Obeticholic acid for the treatment of primary biliary cholangitis – first data from a real world cohort

Background & aims:

Primary biliary cholangitis (PBC) is a rare chronic cholestatic liver disease that may progress to biliary cirrhosis if left untreated with the first-line therapy ursodeoxycholic acid (UDCA). Unfortunately, up to one third of PBC patients does not respond to UDCA. These patients are at risk for developing cirrhosis, complications of portal hypertension, hepatocellular carcinoma, liver transplant, or death. Recently, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved obeticholic acid (OCA) to be used in PBC patients with inadequate response to UDCA. However, data in real-life cohorts are limited. We report the first experiences of OCA treated patients in a tertiary care center.

Patients and methods:

We included all patients that were treated with OCA for at least six months since January 2017 until October 2018 at the University Hospital Heidelberg.

Results:

In total ten patients were included into final analysis. The median age at first diagnosis of PBC was 38.8 years. The median age at beginning of OCA therapy was 50.2 years. 9/10 (90.0) patients had a co-medication with UDCA. 2/10 (20.0%) patients had compensated cirrhosis.

Median laboratory values before OCA treatment were AP: 369.5 ± 109.9 U/l; GGT: 337.0 ± 229.2 U/l; bilirubin 1.2 ± 0.1 mg/dl; AST 52.5 ± 28.8 U/l; ALT 54.0 ± 22.7. 7 U/l. In three patients OCA dosing was escalated to 10 g after six months. The most frequent adverse event attributable to OCA therapy was temporary pruritus. During the study period liver function tests improved in 8/10 (80.0%). At the end of the observational period the laboratory median values were AP: 258.0 ± 24.4 U/l; GGT: 93.0 ± 57.8 U/l; bilirubin 1.0 ± 0.1 mg/dl; AST 34.0 ± 9.6 U/l; ALT 42.0 ± 9.4 U/l. OCA therapy was well tolerated in patients with compensated liver cirrhosis.

Conclusion:

Treatment with OCA in PBC patients results in improvement of laboratory values in the majority of cases. Temporary pruritus is the most common adverse event attributable to OCA therapy. Treatment of OCA is safe in patients with compensated cirrhosis in a real-world cohort.