POS0432 DRIVERS OF DISEASE BURDEN IN PATIENTS WITH SYSTEMIC SCLEROSIS ACROSS CLINICAL AND DEMOGRAPHIC SUBGROUPS REVEALED BY THE EULAR SYSTEMIC SCLEROSIS IMPACT OF DISEASE (ScleroID) QUESTIONNAIRE
Authors: S. Muraru, A. Garaiman, K. Fligelstone, A. Kennedy, A. Roennow, Y. Allanore, P. Carreira, L. Czirják, C. P. Denton, R. Hesselstrand, G. Sandqvist, O. Kowal-Bielecka, C. Bruni, M. Matucci-Cerinic, C. Mihai, A. M. Gheorghiu, U. Müller-Ladner, T. K. Kvien, T. Heiberg, O. Distler, M. O. Becker, R. Dobrota
Journal: Annals of the Rheumatic Diseases (2024)

<h3>Background:</h3> Patient-reported outcome measures (PROMs) are crucial for understanding the disease burden of patients with systemic sclerosis (SSc). A team of patients and physicians developed the ScleroID as a specific impact of disease questionnaire for research and clinical use. However, it is yet unknown how ScleroID correlates with clinical aspects and how it varies between demographic groups. <h3>Objectives:</h3> We aim to investigate drivers of disease burden in patients with SSc, as reported by the ScleroID. <h3>Methods:</h3> In this post-hoc analysis of the ScleroID cohort [1], including SSc patients from 9 European centers, we analyzed the global disease burden as reflected by the ScleroID score and its individual health dimensions (Figure 1A) in relation to clinical and demographic variables. Moreover, we compared the ScleroID to the patient's and physician's global assessment of disease activity (PtGA, PhGA) and to self-perceived changes in disease activity, reported at baseline and at 1 year follow-up on Likert scale questions. We investigated longitudinally the clinical progression and the ScleroID trajectory. Disease progression was defined as new onset or progression of interstitial lung disease (ILD), new onset digital ulcers or an increase in mRSS of &gt;3 points and ≥25%. For correlations between continuous variables, we computed the Spearman coefficient. To analyze differences between groups, we used the Wilcoxon rank sum or the Kruskal-Wallis test, with pairwise comparisons and adjusted significance level (Bonferroni). The analysis was performed in R. <h3>Results:</h3> We included 471 patients, with a median age of 57 years, 84.7% female, median disease duration of 9 years, 62% with limited cutaneous SSc. At baseline, the ScleroID score strongly correlated with the PtGA (r= 0.78, p&lt;0.001), but only weakly with the PhGA (r= 0.29, p&lt;0.001). Moreover, the ScleroID score showed a good correlation with patient-reported current disease status (Figure 1B). ScleroID weakly correlated with the first European Scleroderma Study Group activity index (r= 0.23, p&lt;0.001). The disease burden assessed by ScleroID was higher in retired and medically disabled patients compared to working or studying individuals (p&lt;0.001 between all 3 categories), as well as in lower educated patients compared to those with university degrees (p= 0.01). Women reported a higher disease burden than men (p=0.001). We noted higher ScleroID scores in patients with a disease duration &gt;5 years (p=0.01), digital ulcers (p=0.02), dyspnea NYHA stages 3-4 (p&lt;0.001), ILD (p=0.03), and esophageal symptoms (p&lt;0.001). Interestingly, not only the corresponding ScleroID health dimension, but also multiple others, were affected (Table 1). At follow-up, the ScleroID score correlated with the PtGA (r= 0.87, p&lt;0.001) and PhGA (r= 0.39, p&lt;0.001), similarly to baseline. However, the change in ScleroID from baseline to follow-up showed weak correlation with change in PtGA (r= 0.34, p&lt;0.001) and no correlation with change in PhGA (r= -0.06, NS). Follow-up visits with clinical data were available for 82 patients. Patients with clinical disease progression (n=9) had significantly higher changes in the ScleroID from baseline to follow-up (p= 0.03, median change 0.76 points). However, only 5/9 patients self-reported worsening on the Likert scale, the other 4 noting a stable status. <h3>Conclusion:</h3> Disease duration &gt;5 years, female sex, digital ulcers, severe dyspnea, ILD and esophageal reflux, as well as work disability and a lower education status, were important drivers of a high disease burden as reflected by higher ScleroID scores. At follow-up, we found greater changes in ScleroID scores among patients with clinical disease progression, which was only partially recognized by the patients themselves. This aspect, together with the weak correlation with PhGA and with disease activity scores, emphasizes the need for integrating PROMs in the regular monitoring for clinical care and research. <h3>REFERENCES:</h3> [1] Becker MO, Dobrota R 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;81(4):507-15. <h3>Acknowledgements:</h3> <b>NIL.</b> <h3>Disclosure of Interests:</h3> Sinziana Muraru Participation fee virtual EULAR 2021 from AstraZeneca, Alexandru Garaiman: None declared, Kim Fligelstone: None declared, Ann Kennedy: None declared, Annelise Roennow: None declared, Yannick Allanore Actelion, Bayer, Roche/Genentech, Inventiva, Medac, Pfizer, Sanofi, Servier, and UCB, Bristol-Myers Squibb, Roche/Genentech, Inventiva, Pfizer, Sanofi, and Servier, Patricia Carreira Janssen, Lilly, VivaCell, Emerald Health Pharmaceuticals, Gesynta Pharma, Boehringer Ingelheim, AbbVie, Sanofi Genzyme, Mitsubishi Tanabe, Janssen, Lilly, VivaCell, Emerald Health Pharmaceuticals, Gesynta Pharma, Boehringer Ingelheim, AbbVie, Sanofi Genzyme, Mitsubishi Tanabe, László Czirják Boehringer, AstraZeneca, Roche, Novartis, AbbVie, Boehringer, AstraZeneca, Pfizer, Christopher P Denton Janssen and Boehringer Ingelheim, Janssen, GlaxoSmithKline, Boehringer Ingelheim, Roche, CSL Behring, Corbus, Acceleron, Horizon, Arxx Therapeutics, Lilly, Novartis, Certa, GSK, Abbvie, Horizon, Arxx, Servier, Roger Hesselstrand Boehringer Ingelheim AB, Gunnel Sandqvist: None declared, Otylia Kowal-Bielecka Abbvie, AstraZeneca, Boehringer-Ingelheim, Janssen-Cilag, Medac, MSD, Novartis, Pfizer, AstraZeneca, CSL Behring, Boehringer-Ingelheim, Cosimo Bruni Boehringer-Ingelheim, Scleroderma clinical trial consortium, Scleroderma research foundation, Gruppo Italiano Lotta alla Sclerodermia, Novartis Foundation for medical-biological research, EMDO Foundation, Iten-Kohaut Foundation, Abbvie, Wellcome trust., Marco Matucci-Cerinic Actelion, Janssen, Inventiva, Bayer, Biogen, Boehringer, CSL Behring, Corbus, Galapagos, Mitsubishi, Samsung, Regeneron, Acceleron, MSD, Chemomab, Lilly, Pfizer, Roche, Actelion, Janssen, Inventiva, Bayer, Biogen, Boehringer, CSL Behring, Corbus, Galapagos, Mitsubishi, Samsung, Regeneron, Acceleron, MSD, Chemomab, Lilly, Pfizer, Roche., Carina Mihai Biotest, Novartis, Mepha, Boehringer-Ingelheim, Janssen Cilag, Ana Maria Gheorghiu Novartis, Sandoz, Boehringer-Ingelheim, Boehringer-Ingelheim, Ulf Müller-Ladner: None declared, Tore K. Kvien Janssen, Sandoz, Grünenthal, UCB, AbbVie, Galapagos, Janssen, Pfizer, Sandoz, AbbVie, BMS, Novartis, Pfizer and UCB (to institution), Turid Heiberg: None declared, Oliver Distler 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant, Amgen, AnaMar, Argenx, Arxx, AstraZeneca, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, Galderma, Galapagos, Glenmark, Gossamer, Horizon, Janssen, Kymera, Lupin, Medscape, Merck, Miltenyi Biotec, Mitsubishi Tanabe, Novartis, Orion, Prometheus, Redxpharma, Roivant, Topadur and UCB, 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant, Amgen, AnaMar, Argenx, Arxx, AstraZeneca, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, Galderma, Galapagos, Glenmark, Gossamer, Horizon, Janssen, Kymera, Lupin, Medscape, Merck, Miltenyi Biotec, Mitsubishi Tanabe, Novartis, Orion, Prometheus, Redxpharma, Roivant, Topadur and UCB, Boehringer Ingelheim, Kymera, Mitsubishi Tanabe, Mike O. Becker GSK, Amgen, Novartis and outside of this research area: Vifor., GSK, Amgen, Novartis and outside of this research area: Vifor., GSK, Amgen, Novartis and outside of this research area: Vifor., Rucsandra Dobrota Actelion, Boehringer-Ingelheim, Pfizer, Actelion, Iten-Kohaut foundation, congress participation support from Amgen.