Alimentary Tract| Volume 52, ISSUE 3, P268-273, March 2020

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Real-world evidence of tofacitinib effectiveness and safety in patients with refractory ulcerative colitis

Published:November 12, 2019DOI:



      Phase III trials demonstrated effectiveness of tofacitinib, an oral Janus kinase inhibitor, to induce and maintain remission in patients with moderate-to-severe active ulcerative colitis (UC).


      We report the real-world effectiveness and safety of tofacitinib in patients with UC in France.


      From February 2017 to December 2018, we performed a national French cohort study, which included all consecutive patients with an active UC refractory to anti-TNF and vedolizumab, who received tofacitinib. Outcomes were survival without colectomy, survival without tofacitinib discontinuation and steroid-free clinical remission at weeks 14, 24 and 48.


      Thirty-eight patients were included, with a median follow-up of 41.5 (18.5–56.8) weeks. Survival without colectomy was 77% [95% confidence interval (95%CI): 59.3–87.9] at week 24 and 70% (95%CI: 50.9–82.8) at week 48. Survival without treatment discontinuation was 70% (95%CI: 52.6–82.3) at week 24. Steroid-free clinical remission was observed in 13 (34%) patients at week 48. Adverse events occurred in 14 (37%) patients, including 6 severe adverse events and three herpes zoster infections.


      In a highly refractory UC population, one third of patients treated with tofacitinib achieved steroid-free clinical remission at week 14 and 70% of patients avoided colectomy at one year, with an acceptable safety profile. These data confirm tofacitinib effectiveness in UC, especially after multiple biologic failures.


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        • Fumery M.
        • Singh S.
        • Dulai P.S.
        • et al.
        Natural history of adult ulcerative colitis in population-based cohorts: a systematic review.
        Clin Gastroenterol Hepatol. 2018; 16 (343-356.e3)
        • Frolkis A.D.
        • Dykeman J.
        • Negrón M.E.
        • et al.
        Risk of surgery for inflammatory bowel diseases has decreased over time: a systematic review and meta-analysis of population-based studies.
        Gastroenterology. 2013; 145: 996-1006
        • Amiot A.
        • Serrero M.
        • Peyrin-Biroulet L.
        • et al.
        One-year effectiveness and safety of vedolizumab therapy for inflammatory bowel disease: a prospective multicentre cohort study.
        Aliment Pharmacol Ther. 2017; 46: 310-321
        • Sandborn W.J.
        • Panés J.
        • D’Haens G.R.
        • et al.
        Safety of tofacitinib for treatment of ulcerative colitis, based on 4.4 years of data from global clinical trials.
        Clin Gastroenterol Hepatol. 2018;
        • Sandborn W.J.
        • Ghosh S.
        • Panes J.
        • et al.
        Tofacitinib, an oral Janus kinase inhibitor, in active ulcerative colitis.
        N Engl J Med. 2012; 367: 616-624
        • Sandborn W.J.
        • Su C.
        • Sands B.E.
        • et al.
        Tofacitinib as induction and maintenance therapy for ulcerative colitis.
        N Engl J Med. 2017; 376: 1723-1736
        • Sturm A.
        • Maaser C.
        • Calabrese E.
        • et al.
        ECCO-ESGAR guideline for diagnostic assessment in IBD part 2: IBD scores and general principles and technical aspects.
        J Crohns Colitis. 2019; 13: 273-284
        • D’Haens G.
        • Sandborn W.J.
        • Feagan B.G.
        • et al.
        A review of activity indices and efficacy end points for clinical trials of medical therapy in adults with ulcerative colitis.
        Gastroenterology. 2007; 132: 763-786
        • Satsangi J.
        • Silverberg M.S.
        • Vermeire S.
        • et al.
        The Montreal classification of inflammatory bowel disease: controversies, consensus, and implications.
        Gut. 2006; 55: 749-753
        • Travis S.P.L.
        • Schnell D.
        • Krzeski P.
        • et al.
        Reliability and initial validation of the ulcerative colitis endoscopic index of severity.
        Gastroenterology. 2013; 145: 987-995
        • Higgins P.D.R.
        • Schwartz M.
        • Mapili J.
        • et al.
        Patient defined dichotomous end points for remission and clinical improvement in ulcerative colitis.
        Gut. 2005; 54: 782-788
        • Schroeder K.W.
        • Tremaine W.J.
        • Ilstrup D.M.
        Coated oral 5-aminosalicylic acid therapy for mildly to moderately active ulcerative colitis. A randomized study.
        N Engl J Med. 1987; 317: 1625-1629
        • Lewis J.D.
        • Chuai S.
        • Nessel L.
        • et al.
        Use of the noninvasive components of the Mayo score to assess clinical response in ulcerative colitis.
        Inflamm Bowel Dis. 2008; 14: 1660-1666
        • Turner D.
        • Seow C.H.
        • Greenberg G.R.
        • et al.
        A systematic prospective comparison of noninvasive disease activity indices in ulcerative colitis.
        Clin Gastroenterol Hepatol. 2009; 7: 1081-1088
        • Vuitton L.
        • Peyrin-Biroulet L.
        • Colombel J.F.
        • et al.
        Defining endoscopic response and remission in ulcerative colitis clinical trials: an international consensus.
        Aliment Pharmacol Ther. 2017; 45: 801-813
        • Niewiadomski O.
        • Studd C.
        • Hair C.
        • et al.
        Prospective population-based cohort of inflammatory bowel disease in the biologics era: disease course and predictors of severity.
        J Gastroenterol Hepatol. 2015; 30: 1346-1353
        • Weisshof R.
        • Aharoni Golan M.
        • Sossenheimer P.H.
        • et al.
        Real-world experience with tofacitinib in IBD at a tertiary center.
        Dig Dis Sci. 2019;
        • Paschos P.
        • Katsoula A.
        • Salanti G.
        • et al.
        Systematic review with network meta-analysis: the impact of medical interventions for moderate-to-severe ulcerative colitis on health-related quality of life.
        Aliment Pharmacol Ther. 2018; 48: 1174-1185
        • Singh S.
        • Fumery M.
        • Sandborn W.J.
        • et al.
        Systematic review with network meta-analysis: first- and second-line pharmacotherapy for moderate-severe ulcerative colitis.
        Aliment Pharmacol Ther. 2018; 47: 162-175
        • Bonovas S.
        • Lytras T.
        • Nikolopoulos G.
        • et al.
        Systematic review with network meta-analysis: comparative assessment of tofacitinib and biological therapies for moderate-to-severe ulcerative colitis.
        Aliment Pharmacol Ther. 2018; 47: 454-465