Real-world evidence of tofacinitib in ulcerative colitis: short and long-term effectiveness and safety

Am J Gastroenterol. 2022 Dec 14. doi: 10.14309/ajg.0000000000002145. Online ahead of print.


Aims: To assess the durability, short and long-term effectiveness and safety of tofacitinib in ulcerative colitis (UC) in clinical practice.

Methods: Retrospective, multicentre study including UC patients who had received the first tofacitinib dose at least 8 weeks before the inclusion. Clinical effectiveness was based on Partial Mayo Score (PMS).

Results: A total of 408 patients were included. Of them, 184 (45%) withdrew tofacitinib during follow-up (mean=18 months). The probability of maintaining tofacitinib was 67% at 6 m, 58% at 12 m, and 49% at 24 m. Main reason for tofacitinib withdrawal was primary non-response (44%). Older age at the start of tofacitinib and higher severity of clinical activity were associated with tofacitinib withdrawal. The proportion of patients in remission was 38% at week 4, 45% at week 8, and 47% at week 16. Having moderate-severe vs. mild disease activity at baseline and older age at tofacitinib start were associated with lower and higher likelihood of remission at week 8, respectively. Of 171 patients in remission at week 8, 83 (49%) relapsed. The probability of maintaining response was 66% at 6 m and 54% at 12 m. There were 93 adverse events related to tofacitinib treatment [including 2 pulmonary thromboembolisms (in patients with risk factors) and 2 peripheral vascular thrombosis], and 29 led to tofacitinib discontinuation.

Conclusions: Tofacitinib is effective both in the short and in the long-term in patients with UC. The safety profile is similar to that previously reported.