Real-world Cohort Study on the Effectiveness and Safety of Filgotinib Use in Ulcerative Colitis

Abstract

Abstract Background Filgotinib is a small molecule with preferential inhibition of Janus kinase type 1, approved for the treatment of ulcerative colitis in Scotland in May 2022. We present the first real-world experience on its use in clinical practice. Methods In this retrospective, observational, cohort study we assessed patients with active ulcerative colitis who received filgotinib in NHS Lothian, Scotland. Baseline demographic, phenotype, and follow-up data were collected via review of electronic medical records. Results We included 91 patients with median treatment duration of 39 weeks (interquartile range [IQR] 23-49). Among the cohort, 67% [61/91] were biologic- and small molecule-naïve, and 20.9% [19/91] had failed one and 12.1% [11/91]  two or more classes of advanced therapy. Of the biologic- and small molecule-naïve patients, 18% [11/61] were also thiopurine-naïve. Clinical remission [partial Mayo score <2] was achieved in 71.9% [41/57] and 76.4% [42/55] of patients at Weeks 12 and 24 respectively. Biochemical remission [C reactive protein ≤5 mg/L] was achieved in 87.3% [62/71] at Week 12 and 88.9% [40/45] at Week 24. Faecal biomarker [calprotectin <250 µg/g] remission was achieved in 82.8% [48/58] at Week 12 and 79.5% [35/44] at Week 24. At the end of follow-up, median 42 weeks [IQR 27-50], 82.4% [75/91] of patients remained on filgotinib. Severe adverse events leading to drug discontinuation occurred in 2.2% [2/91] and there were 8.8% [8/91] moderate adverse events that required temporary discontinuation. Conclusion These are the first reported data on the real-world efficacy and safety of filgotinib in ulcerative colitis. Our findings demonstrate that filgotinib is an effective and low-risk treatment option for these patients.