Table 1

Lupus clinical trial outcome measures

Project/measure nameProject lead(s)Lupus phenotypeFocus of measureSummary description
Completed initiatives
Cutaneous LE Disease Area and Severity Index (CLASI)Victoria Werth
University of Pennsylvania
CLEDisease activity
  • Measures both cutaneous lupus activity and damage8 9; response correlates with changes in QoL and biomarkers, and meaningful response from patient perspective determined10 11

  • Validation studies show excellent inter-rater and intra-rater reliability, responsiveness8 12 13

  • Used in prospective international phase II and III trials, showing differences in response in treatment relative to placebo arms14 15

  • Widely used as both primary outcome in phase I and II CLE trials and secondary outcome in SLE phase I–III trials14 15

Definition of Remission in SLE (DORIS)Ronald van Vollenhoven
Amsterdam University Medical Center Netherlands
SLETreat-to-target endpoint: remission
  • Includes a SLE disease activity index without points for anti-DNA or low complement (cSLEDAI) and a PhGA (Physician Global Assessment) <0.5 (0–3), irrespective of serology5 6

LFA rapid evaluation of activity in lupus (LFA-REAL)Anca Askanase
Columbia University
Stan Kamp and Joan Merrill
Oklahoma Medical Research Foundation
SLEDisease activity
  • Two-part system with patient-reported outcome and clinician-reported outcome measures that allow for integration of both for targeted treatment decision-making

  • Use does not require specialised training or fluency in English

  • Has been used as an exploratory endpoint in several clinical trials and has proven effective16 17

Lupus Low Disease Activity State (LLDAS)Eric Morand
Monash University
SLETreat-to-target endpoint: remission
  • Concept definition is ‘A state, which if sustained is associated with a low likelihood of adverse outcome, considering disease activity and medication safety’. Incorporates thresholds for disease activity and treatment burden especially glucocorticoid dose

  • Formally validated in prospective multinational studies as protective against flare, damage, loss of quality of life and mortality.18 19 Also validated in many retrospective cohort studies

  • Extensive evaluation in post hoc analysis of clinical trials data, where it has good to excellent performance discriminating active treatment from placebo20–22

  • Widely adopted prospectively as a key secondary outcome measure in SLE trials23 24

SLE Disease Activity Score (SLE-DAS)Luís Inés
Coimbra University Hospital Centre
SLERemission and level of disease activity
Change measure
Treatment response
  • A SLE continuous measure with high sensitivity for changes in disease activity

  • Includes 17 weighted clinical and laboratory parameters attributed to SLE disease activity including continuous measures, important manifestations absent from SLEDAI and an improved weighting system

  • Validated in peer reviewed publications25–28

Ongoing initiatives
Outcome Measures in Rheumatology (OMERACT)Zahi Touma
University of Toronto
SLEPathophysiology and impact of health conditions
  • Identifying key domains such as disease activity, health-related quality of life and functional ability

  • Goal is to update original core domain set from 1998

  • Will continue to be updated following the results of additional studies aiming to generate a preliminary list of domains relevant for patients with SLE, physicians and other stakeholders and further analyses and vote on the final OMERACT SLE core domain set

Lupus Multivariable Outcome Score (LuMOS)Michal Abrahamowicz McGill University
Peter Lipsky
AMPEL BioSolutions
SLETreatment response
  • Developed to capture change with the least number of features in trials; aggregates changes in SLEDAI score, selected BILAG items, prednisone dose and selected biomarkers29

  • Validated using the Bliss-76 study7

  • Study findings suggest that LuMOS 2.0 formula may be a potential primary endpoint in future SLE trials

LRA/BMS Outcome InstrumentKen Kalunian
UC San Diego
SLEMultiple—TBDIn development
Treatment Response Measure for SLE
Eric Morand
Monash University
SLETreatment response
  • Multi-domain ClinRO being developed for use in randomised clinical trials. Will measure domains that impact how the patient feels, functions and survives in accordance with FDA guidance30