Anouti Ahmad, Sudhakara Pavithra, Pratt Chelsea, Mourya Reena, VanWagner Lisa, Shivakumar Pranavkumar, Pandurangi Sindhu
Department of Pediatrics, Children's Medical Center Dallas, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Children's Medical Center Dallas, Dallas, Texas, USA.
J Pediatr Gastroenterol Nutr. 2025 Sep;81(3):497-506. doi: 10.1002/jpn3.70131. Epub 2025 Jun 27.
Biliary atresia (BA) is a progressive, fibrosing cholangiopathy of infancy characterized by inflammatory obstruction of the bile ducts, ultimately leading to end-stage liver disease if untreated. Early diagnosis and timely surgical intervention via hepatoportoenterostomy (HPE) are critical for improving outcomes; however, prognostication remains challenging due to heterogeneous responses to surgery and variable clinical trajectories. This review provides a comprehensive synthesis of current research on prognostic biomarkers in BA, encompassing clinical indicators, routine laboratory parameters, novel serum biomarkers, histopathologic features, hepatic gene expression profiles, imaging modalities, and both in vitro and computational prognostic modeling systems. While traditional clinical factors, such as age at HPE and postoperative serum bilirubin levels, continue to serve as important predictors of outcome, they lack sufficient discriminatory power for individualized risk stratification. Recent advances have identified emerging biomarkers, including inflammatory cytokines, immune activation markers, and indicators of fibrosis and extracellular matrix remodeling, which show potential in correlating with disease progression and native liver survival. Imaging modalities such as ultrasound elastography have also demonstrated promise in noninvasively assessing liver stiffness and predicting clinical outcomes. Furthermore, the identification of hepatic gene expression signatures and multigene prognostic classifiers offers new avenues for precision risk assessment. However, most of these advancements have not translated into clinical practice due to small sample sizes and limited external validation. Future research efforts must focus on large-scale, multicenter studies to validate findings and establish robust, integrative prognostic models that can inform clinical decision-making and facilitate personalized therapeutic strategies in BA.
胆道闭锁(BA)是一种婴儿期进行性纤维化胆管病,其特征为胆管的炎性梗阻,若不治疗最终会导致终末期肝病。早期诊断并通过肝门空肠吻合术(HPE)及时进行手术干预对于改善预后至关重要;然而,由于对手术的反应异质性和临床病程多变,预后评估仍然具有挑战性。本综述全面综合了目前关于BA预后生物标志物的研究,涵盖临床指标、常规实验室参数、新型血清生物标志物、组织病理学特征、肝脏基因表达谱、成像方式以及体外和计算机预后建模系统。虽然传统临床因素,如HPE时的年龄和术后血清胆红素水平,仍然是重要的预后预测指标,但它们在个体风险分层方面缺乏足够的鉴别能力。最近的进展已经确定了新出现的生物标志物,包括炎性细胞因子、免疫激活标志物以及纤维化和细胞外基质重塑指标,这些生物标志物在与疾病进展和肝脏生存相关方面显示出潜力。超声弹性成像等成像方式在无创评估肝脏硬度和预测临床结果方面也显示出前景。此外,肝脏基因表达特征和多基因预后分类器的鉴定为精准风险评估提供了新途径。然而,由于样本量小和外部验证有限,这些进展大多尚未转化为临床实践。未来的研究工作必须集中在大规模、多中心研究上,以验证研究结果并建立强大的综合预后模型,为临床决策提供依据,并促进BA的个性化治疗策略。