Post-marketing Surveillance of Tofacitinib in Patients With Ulcerative Colitis in Japan: A Post Hoc Analysis of Safety and Effectiveness by Prior Biologic Status

Background: This post hoc analysis of a 60-week post-marketing surveillance (PMS) study in Japan assessed tofacitinib safety and effectiveness in patients with ulcerative colitis (UC), stratified by prior biologic exposure. Methods: The PMS study registered all patients with UC receiving tofacitinib in Japan (May 2018-June 2021). Outcomes included the proportion of patients with clinically important adverse events (AEs), incidence rates (IR; unique patients with events/100 patient-years of exposure) of clinically important AEs, reasons for discontinuation, and effectiveness (partial Mayo score remission). Patients were stratified by prior biologic exposure (biologic-experienced/biologic-naïve). Prior biologics included infliximab, adalimumab, golimumab, and others (eg, ustekinumab/vedolizumab). Results: Overall, 1367 biologic-experienced and 615 biologic-naïve patients were included. More biologic-naïve versus biologic-experienced patients (30.6% vs. 16.9%) had disease duration <2 years. Proportions of AEs were similar between groups, although IRs (95% confidence intervals) were numerically higher in biologic-experienced versus biologic-naïve patients for herpes zoster (HZ; 6.77 [5.30, 8.53] vs. 4.10 [2.50, 6.33]) and serious infections (SI; 1.92 [1.19, 2.94] vs. 0.60 [0.12, 1.77]). Insufficient clinical response (biologic-experienced: 53.3%; biologic-naïve: 38.9%) was the primary reason for discontinuation. At week 60, remission rates were 63.07% for biologic-naïve patients and 56.37% for biologic-experienced patients. Conclusions: Safety was generally similar between biologic-experienced and biologic-naïve patients, although HZ and SI IRs were numerically higher in biologic-experienced patients. Remission rates were numerically higher in biologic-naïve versus biologic-experienced patients, although differences were small. Discontinuation was mostly due to insufficient clinical response. Missing concomitant corticosteroid/tofacitinib dose data and endoscopic assessment might limit the analysis. ClinicalTrials.gov NCT03643211.