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

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.

Responsiveness of the Patient‐Reported Outcomes Measurement Information System Global Health Short Form in Outpatients With Systemic Lupus Erythematosus

“…We found that PROMIS10 was not sensitive to changes in physicianassessed SLE disease activity. Similar findings have been shown in other disease specific and universal patient-reported outcome measures used in patients with SLE (21,22). We suspect that the difference in performance of the anchors reflects the different latent variables measured by patient-reported outcome surveys and physician-evaluated disease activity instruments.…”

“…Few studies have examined the responsiveness of patientreported outcome measures to patient-reported changes in SLE. A study of 101 SLE patients in the UK who were followed at 4-week intervals for 10 months, found small to moderate effect sizes (-0.11 to 0.53) for deterioration and improvement of patient-reported health status in physical function and mental health domains of the SF-36 and the LupusQoL, a disease-specific patient-reported outcome measure (21). Similarly, a study of 185 French patients with SLE who completed up to 3 surveys 3 months apart found low to moderate sensitivity to change using a patient-derived global assessment of disease impact for both the SF-36 PCS and MCS (SRM -0.25 to -0.61) and related domains of the LupusQoL (25).…”

Impact of belimumab on patient-reported outcomes in systemic lupus erythematosus: review of clinical studies

“…More recently, in 2018, Izadi et al reviewed the literature to assess the responsiveness of different PROs to interventions in RCTs. This review confirmed that SF-36 and LupusQoL have strong evidence for adequate psychometric measurement properties 58. In addition, the LIT and Patient-Reported Outcomes Measurement Information System (PROMIS) showed strong evidence for measurement properties; however, PROMIS responsiveness has not been studied in lupus, nor have LupusPRO or LIT been assessed in RCTs.…”

Health-Related Quality of Life Improvements in Systemic Lupus Erythematosus Derived from a Digital Therapeutic Plus Tele-Health Coaching Intervention: Randomized Controlled Pilot Trial

“…The primary outcomes were changes between baseline and week 16 in 11 domains reflecting various aspects of HRQoL, as assessed by 3 validated patient-reported outcome measures (PROMs): (1) Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), consisting of 13 questions aggregated into 1 domain measuring fatigue; (2) Brief Pain Inventory-Short Form (BPI-SF), consisting of 15 questions classified into 2 domains (pain severity and pain interference), and; (3) Lupus Quality of Life (LupusQoL), consisting of 34 questions classified into 8 domains (fatigue, physical health, planning, burden to others, emotional health, pain, intimate relationships, and body image). All 3 outcome measures have been previously described [ 32 - 34 ] and validated for use in SLE [ 29 , 35 , 36 ]. The participants were asked to complete these PROMs on a secure website prior to the start of the intervention and at weeks 4, 8, 12, and 16.…”

“…Prior to the study, the sample size was computed based on the Mann–Whitney U test to provide approximately 80% power to detect an effect size proportional to a mean difference in improvement of about 10% with a standard deviation of 10% without correcting for multiple comparisons. This effect size was chosen based on early user experience with the program as well as consideration of previously established minimally important differences for the outcome measures [ 35 - 37 ]. It was determined that a sample size of 50 was sufficient to allow for attrition and still produce the needed power with the remaining participants expected to complete the study.…”