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

Abstract 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.