Tofacitinib as salvage therapy for 55 patients hospitalised with refractory severe ulcerative colitis: A GETAID cohort

Abstract

Summary Background Up to 25% of patients with ulcerative colitis (UC) will require hospitalization for severe flare. In patients hospitalised for severe flare, who previously experienced multiple drug failures, including steroids and anti‐TNF agents, new quick‐acting medical options are needed. Tofacitinib is effective in refractory UC and has a rapid onset of action. Aim To evaluate effectiveness and safety of tofacitinib as rescue therapy in patients hospitalised for UC flare. Methods We conducted an observational and multicentre study with both retrospective and prospective collections in 14 GETAID centres. The primary objective was to assess the survival without colectomy following tofacitinib initiation in patients hospitalised for a UC flare. We determined rates of clinical response, clinical remission, and steroid‐free clinical remission at week 6 and week 14 and safety. Results Fifty‐five patients were included (49 with prior infliximab failure and 19 previously exposed to ciclosporin). With a median follow‐up of 6.5 months (interquartile range [IQR] [3‐12.3]), rate of colectomy‐free survival was estimated at 78.9% (95 CI [68.5‐90.9]) and 73.6% (95 CI [61.9‐87.3]) at 3 and 6 months, respectively. Rates of clinical response, clinical remission and steroid‐free clinical remission were 60%, 45.5% and 37.5% at week 6 and 41.8%, 34.5% and 32.7% at week 14. Regarding safety, no death was observed, three patients withdrew tofacitinib due to adverse events. Two herpes zoster infections occurred in patients aged over 60 years old. No venous thrombotic or major adverse cardiovascular events occurred. Conclusion Tofacitinib appears as a promising option in patients hospitalised with a UC flare but needs further validation in controlled trials.

Korean clinical practice guidelines on biologics and small molecules for moderate-to-severe ulcerative colitis

“…159 In the GETAID cohort in France, salvage therapy with tofacitinib (10 mg twice daily) was evaluated in 55 hospitalized patients who had refractory ASUC (median follow-up of 6.5 months). 160 The incidence of colectomy at month 3 was estimated to be 21.1% in the GE-TAID cohort, which is comparable with that in patients treated with infliximab or cyclosporine in the CYSIF trial. However, data concerning the efficacy of tofacitinib in ASUC patients are very limited and the level of evidence is very low.…”

“…Post-hoc analysis of OCTAVE 1 and 2 suggested that stool frequency and rectal bleeding may decrease in about one-third of moderate-to-severe UC patients within 3 days of therapy commencement [ 159 ]. In the GETAID cohort in France, salvage therapy with tofacitinib (10 mg twice daily) was evaluated in 55 hospitalized patients who had refractory ASUC (median follow-up of 6.5 months) [ 160 ]. The incidence of colectomy at month 3 was estimated to be 21.1% in the GETAID cohort, which is comparable with that in patients treated with infliximab or cyclosporine in the CYSIF trial.…”

Tofacitinib to Treat Severe Acute Refractory Colitis in a Teenager: Case Report and Review of the Literature

“…In 2021, the GETAID-TALC Study Group published a multicentric study of 55 adult patients with severe refractory UC (median follow-up of 6.5 mo) treated by tofacitinib as a rescue therapy. At week 14, 33% of patients were in steroid-free clinical remission, and at 6 months, the rate of colectomy-free survival was 73.6% (95 confidence interval [CI], 61.9%–87.3%) ( 28 ). Berinstein et al ( 29 ) published a study of 40 adult patients with ASC treated with tofacitinib and concomitant IVCS.…”

Management of inflammatory bowel disease beyond tumor necrosis factor inhibitors: novel biologics and small-molecule drugs

“…Post hoc analyses of the OCTAVE1 and 2 results showed that stool frequency and rectal bleeding decreased in approxi-mately one-third of patients with moderate-to-severe UC within 3 days [71]. In the GETAID cohort in France, salvage therapy with 10 mg tofacitinib twice daily was evaluated in 55 patients with refractory acute severe UC [72]. The incidence of colectomy at 3 months in the GETAID cohort was comparable with that in the infliximab or cyclosporine treatment groups.…”