Ding Dawei, Jia Gui, Guo Guanya, Cui Lina, Han Ying
State Key Laboratory of Holistic Integrative Management of Gastrointestinal Cancers, National Clinical Research Center for Digestive Diseases, Xijing Hospital of Digestive Diseases, The Air Force Military Medical University, Xi'an, 710032, Shaanxi, China.
Hepatol Int. 2025 Mar 28. doi: 10.1007/s12072-025-10820-8.
Primary biliary cholangitis (PBC) is divided into early and advanced stages, which are two distinct disease states, and whether this division is optimal remains to be demonstrated.
A risk stratification strategy was re-established according to histological stages and response criteria were defined accordingly.
We retrospectively analyzed 721 patients with histological data. The endpoint events were liver-related death and liver transplantation (LT).
Histological stage IV was associated with LT-free survival compared to stage III (HR: 2.764, 95% CI: 1.457-5.247, p = 0.002); and stage III was not associated with LT-free survival compared to stage II (HR: 1.632, 95% CI: 0.833-3.195, p = 0.153). Total bilirubin was associated with LT-free survival (HR: 1.162, 95% CI: 1.079-1.251, p < 0.001), whereas alkaline phosphatase was not associated with LT-free survival in cirrhotic patients (HR: 1.256, 95% CI: 0.958-1.648, p = 0.100). Compared to Paris I, Paris II, and Toronto, Rotterdam had the highest area under the receiver operating characteristic curve (AUC) for predicting the 5-year endpoint events in cirrhotic patients (0.652 [0.558-0.745]). Patients who had poor response according to Rotterdam criteria had worse prognosis than those who were biochemical responders (p = 0.036). Compared to Paris II and Paris I (for stage III) + Paris II (for stage I-II), Paris I, Rotterdam, and Toronto had higher AUC in non-cirrhotic patients (p < 0.05).
Risk stratification based on histological classification of non-cirrhosis versus cirrhosis demonstrates superior clinical utility compared to the early versus advanced stage stratification. Furthermore, the Rotterdam criteria proved to be clinically applicable for assessing biochemical responses specifically in patients with histological cirrhosis.
原发性胆汁性胆管炎（PBC）分为早期和晚期，这是两种不同的疾病状态，这种划分是否最佳仍有待证明。
根据组织学阶段重新建立风险分层策略，并相应地定义反应标准。
我们回顾性分析了721例有组织学数据的患者。终点事件为肝相关死亡和肝移植（LT）。
与III期相比，组织学IV期与无肝移植生存相关（HR：2.764，95%CI：1.457 - 5.247，p = 0.002）；与II期相比，III期与无肝移植生存无关（HR：1.632，95%CI：0.833 - 3.195，p = 0.153）。总胆红素与无肝移植生存相关（HR：1.162，95%CI：1.079 - 1.251，p < 0.001），而碱性磷酸酶在肝硬化患者中与无肝移植生存无关（HR：1.256，95%CI：0.958 - 1.648，p = 0.100）。与巴黎I、巴黎II和多伦多标准相比，鹿特丹标准在预测肝硬化患者5年终点事件方面具有最高的受试者操作特征曲线下面积（AUC）（0.652 [0.558 - 0.745]）。根据鹿特丹标准反应不佳的患者预后比生化反应者更差（p = 0.036）。与巴黎II以及巴黎I（用于III期） + 巴黎II（用于I - II期）相比，巴黎I、鹿特丹和多伦多标准在非肝硬化患者中具有更高的AUC（p < 0.05）。
与早期和晚期分层相比，基于非肝硬化与肝硬化组织学分类的风险分层显示出更好的临床实用性。此外，鹿特丹标准被证明在临床上适用于评估组织学肝硬化患者的生化反应。
