RT Journal Article
SR Electronic
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
FD EULAR
SP e002477
DO 10.1136/rmdopen-2022-002477
VO 8
IS 2
A1 Simon Kuster
A1 Suzana Jordan
A1 Muriel Elhai
A1 Ulrike Held
A1 Klaus Steigmiller
A1 Cosimo Bruni
A1 Fabio Cacciapaglia
A1 Serena Vettori
A1 Elise Siegert
A1 Simona Rednic
A1 Veronica Codullo
A1 Paolo Airo
A1 Yolanda Braun-Moscovici
A1 Nicolas Hunzelmann
A1 Maria Joao Salvador
A1 Valeria Riccieri
A1 Ana-Maria Gheorghiu
A1 Juan José Alegre Sancho
A1 Katarzyna Romanowska-Prochnicka
A1 Ivan Castellví
A1 Ina Kötter
A1 Marie-Elise Truchetet
A1 FJ López-Longo
A1 Pavel I Novikov
A1 Alessandro Giollo
A1 Yuichiro Shirai
A1 Laura Belloli
A1 Elisabetta Zanatta
A1 Eric Hachulla
A1 Vanessa Smith
A1 Chris Denton
A1 Ruxandra M Ionescu
A1 Tim Schmeiser
A1 Joerg H W Distler
A1 Armando Gabrielli
A1 Anna-Maria Hoffmann-Vold
A1 Masataka Kuwana
A1 Yannick Allanore
A1 Oliver Distler
A1 ,
YR 2022
UL http://rmdopen.bmj.com/content/8/2/e002477.abstract
AB 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.