Tofacitinib for Patients with Anti-TNF Refractory Ulcerative Proctitis: A Multicentre Cohort Study from the GETAID

• Published in: Journal of Crohn's and colitis Vol. 18; no. 3; pp. 424 - 430
• Main Authors: Uzzan, Mathieu, Nachury, Maria, Nuzzo, Alexandre, Amiot, Aurélien, Caron, Bénédicte, Benezech, Alban, Buisson, Anthony, Bouguen, Guillaume, Le Berre, Catherine, Reenaers, Catherine, Le Cosquer, Guillaume, Savoye, Guillaume, Charkaoui, Maeva, Vidon, Mathias, Guillo, Lucas, Fumery, Mathurin, Peyrin-Biroulet, Laurent, Kirchgesner, Julien, Bouhnik, Yoram
• Format: Journal Article
• Language: English
• Published: England 01.03.2024
• Subjects: Humans; Piperidines; Proctitis - drug therapy; Pyrimidines; Quality of Life; Retrospective Studies; Tumor Necrosis Factor Inhibitors - therapeutic use; Ulcerative proctitis; refractory proctitis; tofacitinib
• ISSN: 1873-9946; 1876-4479
• DOI: 10.1093/ecco-jcc/jjad169

Although ulcerative proctitis [UP] can dramatically impair quality of life, treatment efficacy has been poorly investigated in UP as it was historically excluded from phase 2/3 randomised controlled trials in ulcerative colitis. Our aim was to assess the effectiveness and safety of tofacitinib for the treatment of UP. We conducted a retrospective, multicentre study in 17 GETAID centres, including consecutive patients with UP treated with tofacitinib. The primary endpoint was steroid-free remission between Week 8 and Week 14, defined as a partial Mayo score of 2 [and no individual subscore above 1]. Secondary outcomes included clinical response and steroid-free remission after induction and at 1 year. All the 35 enrolled patients previously received anti-tumour necrosis factor [TNF] therapy and 88.6% were exposed to at least two lines of biologics. At baseline, the median partial Mayo score was 7 (intequartile range [IQR] [5.5-7]). After induction [W8-W14], 42.9% and 60.0% of patients achieved steroid-free remission and clinical response, respectively. At 1 year, the steroid-free clinical remission and clinical response rates were 39.4% and 45.5%, respectively, and 51.2% [17/33] were still receiving tofacitinib treatment. Survival without tofacitinib withdrawal was estimated at 50.4% (95% confidence interval [CI] [35.5-71.6]) at 1 year. Only a lower partial Mayo at baseline was independently associated with remission at induction (0dds ratio [OR] = 0.56 for an increase of 1, (95% CI [0.33-0.95], p = 0.03). Five [14.3%] adverse events were reported, with one leading to treatment withdrawal [septic shock secondary to cholecystitis]. Tofacitinib may offer a therapeutic option for patients with refractory UP.