Kobayashi Hiroyuki, Sugiura Ayumi, Koyama-Otagiri Mizuki, Nimura Takayuki, Kurasawa Yukifumi, Shirakawa Haruaki, Joshita Satoru
Gastroenterology, Shinshu University School of Medicine, Matsumoto, JPN.
Internal Medicine, Sato Hospital, Nakano, JPN.
Cureus. 2025 Apr 29;17(4):e83176. doi: 10.7759/cureus.83176. eCollection 2025 Apr.
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.
背景与目的 原发性胆汁性胆管炎（PBC）是一种慢性自身免疫性肝病，采用熊去氧胆酸（UDCA）治疗，不过部分患者反应欠佳。本研究评估了匹伐他汀对单用UDCA或联合苯扎贝特治疗效果不佳的血脂异常PBC患者的肝功能和血脂谱的中期影响。方法 对2021年在信州大学医院或日本国立医院机构淀川医院开始接受匹伐他汀和UDCA治疗的25例PBC患者（17例女性；中位年龄：71岁）进行回顾性分析。患者分为两组，一组在UDCA基础上加用匹伐他汀（n = 10），另一组从UDCA + 苯扎贝特转换为匹伐他汀治疗（n = 15）。对生化指标进行了4年的监测。结果 中位碱性磷酸酶（ALP）在第1 - 4年分别从138 U/L降至85、78、82和77 U/L。同期ALP正常化比例从36%增至86%（P < 0.001）。γ-谷氨酰转移酶在1年时从53降至36 U/L并保持稳定。丙氨酸氨基转移酶也有类似改善（从26降至19 U/L，P = 0.007）。天门冬氨酸氨基转移酶、胆红素、肌酐或估算肾小球滤过率（eGFR）未见显著变化。未报告严重不良反应。结论 匹伐他汀联合UDCA可使肝酶持续改善，且在标准治疗无效的血脂异常PBC患者中耐受性良好。有必要开展前瞻性研究以评估其长期获益，包括在无血脂异常患者中的获益情况。
