TY - JOUR T1 - Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort JF - RMD Open JO - RMD Open DO - 10.1136/rmdopen-2022-002477 VL - 8 IS - 2 SP - e002477 AU - Simon Kuster AU - Suzana Jordan AU - Muriel Elhai AU - Ulrike Held AU - Klaus Steigmiller AU - Cosimo Bruni AU - Fabio Cacciapaglia AU - Serena Vettori AU - Elise Siegert AU - Simona Rednic AU - Veronica Codullo AU - Paolo Airo AU - Yolanda Braun-Moscovici AU - Nicolas Hunzelmann AU - Maria Joao Salvador AU - Valeria Riccieri AU - Ana-Maria Gheorghiu AU - Juan José Alegre Sancho AU - Katarzyna Romanowska-Prochnicka AU - Ivan Castellví AU - Ina Kötter AU - Marie-Elise Truchetet AU - FJ López-Longo AU - Pavel I Novikov AU - Alessandro Giollo AU - Yuichiro Shirai AU - Laura Belloli AU - Elisabetta Zanatta AU - Eric Hachulla AU - Vanessa Smith AU - Chris Denton AU - Ruxandra M Ionescu AU - Tim Schmeiser AU - Joerg H W Distler AU - Armando Gabrielli AU - Anna-Maria Hoffmann-Vold AU - Masataka Kuwana AU - Yannick Allanore AU - Oliver Distler A2 - , Y1 - 2022/11/01 UR - http://rmdopen.bmj.com/content/8/2/e002477.abstract N2 - Objectives Tocilizumab showed trends for improving skin fibrosis and prevented progression of lung fibrosis in systemic sclerosis (SSc) in randomised controlled clinical trials. We aimed to assess safety and effectiveness of tocilizumab in a real-life setting using the European Scleroderma Trial and Research (EUSTAR) database.Methods Patients with SSc fulfilling the American College of Rheumatology (ACR)/EULAR 2013 classification criteria, with baseline and follow-up visits at 12±3 months, receiving tocilizumab or standard of care as the control group, were selected. Propensity score matching was applied. Primary endpoints were the modified Rodnan skin score (mRSS) and FVC at 12±3 months compared between the groups. Secondary endpoints were the percentage of progressive/regressive patients for skin and lung at 12±3 months.Results Ninety-three patients with SSc treated with tocilizumab and 3180 patients with SSc with standard of care fulfilled the inclusion criteria. Comparison between groups did not show significant differences, but favoured tocilizumab across all predefined primary and secondary endpoints: mRSS was lower in the tocilizumab group (difference −1.0, 95% CI −3.7 to 1.8, p=0.48). Similarly, FVC % predicted was higher in the tocilizumab group (difference 1.5 (−6.1 to 9.1), p=0.70). The percentage of progressive/regressive patients favoured tocilizumab over controls. These results were robust regarding the sensitivity analyses. Safety analysis confirmed previously reported adverse event profiles.Conclusion Although this large, observational, controlled, real-life EUSTAR study did not show significant effectiveness of tocilizumab on skin and lung fibrosis, the consistency of direction of all predefined endpoints generates hypothesis for potential effectiveness in a broader SSc population.Data are available on reasonable request. Anonymised data might be available from OD at the Department of Rheumatology, University Hospiztal Zurich, University of Zurich, Switzerland on reasonable request. ER -