Tofacitinib for the treatment of chronic pouchitis: A pilot study

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.

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).

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.

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.