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Review
Patient-reported outcome measures for use in clinical trials of SLE: a review
  1. Zara Izadi1,
  2. Julie Gandrup2,
  3. Patricia P Katz2 and
  4. Jinoos Yazdany2
  1. 1 Department of Epidemiology and Biostatistics, University of California, San Francisco, California, USA
  2. 2 Division of Rheumatology, University of California, San Francisco, California, USA
  1. Correspondence to Dr Zara Izadi; zara.izadi{at}ucsf.edu

Abstract

Inclusion of patient-reported outcomes is important in SLE clinical trials as they allow capture of the benefits of a proposed intervention in areas deemed pertinent by patients. We aimed to compare the measurement properties of health-related quality of life (HRQoL) measures used in adults with SLE and to evaluate their responsiveness to interventions in randomised controlled trials (RCTs). A systematic review was undertaken using full original papers in English identified from three databases: MEDLINE, EMBASE and PubMed. Studies describing the validation of HRQoL measures in English-speaking adult patients with SLE and SLE drug RCTs that used an HRQoL measure were retrieved. Twenty-five validation papers and 26 RCTs were included in the indepth review evaluating the measurement properties of 4 generic (Medical Outcomes Study Short-Form 36 (SF36), Patient Reported Outcomes Measurement Information System (PROMIS) item-bank, EuroQol-5D, and Functional Assessment of Chronic Illness Therapy-Fatigue) and 3 disease-specific (Lupus Quality of Life (LupusQoL), Lupus Patient Reported Outcomes, Lupus Impact Tracker (LIT)) instruments. All measures had good convergent and discriminant validity. PROMIS provided the strongest evidence for known-group validity and reliability among generic instruments; however, data on its responsiveness have not been published. Across measures, standardised response means were generally indicative of poor-moderate sensitivity to longitudinal change. In RCTs, clinically important improvements were reported in SF36 scores from baseline; however, between-arm differences were frequently non-significant and non-important. SF36, PROMIS, LupusQoL and LIT had the strongest evidence for acceptable measurement properties, but few measures aside from the SF36 have been incorporated into clinical trials. This review highlights the importance of incorporating a broader range of SLE-specific HRQoL measures in RCTs and warrants further research that focuses on longitudinal responsiveness of newer instruments.

  • outcomes research
  • patient perspective
  • quality of life
  • systemic lupus erythematosus
  • patient-reported outcomes

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0

https://doi.org/10.1136/lupus-2018-000279

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    Footnotes

    • Contributors ZI is the primary author responsible for planning, conduct and reporting of the work described in the article. JG is an independent reviewer and has contributed to the conduct and reporting of the work described in the article. PPK and JY have provided significant input into the planning and reviewed the overall work.

    • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

    • Funding JY is supported by the Robert L Kroc Chair in Rheumatic and Connective Tissue Diseases, NIH/NIAMS P30 AR070155 and the Russell/Engleman Medical Research Center for Arthritis. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

    • Competing interests None declared.

    • Patient consent Not required.

    • Provenance and peer review Commissioned; externally peer reviewed.

    • Data statement All clinical trial data were retrieved from published manuscripts. Patient-level data or unpublished data were not obtained for this review. As such no further data is available for sharing beyond the content of this review.