Pos0342 performance of the Eular Systemic Sclerosis Impact of Disease (Scleroid) Questionnaire as a Patient Reported Outcome Measure for Patients With Diffuse Systemic Sclerosis

Abstract

BackgroundThere is a high unmet need for disease-modifying antifibrotic therapies in diffuse cutaneous systemic sclerosis (dcSSc) which could improve the outcome of this severe disease. Patient reported outcome measures (PROMs) are important and mandatory for randomized clinical trials (RCTs). The EULAR-endorsed ScleroID is the first comprehensive PROM specifically developed by SSc patients and experts to reflect the disease impact of SSc and showed a good performance in the clinical validation study [1]. However, most RCTs focus on dcSSc patients, hence a validated PROM to reflect the disease burden experienced by patients with dcSSc is needed and a detailed analysis of ScleroID in this subset of patients is lacking.ObjectivesTo investigate the performance of the EULAR ScleroID in patients with dcSSc as a prerequisite for its use as a PROM in RCTs testing potentially disease-modifying drugs.MethodsThis is a subanalysis of all patients with dcSSc from the large, multicentric, ScleroID validation cohort [1]. As comparators, SSc-HAQ, EQ-5D, SF-36 were included. The study had a longitudinal arm with a reliability visit at 7+/- 3 days and a 12-month follow-up visit [1]. The performance of ScleroID in dcSSc was assessed according to the OMERACT filter [1].Results152 dcSSc patients with a baseline visit were analyzed (44, 28.9% male, median age 54 years, median disease duration 7 years). ScleroID performed well as a PROM reflecting the disease impact of dcSSc: it showed a good construct validity with high Spearman’s correlation coefficients with comparators (SSc-HAQ, 0.79, 95%CI [0.69, 0.86]; HAQ-DI, 0.72 95%CI [0.60, 0.80]; SF-36 physical score, -0.69 95%CI [-0.77, -0.60]). Furthermore, the internal consistency was strong: Cronbach’s alpha was 0.87 and the split half reliability coefficient was 0.88. In the longitudinal arm, 44 patients had a test-retest reliability visit and 113 a follow-up visit, of whom 19/113 (16.8%) reported a significant change at 12 months follow-up (11 improved, 8 worsened). ScleroID showed a good consistency and discrimination ability with excellent test-retest reliability (intra-class correlation coefficient 0.89, 95%CI [0.84, 0.92]) and moderate sensitivity to change (standardized response mean 0.54), superior to the comparators (SSc-HAQ, 0.01; HAQ-DI, -0.07; SF-36 physical score, -0.43). The results are summarized in Figure 1.ConclusionThe EULAR ScleroID, which is a novel, brief, disease specific, patient-derived, disease impact PROM, performs well for patients with dcSSc. This supports the inclusion and further analysis of ScleroID as a PROM to reflect patients’ perspective in RCTs.Reference[1]Becker M, Dobrota R, Garaiman A, et al. Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire. Ann Rheum Dis. 2022 Apr;81(4):507-515.Figure 1.Performance of ScleroID in dcSSc by the OMERACT filter for truth and discrimination.AcknowledgementsWe sincerely thank the patients’ representatives who contributed to the ScleroID study, without whom this project would not have been possible.Disclosure of InterestsRucsandra Dobrota Speakers bureau: Actelion, Consultant of: Boehringer-Ingelheim, Grant/research support from: Pfizer, Actelion, Alexandru Garaiman: None declared, Kim Fligelstone: None declared, Ann Kennedy: None declared, Annelise Roennow: None declared, Yannick Allanore Consultant of: AbbVie, AstraZeneca, Bayer, Boehringer-Ingelheim, Mylan, Janssen, Medsenic, Prometheus, Sanofi, Roche, Grant/research support from: Alpine Immunosciences, Medsenic, OSE Immunotherapeutics, Patricia Carreira: None declared, László Czirják: None declared, Christopher P Denton: None declared, Roger Hesselstrand: None declared, Gunnel Sandqvist: None declared, Otylia Kowal-Bielecka: None declared, Cosimo Bruni Speakers bureau: Eli-Lilly, Consultant of: Boehringer-Ingelheim, Grant/research support from: Gruppo Italiano Lotta alla Sclerodermia (GILS), European Scleroderma Trials and Research Group (EUSTAR), Foundation for research in Rheumatology (FOREUM), Scleroderma Clinical Trials Consortium (SCTC), AbbVie, Marco Matucci Cerinic: None declared, Carina Mihai Speakers bureau: Boehringer Ingelheim, Janssen, MED Talks Switzerland, Mepha, and PlayToKnow AG, Consultant of: Boehringer Ingelheim, Janssen, MED Talks Switzerland, Mepha, and PlayToKnow AG, Ana Maria Gheorghiu: None declared, Ulf Müller-Ladner: None declared, Joe Sexton: None declared, Tore K. Kvien: None declared, Turid Heiberg: None declared, Oliver Distler Speakers bureau: Bayer, Boehringer Ingelheim, Janssen, Medscape, Consultant of: 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant Siences, Amgen, AnaMar, Arxx, AstraZeneca, Baecon, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, Galapagos, Glenmark, Horizon, Inventiva, Kymera, Lupin, Miltenyi Biotec, Mitsubishi Tanabe, MSD, Novartis, Prometheus, Redxpharna, Roivant, Sanofi and Topadur, Grant/research support from: Kymera, Mitsubishi Tanabe, Boehringer Ingelheim, Mike O. Becker Speakers bureau: Mepha, Bayer, MSD, GSK, Amgen, Novartis and Vifor, Consultant of: Mepha, Bayer, MSD, GSK, Amgen, Novartis and Vifor.