학술논문
Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort
Document Type
article
Author
Pavel I Novikov; Thierry Martin; Eric Hachulla; Vanessa Smith; Maurizio Cutolo; Yannick Allanore; Camillo Ribi; Oliver Distler; Britta Maurer; Paolo Airo; Cosimo Bruni; Veronica Codullo; Luc Mouthon; Marie Vanthuyne; Elisabetta Zanatta; Simona Rednic; Ulf Müller-Ladner; Nemanja Damjanov; Anna-Maria Hoffmann-Vold; Michele Iudici; Suzana Jordan; Jean Sibilia; Mengtao Li; Roger Hesselstrand; Elise Siegert; László Czirják; Armando Gabrielli; Dominique Farge Bancel; Yolanda Braun-Moscovici; Nicolas Hunzelmann; Raffaele Pellerito; Jörg Henes; Bojana Stamenkovic; Carlo Francesco Selmi; Mohammed Tikly; Merete Engelhart; Valeria Riccieri; Cord Sunderkötter; Francesca Ingegnoli; Francesco Paolo Cantatore; Susanne Ullman; Maria Rosa Pozzi; Piotr Wiland; Juan Jose Alegre-Sancho; Brigitte Krummel-Lorenz; Ellen De Langhe; Branimir Anic; Carolina de Souza Müller; Simon Stebbings; Alessandra Vacca; Lisa Stamp; Kamal Solanki; Douglas Veale; Esthela Loyo; Edoardo Rosato; Cristina-Mihaela Tanaseanu; Rosario Foti; Codrina Ancuta; Ira Litinsky; Lesley Ann Saketkoo; Eduardo Kerzberg; Tim Schmeiser; Piercarlo Sarzi Puttini; Fabio Cacciapaglia; Marie-Elise Truchetet; Muriel Elhai; Juan José Alegre Sancho; Ivan Castellví; Serena Vettori; Vivien M Hsu; Masataka Kuwana; Sule Yavuz; Radim Becvar; Patricia E Carreira; Eugene J Kucharz; Bernard Coleiro; Paul Hasler; Carlos de la Puente; Svetlana Agachi; Thierry Zenone; Francesco Del Galdo; Doron Rimar; Alessandro Giollo; Lidia P Ananieva; Ina Kötter; Mislav Radic; Dominik Majewski; Kristine Herrmann; Goda Seskute; Lorinda S Chung; Laura Belloli; Ulrike Held; FJ López-Longo; Joerg H W Distler; Maria João Salvador; Simon Kuster; Klaus Steigmiller; Ana-Maria Gheorghiu; Katarzyna Romanowska-Prochnicka; Yuichiro Shirai; Chris Denton; Ruxandra M Ionescu; Stefan Heitmann Vera Ortiz-Santamaria; Walid Ahmed Abdel Atty Mohamed; Paloma Garcíadela Peña Lefebvre; Vera Bernardino
Source
RMD Open, Vol 8, Iss 2 (2022)
Subject
Language
English
ISSN
2056-5933
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±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.