Ursodeoxycholic acid for the treatment of hepatic sarcoid: A pre-post pilot study.

Aim of the study: Sarcoidosis is characterized by noncaseating granulomas that can affect multiple organs. Due to the lack of prospective studies regarding treatment of hepatic sarcoidosis with ursodeoxycholic acid (UDCA), we set out to evaluate its effects in a single-center, open-label, prospective, pre-post study.

Material and methods: A total of 10 patients were screened from August 2018 to July 2020; seven met the criteria and were enrolled. The study was terminated prior to achieving target enrollment of 10 patients due to the difficulty in recruitment around the COVID-19 pandemic. Most patients were women (4/7; 57.1%) and African American (5/7; 71.4%). One patient dropped out during the first month of observation due to a new diagnosis of esophageal cancer. Six completed the 6-month observation and UDCA treatment periods. One patient stopped UDCA within the first month of active treatment due to the side effect of nausea.

Results: There was a decrease in ALP and GGT after six months of UDCA treatment compared to six months of observation (ALP - 257.6 to 202.2, p = 0.23; GGT - 302.5 to 111.8, p = 0.059), but this did not reach statistical significance. There were also decreases in all key secondary endpoints (ALT - 50.8 to 29.8, p = NS; AST - 40.3 to 31.2, p = NS, VCTE kPa - 8.3 to 6.3, p = NS). As with the primary endpoints, none of the key secondary endpoints reached statistical significance.

Conclusions: There is significant potential for UDCA as first-line treatment of hepatic sarcoid. Multi-center, ideally prospective, studies of longer duration are needed.