Maintenance of Remission with Tofacitinib in Patients with UC – Results of the OCTAVE Open Study

Tofacitinib in the Treatment of UC

Tofacitinib is a medication classified among selective Janus kinase inhibitors (JAKi). Its efficacy and safety in patients with moderately to severely active ulcerative colitis were confirmed in three multicenter, double-blind, randomized, placebo-controlled phase III studies, leading to the registration and availability of this treatment for patients. The OCTAVE Open study, whose results were published in the journal Clinical Gastroenterology and Hepatology, demonstrated the continued efficacy in patients in remission after 52 weeks of maintenance therapy in a pivotal phase III study.  

Methodology and Objectives of the OCTAVE Open Study 

The multicenter, long-term, open-label extension phase III study OCTAVE Open included patients (n = 142) aged 18 years and older with a confirmed diagnosis of moderately to severely active ulcerative colitis who participated in the OCTAVE Sustain study and completed the 52-week maintenance therapy. Some were treated with tofacitinib at 5 mg twice daily (n = 66) and others at 10 mg twice daily (n = 76).

The presented analysis focused on patients who achieved remission in the OCTAVE Sustain study as assessed by the Mayo score (total score ≤ 2, individual score < 1, rectal bleeding subscore = 0). Remission was achieved in 163 patients, of whom 142 continued to receive tofacitinib in the OCTAVE Open study. 

OCTAVE Open evaluated treatment efficacy in terms of remission (Mayo score ≤ 2), endoscopic improvement, clinical response, and partial Mayo score (PMS) at regular intervals (at months 2, 12, 24, and 36).  

Results  

At month 12 of the study, 68.3% of patients remained in remission, with endoscopic improvement observed in 73.9%. Clinical response was observed in 77.5% of patients. By the end of the study at month 36, 50.4% of patients remained in remission and 55.3% continued to show endoscopic improvement. Clinical response was observed in 56.0% of patients. These results were achieved regardless of whether patients in the previous OCTAVE Sustain study used tofacitinib at 5 mg or 10 mg twice daily.

Sixty-seven patients (41.1%) discontinued therapy during the study, 9.2% due to insufficient clinical efficacy and 6.7% due to treatment-related adverse events. 

The study confirmed the favorable safety profile of tofacitinib – no new safety risks associated with long-term treatment were observed. The most common adverse events reported by patients were nasopharyngitis (21.7%) and worsening of UC (23.4%). 

Conclusion 

Post hoc analysis of data from the OCTAVE Open study confirmed the efficacy and safety of orally administered tofacitinib and its benefits in long-term use. The target metrics demonstrating the efficacy of tofacitinib at 5 mg twice daily were maintained for remission in patients throughout the observation period (36 months of maintenance therapy) regardless of the dosing regimen in the preceding OCTAVE Sustain study.  

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Source: Colombel J. F., Osterman M. T., Thorpe A. J. et al. Maintenance of remission with tofacitinib therapy in patients with ulcerative colitis. Clin Gastroenterol Hepatol 2022; 20 (1): 116–125.e5, doi: 10.1016/j.cgh.2020.10.004.