Effectiveness and safety of tocilizumab in patients with systemic sclerosis : a propensity score matched controlled observational study of the EUSTAR cohort
Kuster, Simon (Universitätsspital Zürich)
Jordan, Suzana (Universitätsspital Zürich)
Elhai, Muriel (Université Paris Cité)
Held, Ulrike (Universitätsspital Zürich)
Steigmiller, Klaus (Department of Biostatistics at Epidemiology. Biostatistics and Prevention Institute. University of Zurich)
Bruni, Cosimo (Department of Experimental and Clinical Medicine. Division of Rheumatology. University of Florence)
Cacciapaglia, Fabio (University Hospital Polyclinic of Bari)
Vettori, Serena (University of Campania Luigi Vanvitelli)
Siegert, Elise (Berlin Institute of Health at Charité)
Rednic, Simona (Clinica Reumatologie. University of Medicine and Pharmacy)
Codullo, Veronica (Fondazione IRCCS Policlinico San Matteo (Itàlia))
Airò, Paolo (Azienda Ospedaliera Spedali Civili di Brescia (Brescia, Itàlia))
Braun-Moscovici, Yolanda (B. Shine Department of Rheumatology. Rambam Health Care Campus and Technion)
Hunzelmann, Nicolas (University of Cologne)
Joao Salvador, Maria (Centro Hospitalar e Universitário de Coimbra (Coimbra, Portugal))
Riccieri, Valeria (Università degli Studi di Roma "La Sapienza")
Gheorghiu, Ana-Maria (Dr. Carol Davila University of Medicine and Pharmacy (Bucarest, Romania))
Alegre-Sancho, Juan José (Hospital Universitari Doctor Peset (València))
Romanowska-Prochnicka, Katarzyna (Department of Connective Tissues Diseases. Institute of Rheumatology)
Castellvi, Ivan (Institut d'Investigació Biomèdica Sant Pau)
Kötter, Ina (University Hospital Hamburg-Eppendorf. Division of Rheumatology and Systemic Inflammatory Diseases)
Truchetet, Marie-Elise (Hospital General Universitario Gregorio Marañón)
López-Longo, F.J. (Hopital Pellegrin)
Novikov, Pavel I. (Hopital Pellegrin)
Giollo, Alessandro (University of Verona)
Shirai, Yuichiro (Nippon Medical School)
Belloli, Laura (ASST Grande Ospedale Metropolitano Niguarda)
Zanatta, Elisabetta (Università degli Studi di Padova)
Hachulla, E. (Université de Lille)
Smith, Vanessa (Vib Inflammation Research Centre (IRC))
Denton, Christopher P. (University College London Medical School)
Ionescu, Ruxandra M. (Dr. Carol Davila University of Medicine and Pharmacy (Bucarest, Romania))
Schmeiser, Tim (Krankenhaus St. Josef)
Distler, Joerg H.W. (Erlangen University Hospital)
Gabrielli, Armando (Università Politecnica Delle Marche)
Hoffmann-Vold, A.M. (Rikshospitalet University Hospital Oslo)
Kuwana, Masataka (Nippon Medical School Graduate School of Medicine)
Allanore, Yannick (Université Paris Cité)
Distler, Oliver (Universitätsspital Zürich)

Date:	2022
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.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Published in:	RMD Open, Vol. 8 Núm. 2 (march 2022) , p. e002477, ISSN 2056-5933

DOI: 10.1136/rmdopen-2022-002477
PMID: 36328401

11 p, 784.4 KB

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut de Recerca Sant Pau
Articles > Research articles
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