Value of tofacitinib extended induction therapy in patients with moderate-to-severe ulcerative colitis: A real-world 52-week follow-up study

Abstract

BACKGROUND

Tofacitinib is an oral, selective Janus kinase inhibitor that is approved for the treatment of ulcerative colitis (UC). The 8-week induction protocol involves the administration of 10 mg twice daily (bid) with the possibility of extending the induction period to 16 weeks. The maintenance dose of tofacitinib is either 5 mg or 10 mg bid.

AIM

To assess predictors for clinical remission and drug persistence in patients with UC receiving the extended induction tofacitinib protocol.

METHODS

This was a real-world multicenter retrospective study in patients with moderate-to-severe UC. Patients received physician-directed extended induction tofacitinib treatment. We collected clinical and demographic data at baseline and data regarding clinical, laboratory, and endoscopic evaluations, therapeutic modifications, and adverse events at the 52-week follow-up. Possible predictors for clinical remission at week 52 was the primary endpoint. Differences between patients receiving 5 mg bid vs 10 mg bid at week 52 and identification of predictors for treatment persistence were secondary endpoints.

RESULTS

Thirty-seven consecutive patients from 11 medical centers were included [51.4% males with median age 39 (17-64) years]. Twenty-eight patients continued treatment until week 52 (75.7%) with 67.9% receiving 10 mg tofacitinib; all had prior history of biologic use. We observed that 57.1% of patients achieved clinical remission (66.7% in the 5 mg tofacitinib group and 52.6% in the 10 mg tofacitinib group, P = 0.483). De-escalation to 5 mg tofacitinib was attempted in 17 patients with a success rate of 52.9%. Prior biologic use was significantly more frequent in patients treated with 10 mg tofacitinib. Active smoking was significantly associated with treatment discontinuation at week 52. We identified eight adverse events, and only one led to treatment discontinuation.

CONCLUSION

Our results supported the extended induction strategy with tofacitinib in selected patients with UC. Patients with prior failure of advanced therapies particularly benefitted, highlighting the importance of personalized maintenance regimens.

Keywords: Ulcerative colitis; Tofacitinib; Extended induction; Predictors; Efficacy; Safety; Real-world study

Core Tip: This multicenter, retrospective cohort study evaluated the effectiveness and safety of administering a 16-week extended induction protocol for tofacitinib in patients with moderate-to-severe ulcerative colitis. At week 52 over half of the patients achieved clinical remission with a drug persistence rate of 75.7%. Notably, prior exposure to biologics was associated with the need for continuous administration of escalated doses while active smoking was significantly associated with treatment discontinuation. Extended induction therapy in selected patients may be beneficial. Our study highlights the need for further research to identify predictors of long-term response and optimal dosing strategies.