reviewers from Amgen (Brian Ortemeier and Brad Stolshek), AstraZeneca (David Ginkel and Micki Hultquist and Ogun Sasova), EMD Serono (Amy Kao and Stephen Wax), Pfizer (Connie Chen, Sam Zwillich and Noriko Likuni), Janssen (Chetan Karyekar, Mark Chevrier and Pamela Barry), Lilly (Julie Birt) and Xencor (Debra Zack). All members from the SLE working group, the PRPs and SLE sponsor reviewers were involved in all steps of this review.
Competing interests None declared.
Patient and public involvement Patients and/or the public were involved in the design, or conduct, or reporting, or dissemination plans of this research. Refer to the Methods section for further details.
Patient consent for publication Not required.
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement All data relevant to the study are included in the article or uploaded as supplementary information. All data reviewed as part of this systematic review has been provided within the tables and supplementary materials.