AB1199 PATIENTS AND PHYSICIANS ATTRIBUTE IMPORTANCE DIFFERENTLY WHEN CONSIDERING OUTCOMES AND SEVERITY OF INTERSTITIAL LUNG DISEASE ASSOCIATED TO SYSTEMIC SCLEROSIS: RESULTS OF AN INTERNATIONAL SURVEY
Authors: C. Bruni, L. Petelytska, M. Elhai, I. Galetti, V. Guimarães, Q. Yan, S. Muraru, G. Sauer, N. Msabri Gharbi, P. Sen, K. El Aoufy, S. Jordan, O. Distler
Journal: Annals of the Rheumatic Diseases (2024)

<h3>Background:</h3> The criteria for defining outcomes and severity in systemic sclerosis-associated interstitial lung disease (SSc-ILD) are highly heterogeneous. Moreover, little is known about patients' and physicians' perspective on the most important aspects to consider when dealing with SSc-ILD. <h3>Objectives:</h3> his survey aimed to identify which outcomes and severity measures related to SSc-ILD are considered important for patients and physicians, to be further included when defining the long-term treatment goals of SSc-ILD. <h3>Methods:</h3> An international survey was designed based on a systematic literature review of outcomes and severity measures in SSc-ILD, with combined inputs from both physicians and patients. For physicians, the survey was conducted in English. For patients, the survey was adapted to lay terms and translated into different languages by native speakers (Italian, Portuguese, Spanish, Arabic, Chinese, French, Romanian, Ukrainian, German and Hindi), then spread through SSc patient associations. Survey participants were asked to rate on a scale of 1-10 the importance of each item when considering the outcomes of the treatment and when defining severe SSc-ILD, with higher values representing increasing importance. Items scored in average ≥8 and voted ≥8 by ≥80% of participants were arbitrarily considered as "important". <h3>Results:</h3> 738 participants completed the surveys, including 388 physicians with experience with SSc-ILD and 350 patients with SSc-ILD. Most physicians were based in Europe (70%), were specialists in rheumatology (76%), working at university hospitals (83%) and having been in practice for more than 5 years (82%). Patients with SSc-ILD were mostly from Europe (46%) and North America (39%), most of them being females (92%), aged over 50 years (63%) and living with SSc for longer than 3 years (75%). Both physicians and patients deemed death, a decline of pulmonary functional tests (PFTs), an increase of ILD extent on high-resolution computed tomography (HRCT) and onset/worsening of dyspnoea as important outcomes to be prevented with treatment (Figure 1A-B). Additionally, hospitalization (both with and without increased outpatient clinic burden), referral to lung transplantation, need to start/increase oxygen supplementation and onset/worsening of pulmonary hypertension secondary to ILD (PH-ILD) were important outcomes for physicians. Conversely, patients also considered lung infections, worsening of physical activity and worsening of psychological well-being important outcomes. Similar trends were noted when asking about items to define severe disease (Figure 1 C-D): while referral to lung transplantation, need for oxygen supplementation, ILD extent on HRCT and dyspnoea were important to both physicians and patients, PFTs and PH-ILD were rated important only by physicians, while lung infections, physical activity and daily activities were highly chosen only by patients. Outcomes and items such as cough, impact of ILD on sexual, social, and working life were not deemed important by any group. <h3>Conclusion:</h3> The perspectives of patients and physicians partially overlap, when considering the targets of treatment of SSc-ILD and the items contributing to the perceived disease severity. Based on these insights, both stakeholders' opinion should be included in defining the long-term targets of SSc-ILD treatment, to create an appropriate definition of «progression» to be used as a surrogate outcome in clinical trials and practice.REFERENCES: NIL. <h3>Acknowledgements:</h3> <b>NIL.</b> <h3>Disclosure of Interests:</h3> 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., Liubov Petelytska research grant from Swiss National Research Foundation/Scholars at risk, Muriel Elhai congress support from Janssen and AstraZeneca, Ilaria Galetti: None declared, Vera Guimarães: None declared, Qingran Yan: None declared, Sinziana Muraru congress support from AstraZeneca (virtual EULAR congress 2021), Gesa Sauer: None declared, Nada Msabri Gharbi: None declared, Parikshit Sen: None declared, Khadija El Aoufy: None declared, Suzana Jordan: 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.