Tofacitinib for Patients with Anti-TNF Refractory Ulcerative Proctitis: A Multicentre Cohort Study from the GETAID
Mathieu Uzzan, Maria Nachury, Alexandre Nuzzo, Aurélien Amiot, Bénédicte Caron, Alban Benezech, Anthony Buisson, Guillaume Bouguen, Catherine Le Berre, Cathérine Reenaers, Guillaume Le Cosquer, Guillaume Savoye, Maëva Charkaoui, Mathias Vidon, Lucas Guillo, Mathurin Fuméry, Laurent Peyrin‐Biroulet, Julien Kirchgesner, Yoram Bouhnik, GETAID TOFA-PRO study group, Mathurin Fuméry, Clara Yzet, Franck Brazier, Lore Billiauws, Yoram Bouhnik, Alexandre Nuzzo, Aurélien Amiot, Franck Carbonnel, Clément Bresteau, Antoine Meyer, Christophe Bellanger, Maria Nachury, Pauline Wils, Pierre Desreumaux, Julien Branche, Romain Gérard, Julie Assaraf, Charlotte Gagnière, Mathieu Uzzan, Laurent Beaugerie, Harry Sokol, Philippe Seksik, Anne Bourrier, Julien Kirchgesner, Paul McLellan, Guillaume Le Cosquer, Cyrielle Gilletta, Bénédicte Caron, Laurent Peyrin‐Biroulet, Thomas Guilmoteau, Marie Dodel, Dilek Çoban, Yoram Bouhnik, Mathilde Cohen, Xavier Treton, Lucas Guillo, Mélanie Serrero
Abstract
BACKGROUND: 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. METHODS: 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. RESULTS: 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]. CONCLUSION: Tofacitinib may offer a therapeutic option for patients with refractory UP.