Pemafibrate improves liver biochemistry and GLOBE scores in patients with primary biliary cholangitis: Nationwide, multicenter study by the Japanese Red Cross Liver Study Group.

Aim: We aimed to evaluate the effect of pemafibrate, a selective peroxisome proliferator-activated receptor-α modulator, on patients with primary biliary cholangitis (PBC) complicated by dyslipidemia.

Methods: In total, 61 patients with PBC (Add-on group: 33 patients on ursodeoxycholic acid [UDCA] + pemafibrate combination therapy; Switch group: 28 patients who switched from UDCA + other fibrates to UDCA + pemafibrate combination therapy) were included in the study. Changes in aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), alkaline phosphatase (ALP), and GLOBE scores were retrospectively analyzed from 6 months before to 12 months after treatment. The POISE criteria were also used to evaluate the treatment efficacy after 12 months.

Results: After 12 months of UDCA + pemafibrate combination therapy, AST significantly decreased from 45 ± 3 to 28 ± 3 U/L (p < 0.05), ALT from 49 ± 5 to 32 ± 5 U/L (p < 0.005), GGT from 155 ± 223 to 91 ± 182 U/L (p < 0.005), and ALP from 1.4 ± 0.9 to 0.9 ± 0.8 × upper limit of normal (p < 0.0005) in all patients. ALT, GGT, and ALP levels were significantly lower after 12 months of UDCA + pemafibrate combination therapy in both the Add-on and Switch groups. After 12 months of combination therapy, the mean GLOBE score of all patients significantly decreased from 0.37 to 0.01 (p < 0.05) and the percentage of patients with a GLOBE score of 0.3 or higher decreased.

Conclusions: In patients with PBC who showed an inadequate response to prior therapy, pemafibrate add-on or switch therapy improved liver biochemistry and GLOBE scores. Pemafibrate may be useful as a second-line drug when UDCA alone is inadequate, or as an alternative after combination therapy with other fibrates.