POS0203 EVIDENCE-BASED EXPERT CONSENSUS DEFINITION OF ORGAN INVOLVEMENT IN SYSTEMIC SCLEROSIS – A EUSTAR STUDY
Authors: A. Hoffmann-Vold, L. Petelytska, I. Barua, C. Bruni, C. Campochiaro, A. Gheorghiu, A. Lescoat, M. Vonk, I. Galetti, A. Gabrielli, O. Distler
Journal: Annals of the Rheumatic Diseases (2024)

<h3>Background:</h3> Systemic sclerosis (SSc) is a multiorgan disease but, to date, only few definitions of the various organ manifestation specific for SSc exist. When available, these definitions are mainly derived from other diseases, i.e. interstitial lung disease (ILD), where definitions are adopted from idiopathic pulmonary fibrosis (IPF). For comparison of clinical studies and inclusion of patients in randomized clinical trials, standardization of definitions for organ involvement and behaviour over time is of utmost importance. <h3>Objectives:</h3> To establish expert consensus on definitions of presence of organ involvement, severity of involvement, progression, stability and improvement of organ involvement, and end-stage organ disease. <h3>Methods:</h3> Ten SSc experts from EUSTAR centres formed the steering committee to conduct the EUSTAR study CP-125. In the first stage, a list of items for the definitions on different organs was created based on a narrative literature search and based on availability of the variables collected in the EUSTAR database. In the second step, using an online survey, the experts were asked to assess the proposed items and add additional items felt to be important to define the respective definitions. Finally, during four online meetings, the steering committee discussed the previous results using the nominal group technique (NGT) and voted on each of the statements (Figure 1). The parameters voted for by more than 75% of the experts were regarded as acceptable to be included in the different definitions. <h3>Results:</h3> The literature search revealed 179 definitions from 820 papers. After expert evaluation, 199 items were suggested for 9 organ domains including (1) Lung - ILD; (2) Lung – pulmonary hypertension (PH); (3) Heart; (4) Kidney; (5) Skin; (6) Digital ulcers; (7) Arthritis; (8) Muscle; (9) Gastrointestinal tract. During the NGT meetings, definitions for five categories were discussed in each organ domain including the presence of organ involvement, severity of organ involvement, disease progression, disease improvement and end-stage organ involvement. The list of items selected by the online survey was presented and each voting member was asked to discuss the relevance of these items. Of 74 tools proposed and endorsed items to describe organ involvement in SSc; 9 definitions for presence, 7 definitions for severity, 9 definitions for progression, 4 definitions for improvement and 4 for end-organ disease were agreed on (Table 1). <h3>Conclusion:</h3> A core set of data driven, consensus-based definitions for different organ involvements and disease courses has been defined applying a robust and comprehensive methodology. These definitions are recommended by this SSc expert group to be used as interim tools for research studies. <h3>REFERENCES:</h3> <b>NIL.</b> <h3>Acknowledgements:</h3> <b>NIL.</b> <h3>Disclosure of Interests:</h3> Anna-Maria Hoffmann-Vold Boehringer Ingelheim, Boehringer Ingelheim, Janssen, Medscape, Merck Sharp &amp; Dohme, Novartis and Roche. Consultant for ARXX, BMS, Boehringer Ingelheim, Genentech, Janssen, Medscape, Merck Sharp &amp; Dohme and Roche., Boehringer Ingelheim, Janssen, Liubov Petelytska Research grant from Swiss National Research Foundation/Scholars at risk, Imon Barua: None declared, Cosimo Bruni Boehringer Ingelheim, Foundation for Research in Rheumatology (FOREUM), Gruppo Italiano Lotta alla Sclerodermia (GILS), European Scleroderma Trials and Research Group (EUSTAR), Foundation for research in Rheumatology (FOREUM), Scleroderma Clinical Trials Consortium (SCTC), Scleroderma Research Foundation (SRF), Novartis Foundation for Bio-medical Research, EMDO Foundation. Educational grants from AbbVie and Wellcome Trust. Congress participation support from Boehringer Ingelheim., Corrado Campochiaro: None declared, Ana Maria Gheorghiu: None declared, Alain LESCOAT: None declared, Madelon Vonk Boehringer Ingelheim, Bristol-Myers Squibb, GSK, Janssen Pharmaceutical Companies of Johnson &amp; Johnson, MSD, Novartis, and Roche, Boehringer Ingelheim and Janssen Pharmaceutical Companies of Johnson &amp; Johnson, Boehringer Ingelheim, Ferrer, Galapagos and Janssen Pharmaceutical Companies of Johnson &amp; Johnson, Ilaria Galetti: None declared, Armando Gabrielli: 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, Patent issued "mir-29 for the treatment of systemic sclerosis" (US8247389, EP2331143). Co-founder of CITUS AG., 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.