胆道恶性肿瘤肝移植的适应证演变与新辅助治疗策略进展

Evolution of indications and neoadjuvant treatment strategies for liver transplantation in biliary tract cancer

摘要:

胆道恶性肿瘤（BTC）包括肝内胆管癌（iCCA）、肝门部胆管癌（pCCA）及远端胆管癌（dCCA），整体预后较差。近年来，随着新辅助治疗（NAT）、分子分型及移植标准的不断优化，肝移植在BTC中的应用取得重要进展。对于pCCA，基于Mayo方案的“NAT+肝移植”策略显著改善了不可切除患者的长期生存，但仍面临复发及血管并发症等挑战；对于iCCA，肝移植适应证已由禁忌证逐步扩展至极早期及部分经NAT转化的局部晚期患者，显示出与肝细胞癌相当的生存获益。与此同时，靶向治疗及免疫治疗的发展为NAT方案优化提供了新的方向。本文综述BTC肝移植的适应证演变、预后影响因素、患者筛选策略及当前研究进展，并探讨未来发展方向，以期为临床决策提供参考。

Abstract:

Biliary tract carcinoma (BTC), including intrahepatic cholangiocarcinoma (iCCA), perihilar cholangiocarcinoma (pCCA), and distal cholangiocarcinoma (dCCA), is associated with poor prognosis. In recent years, advances in neoadjuvant therapy (NAT), molecular stratification, and transplant selection criteria have substantially expanded the role of liver transplantation in BTC. For pCCA, the combination of NAT and liver transplantation, particularly under the Mayo protocol, has significantly improved long-term survival in selected unresectable patients, although recurrence and vascular complications remain major challenges. For iCCA, liver transplantation has evolved from a contraindication to a potential therapeutic option in highly selected patients, especially those with very early-stage disease or those successfully downstaged by NAT, achieving survival outcomes comparable to hepatocellular carcinoma. Meanwhile, the emergence of targeted therapy and immunotherapy provides new opportunities for optimizing NAT strategies. This review summarizes the evolution of indications, prognostic factors, candidate selection, and recent advances in liver transplantation for BTC, and discusses future perspectives to inform clinical decision-making.