Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort. Issue 2 (3rd November 2022)

• Record Type: Journal Article
• Authors: Kuster, Simon; Jordan, Suzana; Elhai, Muriel; Held, Ulrike; Steigmiller, Klaus; Bruni, Cosimo; Cacciapaglia, Fabio; Vettori, Serena; Siegert, Elise; Rednic, Simona; Codullo, Veronica; Airo, Paolo; Braun-Moscovici, Yolanda; Hunzelmann, Nicolas; Joao Salvador, Maria; Riccieri, Valeria; Gheorghiu, Ana-Maria; Alegre Sancho, Juan José; Romanowska-Prochnicka, Katarzyna; Castellví, Ivan; Kötter, Ina; Truchetet, Marie-Elise; López-Longo, FJ; Novikov, Pavel I; Giollo, Alessandro; Shirai, Yuichiro; Belloli, Laura; Zanatta, Elisabetta; Hachulla, Eric; Smith, Vanessa; Denton, Chris; Ionescu, Ruxandra M; Schmeiser, Tim; Distler, Joerg H W; Gabrielli, Armando; Hoffmann-Vold, Anna-Maria; Kuwana, Masataka; Allanore, Yannick; Distler, Oliver;
• Other Names: author non-byline.; Becvar Radim author non-byline.; Cutolo Maurizio author non-byline.; Carreira Patricia E author non-byline.; Czirják László author non-byline.; Iudici Michele author non-byline.; Kucharz Eugene J author non-byline.; Coleiro Bernard author non-byline.; Bancel Dominique Farge author non-byline.; Hesselstrand Roger author non-byline.; Radic Mislav author non-byline.; Pellerito Raffaele author non-byline.; Damjanov Nemanja author non-byline.; Henes Jörg author non-byline.; Vera Ortiz-Santamaria Stefan Heitmann author non-byline.; Hasler Paul author non-byline.; Stamenkovic Bojana author non-byline.; Selmi Carlo Francesco author non-byline.; Tikly Mohammed author non-byline.; Ananieva Lidia P author non-byline.; Müller-Ladner Ulf author non-byline.; Engelhart Merete author non-byline.; Puente Carlos de la author non-byline.; Sunderkötter Cord author non-byline.; Ingegnoli Francesca author non-byline.; Mouthon Luc author non-byline.; Cantatore Francesco Paolo author non-byline.; Ullman Susanne author non-byline.; Pozzi Maria Rosa author non-byline.; Wiland Piotr author non-byline.; Vanthuyne Marie author non-byline.; Alegre-Sancho Juan Jose author non-byline.; Krummel-Lorenz Brigitte author non-byline.; Herrmann Kristine author non-byline.; Langhe Ellen De author non-byline.; Anic Branimir author non-byline.; Yavuz Sule author non-byline.; Müller Carolina de Souza author non-byline.; Agachi Svetlana author non-byline.; Zenone Thierry author non-byline.; Stebbings Simon author non-byline.; Vacca Alessandra author non-byline.; Stamp Lisa author non-byline.; Solanki Kamal author non-byline.; Veale Douglas author non-byline.; Loyo Esthela author non-byline.; Li Mengtao author non-byline.; Mohamed Walid Ahmed Abdel Atty author non-byline.; Rosato Edoardo author non-byline.; Tanaseanu Cristina-Mihaela author non-byline.; Foti Rosario author non-byline.; Ancuta Codrina author non-byline.; Maurer Britta author non-byline.; Lefebvre Paloma Garcíadela Peña author non-byline.; Sibilia Jean author non-byline.; Litinsky Ira author non-byline.; Galdo Francesco Del author non-byline.; Seskute Goda author non-byline.; Saketkoo Lesley Ann author non-byline.; Kerzberg Eduardo author non-byline.; Rimar Doron author non-byline.; Ribi Camillo author non-byline.; Hsu Vivien M author non-byline.; Martin Thierry author non-byline.; Chung Lorinda S author non-byline.; Schmeiser Tim author non-byline.; Majewski Dominik author non-byline.; Bernardino Vera author non-byline.; Puttini Piercarlo Sarzi author non-byline.;
• Is Part Of: RMD open. Volume 8:Issue 2(2022)
• Publication Date: 2022-11-03
• Subjects: systemic sclerosis -- autoimmune diseases -- biological therapy; Musculoskeletal system -- Diseases -- Periodicals; Rheumatism -- Periodicals; 616.7005
• DOI: 10.1136/rmdopen-2022-002477
• Languages: English
• ISSNs: 2056-5933

Abstract

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.