Liao Wanying, Fu Siyang, Yang Aiming, Yang Yingyun
Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China.
Therap Adv Gastroenterol. 2025 Sep 3;18:17562848251370111. doi: 10.1177/17562848251370111. eCollection 2025.
Managing patients with primary biliary cholangitis (PBC) who demonstrate an inadequate response to ursodeoxycholic acid or experience intolerable side effects remains a significant clinical challenge.
This study aims to investigate the efficacy and safety of peroxisome proliferator-activated receptor (PPAR) agonists in the treatment of PBC.
Meta-analysis and systematic review.
A systematic search of publications in PubMed, Embase, Web of Science, and the Cochrane Central Register of Controlled Trials was performed. Randomized controlled trials published in English that involved the treatment of PPAR agonists and reported on the levels of alkaline phosphatase (ALP), biochemical response rates, pruritus score, or severe and serious adverse events (AEs) were selected. The primary outcomes assessed were the effects of PPAR agonists on ALP levels and biochemical response rates. Secondary outcomes included the rates of severe or serious AEs and relief of pruritus.
Fourteen studies with 1137 patients were included. Compared to the control group, PPAR agonists significantly reduced ALP levels by a mean difference of -155.87 U/L (95% confidence interval (CI): -208.30 to -103.44; random-effects). Patients who received PPAR agonists showed a significantly higher biochemical response rate (risk ratio (RR), 4.42; 95% CI: 2.37-8.26; random-effects). Furthermore, there was no significant difference in the rate of severe (RR, 1.05; 95% CI, 0.49-2.28) or serious AEs (RR, 1.02; 95% CI, 0.65-1.60) between the PPAR agonists and placebo groups.
PPAR agonists are effective and safe to treat patients with PBC.
CRD42024545743.
对于原发性胆汁性胆管炎（PBC）患者，若对熊去氧胆酸反应不佳或出现难以耐受的副作用，其管理仍是一项重大的临床挑战。
本研究旨在探讨过氧化物酶体增殖物激活受体（PPAR）激动剂治疗PBC的疗效和安全性。
荟萃分析和系统评价。
对PubMed、Embase、Web of Science和Cochrane对照试验中央注册库中的出版物进行系统检索。选择以英文发表的随机对照试验，这些试验涉及PPAR激动剂治疗，并报告了碱性磷酸酶（ALP）水平、生化反应率、瘙痒评分或严重不良事件（AE）。评估的主要结局是PPAR激动剂对ALP水平和生化反应率的影响。次要结局包括严重或严重AE的发生率和瘙痒缓解情况。
纳入了14项研究，共1137例患者。与对照组相比，PPAR激动剂使ALP水平显著降低，平均差值为-155.87 U/L（95%置信区间（CI）：-208.30至-103.44；随机效应）。接受PPAR激动剂治疗的患者生化反应率显著更高（风险比（RR），4.42；95% CI：2.37 - 8.26；随机效应）。此外，PPAR激动剂组与安慰剂组在严重（RR，1.05；95% CI，0.49 - 2.28）或严重AE（RR，1.02；95% CI，0.65 - 1.60）发生率上无显著差异。
PPAR激动剂治疗PBC患者有效且安全。
PROSPERO试验注册号：CRD42024545743。
