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

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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.

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The efficacy and safety of abatacept in patients with non-life-threatening manifestations of systemic lupus erythematosus: results of a twelve-month, multicenter, exploratory, phase IIb, randomized, double-blind, placebo-controlled trial.

D D'Cruz, L Sigal, Anne Becker … (2010)

To evaluate abatacept therapy in patients with non-life-threatening systemic lupus erythematosus (SLE) and polyarthritis, discoid lesions, or pleuritis and/or pericarditis. In a 12-month, multicenter, exploratory, phase IIb randomized, double-blind, placebo-controlled trial, SLE patients with polyarthritis, discoid lesions, or pleuritis and/or pericarditis were randomized at a ratio of 2:1 to receive abatacept (∼10 mg/kg of body weight) or placebo. Prednisone (30 mg/day or equivalent) was given for 1 month, and then the dosage was tapered. The primary end point was the proportion of patients with new flare (adjudicated) according to a score of A/B on the British Isles Lupus Assessment Group (BILAG) index after the start of the steroid taper. A total of 118 patients were randomized to receive abatacept and 57 to receive placebo. The baseline characteristics were similar in the 2 groups. The proportion of new BILAG A/B flares over 12 months was 79.7% (95% confidence interval [95% CI] 72.4, 86.9) in the abatacept group and 82.5% (95% CI 72.6, 92.3) in the placebo group (treatment difference -3.5 [95% CI -15.3, 8.3]). Other prespecified flare end points were not met. In post hoc analyses, the proportions of abatacept-treated and placebo-treated patients with a BILAG A flare were 40.7% (95% CI 31.8, 49.5) versus 54.4% (95% CI 41.5, 67.3), and the proportions with physician-assessed flare were 63.6% (95% CI 54.9, 72.2) and 82.5% (95% CI 72.6, 92.3), respectively; treatment differences were greatest in the polyarthritis group. Prespecified exploratory patient-reported outcomes (Short Form 36 health survey, sleep problems, fatigue) demonstrated a treatment effect with abatacept. The frequency of adverse events (AEs) was comparable in the abatacept and placebo groups (90.9% versus 91.5%), but serious AEs (SAEs) were higher in the abatacept group (19.8 versus 6.8%). Most SAEs were single, disease-related events occurring during the first 6 months of the study (including the steroid taper period). Although the primary/secondary end points were not met in this study, improvements in certain exploratory measures suggest some abatacept efficacy in patients with non-life-threatening manifestations of SLE. The increased rate of SAEs requires further assessment.

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Development and validation of a disease-specific health-related quality of life measure, the LupusQol, for adults with systemic lupus erythematosus.

Ada Ferenkeh-Koroma, M Akil, K Peers … (2007)

To develop and validate a disease-specific health-related quality of life (HRQOL) instrument for adults with systemic lupus erythematosus (SLE). The work consisted of 6 stages. Stage 1 included item generation for questionnaire content from semistructured interviews with SLE patients. In stage 2 item selection for the draft questionnaire was performed by thematic analysis of the patient interview transcripts and expert panel agreement. In stage 3 the content validity of the draft questionnaire was assessed by patients completing the questionnaire and providing critical feedback. In stages 4 and 5 construct validity and internal reliability of the 3 versions of the LupusQoL were evaluated using principal component analysis with varimax rotation and Cronbach's alpha coefficients, respectively. In stage 6 discriminatory validity, concurrent validity, and test-retest reliability were evaluated. Stages 1, 2, and 3 resulted in a preliminary instrument containing 63 items. In stage 4, 8 domains were identified. This factor structure, accounting for 82% of the variance, was confirmed in stage 5. The domains and Cronbach's alpha coefficients were physical health (0.94), emotional health (0.94), body image (0.89), pain (0.92), planning (0.93), fatigue (0.88), intimate relationships (0.96), and burden to others (0.94). Discriminant validity was demonstrated for different levels of disease activity (British Isles Lupus Assessment Group Index) and damage (Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index). High correlations (r = 0.71-0.79) between comparable domains of the Short Form 36 and the LupusQoL assured acceptable concurrent validity. Good test-retest reliability (r = 0.72-0.93) was demonstrated. The LupusQoL is a validated SLE-specific HRQOL instrument with 34 items across 8 domains defined by patients as being important.

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Efficacy and safety of an interleukin 6 monoclonal antibody for the treatment of systemic lupus erythematosus: a phase II dose-ranging randomised controlled trial

Daniel Wallace, Vibeke Strand, Joan Merrill … (2017)

Objectives This phase II trial evaluated the efficacy and safety of an interleukin (IL) 6 monoclonal antibody for systemic lupus erythematosus (SLE). Methods Patients with active disease were randomised to placebo or PF-04236921 10 mg, 50 mg or 200 mg, subcutaneously, every 8 weeks with stable background therapy. SLE Responder Index (SRI-4; primary end point) and British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) were assessed at week 24. Post hoc analysis identified an enriched population based upon planned univariate analyses. Results 183 patients received treatment (placebo, n=45; 10 mg, n=45; 50 mg, n=47; 200 mg, n=46). The 200 mg dose was discontinued due to safety findings and not included in the primary efficacy analysis. The SRI-4 response rates were not significant for any dose compared with placebo; however, the BICLA response rate was significant for 10 mg (p=0.026). The incidence of severe flares was significantly reduced with 10 mg (n=0) and 50 mg (n=2) combined versus placebo (n=8; p<0.01). In patients with greater baseline disease activity (enriched population), the SRI-4 (p=0.004) and BICLA (p=0.012) response rates were significantly different with 10 mg versus placebo. Four deaths (200 mg, n=3; 10 mg, n=1) occurred. The most frequently reported adverse events included headache, nausea and diarrhoea. Conclusions PF-04236921 was not significantly different from placebo for the primary efficacy end point in patients with SLE. Evidence of an effect with 10 mg was seen in a post hoc analysis. Safety was acceptable for doses up to 50 mg as the 200 mg dose was discontinued due to safety findings. Trial registration number NCT01405196; Pre-results.

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Author and article information

Journal

Journal ID (nlm-ta): Lupus Sci Med

Journal ID (iso-abbrev): Lupus Sci Med

Journal ID (hwp): lupusscimed

Journal ID (publisher-id): lupus

Title: Lupus Science & Medicine

Publisher: BMJ Publishing Group (BMA House, Tavistock Square, London, WC1H 9JR )

ISSN (Electronic): 2053-8790

Publication date Collection: 2018

Publication date (Electronic): 21 August 2018

Volume: 5

Issue: 1

Electronic Location Identifier: e000279

Affiliations

[1 ] departmentDepartment of Epidemiology and Biostatistics , University of California , San Francisco, California, USA

[2 ] departmentDivision of Rheumatology , University of California , San Francisco, California, USA

Author notes

[Correspondence to ] Dr Zara Izadi; zara.izadi@ 123456ucsf.edu

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Zara Izadi http://orcid.org/0000-0002-1867-0905

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Publisher ID: lupus-2018-000279

DOI: 10.1136/lupus-2018-000279

PMC ID: 6109821

PubMed ID: 30167315

SO-VID: 2a43866e-4873-4062-81c6-52d7429eddfe

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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

History

Date received : 28 May 2018

Date revision received : 19 July 2018

Date accepted : 21 July 2018

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

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

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

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Asian Journal of Quality of Life (AjQoL)

Most cited references 74

The efficacy and safety of abatacept in patients with non-life-threatening manifestations of systemic lupus erythematosus: results of a twelve-month, multicenter, exploratory, phase IIb, randomized, double-blind, placebo-controlled trial.

Development and validation of a disease-specific health-related quality of life measure, the LupusQol, for adults with systemic lupus erythematosus.

Efficacy and safety of an interleukin 6 monoclonal antibody for the treatment of systemic lupus erythematosus: a phase II dose-ranging randomised controlled trial

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