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Purpose <p>Evaluate clinical efficacy of ursodeoxycholic acid (UDCA) in biliary atresia (BA) infants post-Kasai portoenterostomy (KPE) using real-world data.</p> Methods <p>Retrospective analysis of 698 BA patients from eight Chinese pediatric centers (2020–2025). Age-stratified analyses used propensity score matching (PSM) and inverse probability treatment weighting (IPTW) to adjust baseline differences. Primary outcomes were UDCA utilization and effects on bilirubin (TBil, DBil) and total bile acid (TBA) levels.</p> Results <p>The median follow-up duration for the cohort was 49&#xa0;months (IQR: 25–74), with male patients accounting for 54.2% (379/698). The median age at surgery was 59&#xa0;days (IQR: 46–75). A history of cholangitis was present in 53.1% of patients (371 cases), and 63.5% (443 cases) underwent open Kasai portoenterostomy. After PSM/IPTW adjustment, UDCA within 12&#xa0;months significantly reduced TBil, DBil, and TBA. Initiation before 12&#xa0;months reduced TBil-elevated (TBil &gt; 20&#xa0;μmol/L) odds by 80% (OR = 0.20, 95% CI 0.05–0.72) and hyperbilirubinemia (TBil &gt; 34&#xa0;μmol/L) by 86% (OR = 0.14, 95% CI 0.03–0.50). Low-dose UDCA (≤ 10&#xa0;mg/kg/day) within 12&#xa0;months provided optimal bilirubin/hepatic improvement without dose-dependence. UDCA beyond 36&#xa0;months nearly doubled TBA levels, potentially reflecting dose-dependent TBA elevation.</p> Conclusion <p>UDCA efficacy post-KPE is age-dependent. Low-dose therapy (≤ 10&#xa0;mg/kg/day) within 12&#xa0;months significantly improves jaundice and liver function, with limited benefits at 12–36&#xa0;months. Use beyond 36&#xa0;months may be harmful. Optimal timing/dosing requires prospective validation.</p>

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Dosage and duration of ursodeoxycholic acid therapy Post-Kasai portoenterostomy in biliary atresia: a multicenter cross-sectional study with age stratification

  • 期刊论文

  • 发表日期：

    2025年8月30日

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  • Yu Meng,
  • Shaowen Liu,
  • Qianhui Yang,
  • Yilin Zhao,
  • Tengfei Li,
  • Jiaying Liu,
  • Xin Li,
  • Yuqiang Chen,
  • YanRan Zhang,
  • Ji Qi,
  • Xiaoxia Wu,
  • Pu Yu,
  • Xueqiang Yan,
  • Bin Wang,
  • Zhibo Zhang,
  • Xianwei Zhang,
  • Tiquan Yang,
  • Wei Gao,
  • Jianghua Zhan

摘要

Purpose

Evaluate clinical efficacy of ursodeoxycholic acid (UDCA) in biliary atresia (BA) infants post-Kasai portoenterostomy (KPE) using real-world data.

Methods

Retrospective analysis of 698 BA patients from eight Chinese pediatric centers (2020–2025). Age-stratified analyses used propensity score matching (PSM) and inverse probability treatment weighting (IPTW) to adjust baseline differences. Primary outcomes were UDCA utilization and effects on bilirubin (TBil, DBil) and total bile acid (TBA) levels.

Results

The median follow-up duration for the cohort was 49 months (IQR: 25–74), with male patients accounting for 54.2% (379/698). The median age at surgery was 59 days (IQR: 46–75). A history of cholangitis was present in 53.1% of patients (371 cases), and 63.5% (443 cases) underwent open Kasai portoenterostomy. After PSM/IPTW adjustment, UDCA within 12 months significantly reduced TBil, DBil, and TBA. Initiation before 12 months reduced TBil-elevated (TBil > 20 μmol/L) odds by 80% (OR = 0.20, 95% CI 0.05–0.72) and hyperbilirubinemia (TBil > 34 μmol/L) by 86% (OR = 0.14, 95% CI 0.03–0.50). Low-dose UDCA (≤ 10 mg/kg/day) within 12 months provided optimal bilirubin/hepatic improvement without dose-dependence. UDCA beyond 36 months nearly doubled TBA levels, potentially reflecting dose-dependent TBA elevation.

Conclusion

UDCA efficacy post-KPE is age-dependent. Low-dose therapy (≤ 10 mg/kg/day) within 12 months significantly improves jaundice and liver function, with limited benefits at 12–36 months. Use beyond 36 months may be harmful. Optimal timing/dosing requires prospective validation.