Abstract
BACKGROUND & AIMS: Up to 15% of ulcerative colitis (UC) patients with an ileo-anal pouch will develop chronic or antibiotic refractory pouchitis. We aimed to evaluate the efficacy and safety of tofacitinib in these refractory patients. METHODS: In this prospective single center study, adult UC patients with chronic or antibiotic refractory pouchitis (pouchitis disease activity index [PDAI] ≥ 7) were treated with tofacitinib 10 mg twice daily for 8 weeks. Clinical, biochemical, endoscopic and histologic disease activity was assessed at baseline and at week 8. The primary endpoint was the proportion of patients achieving clinical remission (PDAI < 7 and a reduction of ≥3 points from baseline) or clinical response (decrease ≥2 points from baseline). RESULTS: Thirteen patients were included and treated with tofacitinib. After 8 weeks, 31% achieved PDAI-defined remission (4/13) and 54% achieved response (7/13). Both the total PDAI score (11 [interquartile range 9-12.75] vs. 8 [4.5-9.75], p = 0.033) and the clinical PDAI subscore (4 [3-4] vs. 2 [0.25-3.75], p = 0.014) decreased significantly from baseline compared to week 8 or early withdrawal, respectively. We did not observe a change in endoscopic or histological PDAI subscores. CONCLUSION: In this pilot study, clinical remission in patients with chronic pouchitis was achieved in 31% of patients after 8 weeks of treatment with tofacitinib. Total and clinical PDAI dropped significantly compared with baseline, but we did not observe a significant change in endoscopic or histologic disease activity. Presumably, a treatment duration of 8 weeks is insufficient to induce mucosal healing in these refractory patients.