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

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.

Automated summary

The study evaluated the effectiveness and safety of tofacitinib as rescue therapy in UC patients hospitalized for flare. Results showed high rates of colectomy-free survival, clinical response, and clinical remission with tofacitinib treatment. Safety profile of tofacitinib was favorable with no deaths observed and only a few adverse events reported. Further validation in controlled trials is needed to confirm the promising potential of tofacitinib in hospitalized UC patients.