Post-marketing surveillance of tofacitinib in patients with ulcerative colitis in Japan: a final report of safety and effectiveness data.

BACKGROUND

We present the final analysis of a tofacitinib post-marketing surveillance (PMS) study in Japanese patients with ulcerative colitis (UC).

METHODS

Safety/effectiveness data were evaluated (through Sept/30/2022). All patients with UC in Japan receiving tofacitinib were registered (60-week observation period). Adverse events (AEs) were recorded. Per protocol, several AEs were identified as clinically important/potential risks; all treatment-period data were used to calculate incidence rates (IRs; unique patients with events/100 patient-years [PY] of exposure). Effectiveness was assessed (partial/total Mayo score), with last observation carried forward for imputation of missing data.

RESULTS

Overall, 2043 patients were enrolled (safety analysis set: n = 1982/effectiveness analysis set: n = 1969). Data were excluded for 13 patients from two hospitals from which consent was not obtained for publication and which, therefore, were not permitted for publication. AEs and serious AEs were observed in 33.4% and 5.2% of patients, respectively; one death occurred (intestinal abscess). Herpes zoster (HZ; non-serious and serious) was the most reported infection (n = 92 [IR 5.93/100 PY, 95% confidence interval 4.78, 7.27]). Serious infection, malignancy, cardiovascular and venous thromboembolic events IRs were 1.51/100 PY, 0.62/100 PY, 0.13/100 PY, and 0.31/100 PY, respectively. Overall, 52.4% of patients discontinued treatment, mostly due to inadequate clinical response (48.9%). At Week 60, 1151/1969 patients (58.5%) achieved partial Mayo score remission.

CONCLUSION

The overall safety profile was generally comparable with tofacitinib data from PMS reports from Japan, worldwide and the tofacitinib UC clinical program. However, HZ IR was higher than in the tofacitinib UC clinical program. Tofacitinib effectiveness was consistent with data from the tofacitinib UC clinical program.

CLINICALTRIALS: GOV: NCT03643211.