Effectiveness and safety of tocilizumab in patients with systemic sclerosis : a propensity score matched controlled observational study of the EUSTAR cohort
Kuster, Simon (Department of Rheumatology. University Hospital Zurich. University of Zurich)
Jordan, Suzana (Department of Rheumatology. University Hospital Zurich. University of Zurich)
Elhai, Muriel (Department of Rheumatology. Paris Descartes University. Cochin Hospital. Aphp)
Held, Ulrike (Department of Biostatistics at Epidemiology. Biostatistics and Prevention Institute. University of Zurich)
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 (Rheumatology Unit. Deto. University Hospital Polyclinic of Bari)
Vettori, Serena (Department of Precision Medicine. University of Campania Luigi Vanvitelli)
Siegert, Elise (Berlin Institute of Health at Charité)
Rednic, Simona (Clinica Reumatologie. University of Medicine and Pharmacy)
Codullo, Veronica (Department of Rheumatology. Irccs Foundation Policlinico San Matteo)
Airò, Paolo (Uoc Rheumatology and Clinical Immunology. Spedali Civili di Brescia)
Braun-Moscovici, Yolanda (B. Shine Department of Rheumatology. Rambam Health Care Campus and Technion)
Hunzelmann, Nicolas (University of Cologne)
Joao Salvador, Maria (Department of Rheumatology. Centro Hospitalar e Universitário de Coimbra)
Riccieri, Valeria (Department of Clinical Internal. Anesthesiological and Cardiovascular Sciences. Sapienza University of Rome)
Gheorghiu, Ana-Maria (Department of Internal Medicine and Rheumatology. Cantacuzino Hospital. Carol Davila University of Medicine and Pharmacy)
Alegre-Sancho, Juan José (Hospital Doctor Peset. Servicio de Reumatología)
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. (Department of Rheumatology. National Reference Center for Systemic Autoimmune Rare Diseases. Hopital Pellegrin)
Novikov, Pavel I. (Department of Rheumatology. National Reference Center for Systemic Autoimmune Rare Diseases. Hopital Pellegrin)
Giollo, Alessandro (Rheumatology Section. Department of Medicine. University of Verona)
Shirai, Yuichiro (Department of Allergy and Rheumatology. Nippon Medical School)
Belloli, Laura (Rheumatology Unit. Asst Grande Ospedale Metropolitano Niguarda)
Zanatta, Elisabetta (Rheumatology Unit. Department of Clinical and Experimental Medicine. University of Padova)
Hachulla, Eric (Université de Lille)
Smith, Vanessa (Unit for Molecular Immunology and Inflammation. Vib Inflammation Research Centre (IRC))
Denton, Christopher P. (Centre for Rheumatology. University College London Medical School. Royal Free Campus)
Ionescu, Ruxandra M. (Internal Medicine and Rheumatology Department. St. Maria Hospital. Carol Davila University of Medicine and Pharmacy)
Schmeiser, Tim (Krankenhaus St. Josef)
Distler, Joerg H.W. (Department of Internal Medicine Iii. Erlangen University Hospital)
Gabrielli, Armando (Dipartimento di Scienze Cliniche e Molecolari. Università Politecnica Delle Marche)
Hoffmann-Vold, Anna-Maria (Department of Rheumatology. Rikshospitalet University Hospital Oslo)
Kuwana, Masataka (Nippon Medical School Graduate School of Medicine)
Allanore, Yannick (Department of Rheumatology. Paris Descartes University. Cochin Hospital. Aphp)
Distler, Oliver (Department of Rheumatology. University Hospital Zurich. University of Zurich)

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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