Safety and efficacy of the JAK-inhibitor tofacitinib in patients with primary sclerosing cholangitis: a multicentre, retrospective study

Background/Aims We collected data on Primary Sclerosing Cholangitis (PSC) patients with associated inflammatory bowel disease (IBD) who received past or ongoing treatment with tofacitinib to assess safety and efficacy of tofacitinib on liver and bowel disease.

Method Data was collected retrospectively prior to baseline, after 3 and 12-months follow-up.

Results 42 patients with large duct PSC (69% male) were included with a median age at diagnosis of 32 years (14–61). In 47,6% treatment with tofacitinib was stopped, in 15/20 due to lack of efficacy, after a median treatment period of 7 months (1–21).

Colitis activity improved in 57,6%. Faecal calprotectin dropped from a median of 858,2 ug/g (26,7 – 2000) at baseline to 508,2 ug/g (15 – 2327,p=0.064). At follow up, one patient had developed high-grade dysplasia and one patient colorectal carcinoma.

For those still on treatment, median ALP was 150 U/l (56 – 793) at baseline and 132 U/l (47 – 558,p=0.039) at 12-months follow-up.

Overall, there was no deterioration in liver biochemistry after commencing tofacitinib. One patient had a severe infection, no event of thromboembolism was reported during treatment with tofacitinib. No new cases of hepatobiliary malignancy occurred.

Conclusion In a retrospective analysis of 42 patients with PSC and associated colitis, treatment appeared to be well tolerated and without significant worsening of liver enzymes over a median treatment period of 13 months (1–33). The majority of patients, most of them with several prior treatment regimens, demonstrated an improvement in their colitis activity.

Publication History

Article published online:
26 January 2022