@article {Kustere002477, author = {Simon Kuster and Suzana Jordan and Muriel Elhai and Ulrike Held and Klaus Steigmiller and Cosimo Bruni and Fabio Cacciapaglia and Serena Vettori and Elise Siegert and Simona Rednic and Veronica Codullo and Paolo Airo and Yolanda Braun-Moscovici and Nicolas Hunzelmann and Maria Joao Salvador and Valeria Riccieri and Ana-Maria Gheorghiu and Juan Jos{\'e} Alegre Sancho and Katarzyna Romanowska-Prochnicka and Ivan Castellv{\'\i} and Ina K{\"o}tter and Marie-Elise Truchetet and FJ L{\'o}pez-Longo and Pavel I Novikov and Alessandro Giollo and Yuichiro Shirai and Laura Belloli and Elisabetta Zanatta and Eric Hachulla and Vanessa Smith and Chris Denton and Ruxandra M Ionescu and Tim Schmeiser and Joerg H W Distler and Armando Gabrielli and Anna-Maria Hoffmann-Vold and Masataka Kuwana and Yannick Allanore and Oliver Distler}, editor = {, and , and Becvar, Radim and Cutolo, Maurizio and Carreira, Patricia E and Czirj{\'a}k, L{\'a}szl{\'o} and Iudici, Michele and Kucharz, Eugene J and Coleiro, Bernard and Bancel, Dominique Farge and Hesselstrand, Roger and Radic, Mislav and Pellerito, Raffaele and Damjanov, Nemanja and Henes, J{\"o}rg and Vera Ortiz-Santamaria, Stefan Heitmann and Hasler, Paul and Stamenkovic, Bojana and Selmi, Carlo Francesco and Tikly, Mohammed and Ananieva, Lidia P and M{\"u}ller-Ladner, Ulf and Engelhart, Merete and Puente, Carlos de la and Sunderk{\"o}tter, Cord and Ingegnoli, Francesca and Mouthon, Luc and Cantatore, Francesco Paolo and Ullman, Susanne and Pozzi, Maria Rosa and Wiland, Piotr and Vanthuyne, Marie and Alegre-Sancho, Juan Jose and Krummel-Lorenz, Brigitte and Herrmann, Kristine and Langhe, Ellen De and Anic, Branimir and Yavuz, Sule and M{\"u}ller, Carolina de Souza and Agachi, Svetlana and Zenone, Thierry and Stebbings, Simon and Vacca, Alessandra and Stamp, Lisa and Solanki, Kamal and Veale, Douglas and Loyo, Esthela and Li, Mengtao and Mohamed, Walid Ahmed Abdel Atty and Rosato, Edoardo and Tanaseanu, Cristina-Mihaela and Foti, Rosario and Ancuta, Codrina and Maurer, Britta and Lefebvre, Paloma Garc{\'\i}adela Pe{\~n}a and Sibilia, Jean and Litinsky, Ira and Galdo, Francesco Del and Seskute, Goda and Saketkoo, Lesley Ann and Kerzberg, Eduardo and Rimar, Doron and Ribi, Camillo and Hsu, Vivien M and Martin, Thierry and Chung, Lorinda S and Schmeiser, Tim and Majewski, Dominik and Bernardino, Vera and Puttini, Piercarlo Sarzi}, title = {Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort}, volume = {8}, number = {2}, elocation-id = {e002477}, year = {2022}, doi = {10.1136/rmdopen-2022-002477}, publisher = {BMJ Specialist Journals}, abstract = {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{\textpm}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{\textpm}3 months compared between the groups. Secondary endpoints were the percentage of progressive/regressive patients for skin and lung at 12{\textpm}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.}, URL = {https://rmdopen.bmj.com/content/8/2/e002477}, eprint = {https://rmdopen.bmj.com/content/8/2/e002477.full.pdf}, journal = {RMD Open} }