Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort
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Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort. / 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, F J; 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; EUSTAR Collaborators.
in: RMD OPEN, Jahrgang 8, Nr. 2, e002477, 11.2022.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Effectiveness and safety of tocilizumab in patients with systemic sclerosis: a propensity score matched controlled observational study of the EUSTAR cohort
AU - Kuster, Simon
AU - Jordan, Suzana
AU - Elhai, Muriel
AU - Held, Ulrike
AU - Steigmiller, Klaus
AU - Bruni, Cosimo
AU - Cacciapaglia, Fabio
AU - Vettori, Serena
AU - Siegert, Elise
AU - Rednic, Simona
AU - Codullo, Veronica
AU - Airo, Paolo
AU - Braun-Moscovici, Yolanda
AU - Hunzelmann, Nicolas
AU - Joao Salvador, Maria
AU - Riccieri, Valeria
AU - Gheorghiu, Ana-Maria
AU - Alegre Sancho, Juan José
AU - Romanowska-Prochnicka, Katarzyna
AU - Castellví, Ivan
AU - Kötter, Ina
AU - Truchetet, Marie-Elise
AU - López-Longo, F J
AU - Novikov, Pavel I
AU - Giollo, Alessandro
AU - Shirai, Yuichiro
AU - Belloli, Laura
AU - Zanatta, Elisabetta
AU - Hachulla, Eric
AU - Smith, Vanessa
AU - Denton, Chris
AU - Ionescu, Ruxandra M
AU - Schmeiser, Tim
AU - Distler, Joerg H W
AU - Gabrielli, Armando
AU - Hoffmann-Vold, Anna-Maria
AU - Kuwana, Masataka
AU - Allanore, Yannick
AU - Distler, Oliver
AU - EUSTAR Collaborators
N1 - © Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
PY - 2022/11
Y1 - 2022/11
N2 - 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.
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.
KW - Humans
KW - United States
KW - Pulmonary Fibrosis/etiology
KW - Propensity Score
KW - Scleroderma, Systemic/complications
KW - Antibodies, Monoclonal, Humanized/adverse effects
U2 - 10.1136/rmdopen-2022-002477
DO - 10.1136/rmdopen-2022-002477
M3 - SCORING: Journal article
C2 - 36328401
VL - 8
JO - RMD OPEN
JF - RMD OPEN
SN - 2056-5933
IS - 2
M1 - e002477
ER -