Biochemical and Plasma Lipid Responses to Pemafibrate in Patients With Primary Biliary Cholangitis and Dyslipidemia: A Four-Year Analysis

Background and aims Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease treated with ursodeoxycholic acid (UDCA), though some patients respond inadequately. This study evaluated the mid-term effects of pemafibrate on liver function and lipid profiles in PBC patients with dyslipidemia who were refractory to UDCA alone or with bezafibrate. Methods A retrospective review was conducted on 25 PBC patients (17 female; median age: 71) who began treatment with pemafibrate and UDCA at Shinshu University Hospital or NHI Yodakubo Hospital in 2021. Patients were either given pemafibrate as an add-on to UDCA (n = 10) or switched from UDCA + bezafibrate (n = 15). Biochemical markers were monitored over four years. Results Median alkaline phosphatase (ALP) declined from 138 U/L to 85, 78, 82, and 77 U/L at years 1-4, respectively. ALP normalization increased from 36% to 86% over the same period (P < 0.001). Gamma-glutamyl transferase dropped from 53 to 36 U/L at one year and remained stable. Alanine aminotransferase improved similarly (26-19 U/L, P = 0.007). No significant changes were seen in aspartate aminotransferase, bilirubin, creatinine, or estimated glomerular filtration rate (eGFR). No serious adverse effects were reported. Conclusions Pemafibrate with UDCA led to sustained liver enzyme improvement and was well-tolerated in dyslipidemic PBC patients refractory to standard therapy. Prospective studies are warranted to evaluate its long-term benefits, including in patients without dyslipidemia.