原發性膽汁性膽管炎患者臨床顯著性門靜脈高壓的危險因素分析和預測模型建立

背景：原發性膽汁性膽管炎（PBC）患者病程早期即可出現食管胃靜脈曲張，防治靜脈曲張破裂出血對于PBC患者的遠期預后具有重要意義。目的：探討PBC患者發生臨床顯著性門靜脈高壓（CSPH）的危險因素并構建預測模型。方法：回顧性收集2018年1月—2022年1月上海交通大學醫學院附屬仁濟醫院消化內科收治的PBC患者的臨床資料，依據是否發生CSPH分為CSPH組和非CSPH組。采用多因素Logistic回歸和Lasso回歸篩選危險因素，構建CSPH發生風險預測模型，繪制列線圖并進行內部驗證。結果：458例PBC患者納入研究，CSPH組140例，非CSPH組318例。多因素分析顯示男性（OR=2.89）、抗著絲點抗體（ACA）陽性（OR=2.18）、抗gp210抗體陽性（OR=1.75）以及基線總膽紅素（OR=1.01）、IgA（OR=1.27）升高，白蛋白（OR=0.91）、血小板計數（OR=0.98）降低是CSPH發生的獨立危險因素。最終確定以上述7個變量建立預測模型并繪制列線圖，ROC曲線分析（AUC=0.891）和自助法內部驗證（C指數=0.891）顯示該模型有較高的穩定性和預測效能。結論：本研究構建的CSPH發生風險預測模型對PBC患者的風險分層有一定參考價值。臨床實踐中宜對ACA陽性PBC患者進行密切隨訪，以避免食管胃靜脈曲張破裂出血的發生。

關鍵詞 原發性膽汁性膽管炎； 臨床顯著性門靜脈高壓； 預測模型； 列線圖

Risk Assessment and Predictive Model Establishment of Clinically Significant Portal Hypertension in Patients With Primary Biliary Cholangitis LI Yao1， LIU Yuyang1， QIAN Jun1， MA Xiong1，2， WANG Qixia2. 1Division of Gastroenterology and Hepatology， Renji Hospital， School of Medicine， Shanghai Jiao Tong University; Shanghai Institute of Digestive Disease， Shanghai （200001）; 2Department of Infectious Diseases， Renji Hospital， School of Medicine， Shanghai Jiao Tong University， Shanghai

Correspondence to： WANG Qixia， Email： wqx0221155@126.com

Background： Patients with primary biliary cholangitis （PBC） may develop gastroesophageal varices at an early stage. It is of great significance to prevent variceal bleeding for the long?term outcome of PBC. Aims： To assess the risk factors for clinically significant portal hypertension （CSPH） among PBC patients and develop a predictive model. Methods： The clinical data of PBC patients admitted from January 2018 to January 2022 in the Division of Gastroenterology and Hepatology， Renji Hospital， Shanghai Jiao Tong University School of Medicine were retrospectively collected and analyzed. Patients were divided into CSPH and non?CSPH groups according to the diagnostic criteria of CSPH. Multivariate Logistic regression and Lasso regression were used to identify the risk factors and construct CSPH predictive model which was presented as nomogram and internally validated. Results： Of the 458 subjects enrolled in this study， 140 cases were in CSPH group while 318 cases in non?CSPH group. Multivariate analysis identified that male gender （OR=2.89）， positive for anti?centromere antibody （ACA; OR=2.18） and anti?gp210 antibody （OR=1.75）， high baseline total bilirubin （OR=1.01） and IgA （OR=1.27）， and low baseline albumin （OR=0.91） and platelet count （OR=0.98） were independent risk factors for CSPH. These variables were selected to form the predictive model and nomogram ultimately. Favorable stability and predictive performance of the model were confirmed by ROC curve analysis （AUC=0.891） and bootstrap method （C?index=0.891）. Conclusions： The predictive model constructed in this study has certain reference value for risk stratification of CSPH among PBC patients. Intensive follow?up is recommended for ACA?positive PBC patients in clinical practice for avoidance of gastroesophageal variceal bleeding.

Key words Primary Biliary Cholangitis; Clinically Significant Portal Hypertension; Predictive Model; Nomogram

原發性膽汁性膽管炎（primary biliary cholangitis， PBC）是一種累及肝內小膽管的慢性膽汁淤積性肝病[1]。與其他肝臟疾病不同的是，PBC患者病程早期即可出現食管胃靜脈曲張[2?3]，隨訪期間靜脈曲張破裂出血發生率可達15%[4]；已出現靜脈曲張的PBC患者，5年、10年生存率分別為63%和26%[5]。因此，防治食管胃靜脈曲張破裂出血對于PBC患者的遠期預后具有重要意義。……