Patient-reported outcome measures for use in clinical trials of SLE: a review - PubMed (original) (raw)

Patient-reported outcome measures for use in clinical trials of SLE: a review

Zara Izadi et al. Lupus Sci Med. 2018.

Erratum in

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.

Keywords: outcomes research; patient perspective; patient-reported outcomes; quality of life; systemic lupus erythematosus.

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Conflict of interest statement

Competing interests: None declared.

Figures

Figure 1

Figure 1

Of the 25 validation papers identified, 8 assessed the measurement properties of Short-Form 36, 1 assessed EuroQoL-5D, 7 assessed Lupus Quality of Life questionnaire, 5 assessed Patient Reported Outcomes Measurement Information System item-bank, 3 assessed Lupus Patient Reported Outcomes questionnaire, 6 assessed Lupus Impact Tracker, and 1 assessed Functional Assessment of Chronic Illness Therapy-Fatigue. Some studies assessed multiple quality of life instruments. *Extensively published defined as having >3 validation studies in English-speaking SLE populations or having been used in an RCT. Of the 26 RCT papers identified, 25 used Short-Form 36, 3 used Functional Assessment of Chronic Illness Therapy-Fatigue and 1 used EuroQol-5D. Some studies used >2 quality of life instruments. PRO, patient-reported outcome; RCT, randomised controlled trial.

References

    1. Archenholtz B, Burckhardt CS, Segesten K. Quality of life of women with systemic lupus erythematosus or rheumatoid arthritis: domains of importance and dissatisfaction. Qual Life Res 1999;8:411–6. 10.1023/A:1008915115370 -DOI -PubMed
    1. Jolly M, Katz P. Systemic lupus erythematosus: The promise of PROMIS - is it ready for prime time in SLE? Nat Rev Rheumatol 2017;13:453–4. 10.1038/nrrheum.2017.100 -DOI -PubMed
    1. Mahieu M, Yount S, Ramsey-Goldman R. Patient-reported outcomes in systemic lupus erythematosus. Rheum Dis Clin North Am 2016;42:253–63. 10.1016/j.rdc.2016.01.001 -DOI -PMC -PubMed
    1. Annapureddy N, Devilliers H, Jolly M. Patient-reported outcomes in lupus clinical trials with biologics. Lupus 2016;25:1111–21. 10.1177/0961203316652494 -DOI -PubMed
    1. McElhone K, Abbott J, Teh LS. A review of health related quality of life in systemic lupus erythematosus. Lupus 2006;15:633–43. 10.1177/0961203306071710 -DOI -PubMed

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