Pitfalls and Opportunities in Measuring Patient Outcomes in Lupus

Abstract

Health outcomes and their measures used as end points in systemic lupus erythematosus (SLE) patient care and clinical trials have been varied and are still evolving. Although significant reductions in morbidity and mortality in SLE have been achieved, the medications approved for SLE have remained the same during the past 50 years. Despite the pressing need, aggressive advocacy in the community, and advances in drug development and testing in SLE, no medications have met US Food and Drug Administration guidelines for a new indication claim approval for SLE. This may be attributable to its multisystemic, remitting, and relapsing nature, and difficulty in identifying useful end points and appropriate tools to measure them. Thus, it is pivotal to identify and validate appropriate global, disease-specific, and perhaps organ-specific health outcomes for clinical research. This article reviews recent physician- and patient-generated health outcomes in SLE.

Epratuzumab for patients with moderate to severe flaring SLE: health-related quality of life outcomes and corticosteroid use in the randomized controlled ALLEVIATE trials and extension study SL0006

“…Of interest, given prior recognized discordance, patients and physicians reported similar numbers, with ≥20% improvement in disease activity. The correspondence between these measurements is notable: patient-reported outcomes are strongly predictive of subsequent morbidity and mortality [33], but frequently do not correlate well with physician assessments in SLE [6]. As evident in surveys of patients with SLE [15, 34–36], they focus more on functional limitations resulting from SLE and/or co-morbid conditions (such as fibromyalgia), whereas physicians focus on clinical and laboratory assessments [33, 37].…”

“…The correspondence between these measurements is notable: patient-reported outcomes are strongly predictive of subsequent morbidity and mortality [33], but frequently do not correlate well with physician assessments in SLE [6]. As evident in surveys of patients with SLE [15, 34–36], they focus more on functional limitations resulting from SLE and/or co-morbid conditions (such as fibromyalgia), whereas physicians focus on clinical and laboratory assessments [33, 37]. In surveys, patients also consistently report dissatisfaction with their treatment, in particular with adverse effects associated with corticosteroid use [15], ubiquitous in patients with moderate to severely active disease [38].…”

“…Although regulatory agencies encourage the use of patient-reported outcomes in RCTs, they are often not included in SLE trials, in contrast with other chronic rheumatic diseases [6, 33]. The value of the assessment of HRQOL, especially in view of the broad impact of active disease on physical, emotional and social functioning, is significant and supports inclusion of patient-reported outcomes alongside measures of disease activity and damage in clinical trials.…”

The substantial burden of systemic lupus erythematosus on the productivity and careers of patients: a European patient-driven online survey

“…HRQoL measurements from patient-reported outcome (PRO) questionnaires can complement traditional physician-reported disease activity and damage outcomes [ 12 , 13 ]. For this reason, HRQoL is listed as a core domain to be addressed in SLE clinical trials, in recommendations published by the Outcome Measures in Rheumatology initiative [ 14 ].…”

Disease-specific quality of life in patients with lupus nephritis

“…7 Patient-reported outcomes are a core health outcome, and are included in patient care and clinical trials. 8 Not much is known about the impact of lupus nephritis on SLE patients' daily lives; most studies relating to this are smaller in size, or have used generic QOL instruments. [9][10][11][12][13] Use of diseasetargeted QOL tools may provide more pertinent and unique information on the effects of the disease, which may not be captured using generic QOL tools.…”