Responsiveness of patient-reported outcomes by disease trajectory in SLE.
15 affiliations
- 1Department of Medicine, University of Calgary, Calgary, Alberta, Canada.
- 2Division of Experimental Medicine, McGill University Health Centre, Montreal, Quebec, Canada.
- 3Department of Health Research Methods, McMaster University, Hamilton, Ontario, Canada.
- 4Department of Mathematics and General Sciences, Prince Sultan University, Riyadh, Saudi Arabia.
- 5Faculty of Medicine and Health Sciences, McGill University, Montreal, Quebec, Canada.
- 6Faculty of Medicine, University of Montreal, Montreal, Quebec, Canada.
- 7Referral Center for Systemic Autoimmune Diseases, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico di Milano, Milan, Italy.
- 8Schroeder Arthritis Institute, Krembil Research Institute, University Health Network, Toronto, Ontario, Canada.
- 9Centre for Prognosis Studies in Rheumatic Diseases, University of Toronto Lupus Clinic, Toronto Western Hospital, Toronto, Ontario, Canada.
- 10Department of Dermatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
- 11Department of Surgery, Mary Washington Healthcare, Fredericksburg, VA, USA.
- 12Division of Dermatology, Department of Medicine, University of Toronto, Toronto, Ontario, Canada.
- 13Division of Rheumatology, Department of Medicine Solna, Karolinska Institutet, Karolinska University Hospital, Center for Molecular Medicine (CMM), Stockholm, Sweden.
- 14Department of Rheumatology, Faculty of Medicine and Health, Örebro University, Örebro, Sweden.
- 15Division of Dermatology, McGill University Health Centre, Montreal, Quebec, Canada.
Abstract
Objectives
SLE is a heterogeneous autoimmune disease with persistent symptoms that profoundly impair quality of life yet are often under-recognized by clinician-assessed instruments. Patient-reported outcomes (PROs) are increasingly incorporated in clinical trials to capture treatment benefit from the patient perspective. However, the responsiveness of PRO instruments and their alignment with clinician-assessed disease activity across distinct SLE trajectories remain incompletely defined. We evaluated the longitudinal responsiveness and convergent validity of PRO measures across predefined disease trajectory classes.Methods
Secondary analysis of pooled patient-level data from two phase III randomized clinical trials of belimumab in adults with active SLE (n = 1908), stratified into four previously derived trajectory classes defined by baseline disease activity and treatment response. PROs included Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), 36-Item Short Form Survey (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS), EuroQol 5-Dimension 3-level (EQ-5D-3L) and EuroQol Visual Analogue Scale (EQ-VAS). Responsiveness was quantified using standardized response means (SRMs). Convergent validity was evaluated using repeated-measures correlations between within-person changes in PROs and clinician-assessed disease activity measures (SLEDAI-2K, BILAG and Physician's Global Assessment [PGA]), with sensitivity analyses performed to assess the robustness of findings.Results
FACIT-F and SF-36 PCS generally demonstrated the greatest responsiveness, while EQ-5D-3L showed smaller changes and less consistent responsiveness across classes and timepoints. FACIT-F demonstrated early responsiveness, whereas SF-36 PCS showed sustained responsiveness over later follow-up. PRO changes were most consistently associated with PGA. Sensitivity analyses showed broadly similar patterns.Conclusion
FACIT-F and SF-36 PCS demonstrated the greatest responsiveness and alignment with clinician-assessed disease activity. EQ-5D-3L showed less consistent responsiveness, suggesting domain-specific PROs may better capture treatment benefit in SLE trials and health technology assessments.
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BibTeX
@article{hundal2026responsiveness,
title = {{Responsiveness of patient-reported outcomes by disease trajectory in SLE.}},
author = {Sabrina Hundal and Julian Cappelli and Mohammed Kaouache and Jessica Muha and Savanah Haidar and Lorenzo Beretta and Zahi Touma and John S Barbieri and Stephanie R Goldberg and Anastasiya Muntyanu and Ioannis Parodis and Elena Netchiporouk},
journal = {Rheumatology (Oxford, England)},
year = {2026},
month = oct,
doi = {10.1093/rheumatology/keag576},
url = {https://europepmc.org/article/MED/42879728},
note = {CC BY},
}
RIS
TY - JOUR
TI - Responsiveness of patient-reported outcomes by disease trajectory in SLE.
AU - Sabrina Hundal
AU - Julian Cappelli
AU - Mohammed Kaouache
AU - Jessica Muha
AU - Savanah Haidar
AU - Lorenzo Beretta
AU - Zahi Touma
AU - John S Barbieri
AU - Stephanie R Goldberg
AU - Anastasiya Muntyanu
AU - Ioannis Parodis
AU - Elena Netchiporouk
PY - 2026
DA - 2026/10/01
JO - Rheumatology (Oxford, England)
DO - 10.1093/rheumatology/keag576
UR - https://europepmc.org/article/MED/42879728
AB - Objectives SLE is a heterogeneous autoimmune disease with persistent symptoms that profoundly impair quality of life yet are often under-recognized by clinician-assessed instruments. Patient-reported outcomes (PROs) are increasingly incorporated in clinical trials to capture treatment benefit from the patient perspective. However, the responsiveness of PRO instruments and their alignment with clinician-assessed disease activity across distinct SLE trajectories remain incompletely defined. We evaluated the longitudinal responsiveness and convergent validity of PRO measures across predefined disease trajectory classes.Methods Secondary analysis of pooled patient-level data from two phase III randomized clinical trials of belimumab in adults with active SLE (n = 1908), stratified into four previously derived trajectory classes defined by baseline disease activity and treatment response. PROs included Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), 36-Item Short Form Survey (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS), EuroQol 5-Dimension 3-level (EQ-5D-3L) and EuroQol Visual Analogue Scale (EQ-VAS). Responsiveness was quantified using standardized response means (SRMs). Convergent validity was evaluated using repeated-measures correlations between within-person changes in PROs and clinician-assessed disease activity measures (SLEDAI-2K, BILAG and Physician's Global Assessment [PGA]), with sensitivity analyses performed to assess the robustness of findings.Results FACIT-F and SF-36 PCS generally demonstrated the greatest responsiveness, while EQ-5D-3L showed smaller changes and less consistent responsiveness across classes and timepoints. FACIT-F demonstrated early responsiveness, whereas SF-36 PCS showed sustained responsiveness over later follow-up. PRO changes were most consistently associated with PGA. Sensitivity analyses showed broadly similar patterns.Conclusion FACIT-F and SF-36 PCS demonstrated the greatest responsiveness and alignment with clinician-assessed disease activity. EQ-5D-3L showed less consistent responsiveness, suggesting domain-specific PROs may better capture treatment benefit in SLE trials and health technology assessments.
SN - 1462-0324
SN - 1462-0332
ER -