Measurement properties of selected patient-reported outcome measures for use in randomised controlled trials in patients with systemic lupus erythematosus: a systematic review

Abstract

ObjectiveThe 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.MethodsPublished 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.ResultsAll 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.ConclusionThe 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.

Patient Experience With Fatigue and Qualitative Interview-Based Evidence of Content Validation of The FACIT-Fatigue in Systemic Lupus Erythematosus

“…Participants in this study described fatigue as the most debilitating symptom of their condition. These findings confirm fatigue as an important concept to patients with SLE and suggest the value of including it as a secondary endpoint in randomized controlled trials and longitudinal observational studies to measure efficacy of SLE treatments [14]. Participants also confirmed the importance, relevance, and comprehensiveness of concepts covered in the FACIT-Fatigue.…”

“…Systematic reviews of PRO measures indicated that the FACIT-Fatigue has good face and psychometric validity, and contains concepts appropriate for measuring fatigue in patients with SLE [13,14]. Content validity of the FACIT-Fatigue has been previously established through evidence reported in studies of patients with SLE [13][14][15], as well as evidence collected through focus groups with patients with SLE [16]. The current study expands on existing evidence of content validity of the FACIT-Fatigue by collecting data directly from patients through individual oneon-one qualitative interviews.…”

“…Since its development in 1997 for use in patients with cancer, its appropriateness for use in other diseases has been evaluated, including autoimmune conditions like rheumatoid arthritis; and it has been used in numerous SLE clinical trials [ 12 ]. Systematic reviews of PRO measures indicated that the FACIT-Fatigue has good face and psychometric validity, and contains concepts appropriate for measuring fatigue in patients with SLE [ 13 , 14 ]. Content validity of the FACIT-Fatigue has been previously established through evidence reported in studies of patients with SLE [ 13 – 15 ], as well as evidence collected through focus groups with patients with SLE [ 16 ].…”

Patient-Reported Outcomes from a Phase 4, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Repository Corticotropin Injection (Acthar® Gel) for Persistently Active Systemic Lupus Erythematosus

“…For patients with higher disease activity, treatment with RCI may reduce the amount of work time missed due to SLE and may increase the amount or type of work that patients with SLE are able to complete. Few randomized clinical trials for SLE have reported LupusQoL or WPAI-Lupus results [27,28]. This is the first randomized study to report LupusQoL or WPAI-Lupus results after treatment with RCI.…”

“…Few randomized clinical trials for SLE have reported LupusQoL or WPAI-Lupus results [ 27 , 28 ]. This is the first randomized study to report LupusQoL or WPAI-Lupus results after treatment with RCI.…”