Section of Gastroenterology and Hepatology, Department of Medicine, Baylor College of Medicine, Houston, Texas; Section of Health Services Research, Department of Medicine, Baylor College of Medicine, Houston, Texas; VA HSR&D Center for Innovatio ns in Quality, Effectiveness, and Safety, Michael E. DeBakey Veterans Affairs Medical Center, Houston, Texas.
Section of Gastroenterology and Hepatology, Department of Medicine, Baylor College of Medicine, Houston, Texas.
Clin Gastroenterol Hepatol. 2023 Dec;21(13):3296-3304.e3. doi: 10.1016/j.cgh.2023.04.019. Epub 2023 Apr 30.
BACKGROUND & AIMS: The available risk stratification indices for hepatocellular cancer (HCC) have limited applicability. We developed and externally validated an HCC risk stratification index in U.S. cohorts of patients with cirrhosis.
We used data from 2 prospective U.S. cohorts to develop the risk index. Patients with cirrhosis were enrolled from 8 centers and followed until development of HCC, death, or December 31, 2021. We identified an optimal set of predictors with the highest discriminatory ability (C-index) for HCC. The predictors were refit using competing risk regression and its predictive performance was evaluated using the area under the receiver-operating characteristic curve (AUROC). External validation was performed in a cohort of 21,550 patients with cirrhosis seen in the U.S Veterans Affairs system between 2018 and 2019 with follow-up through 2021.
We developed the model in 2431 patients (mean age 60 years, 31% women, 24% cured hepatitis C, 16% alcoholic liver disease, and 29% nonalcoholic fatty liver disease). The selected model had a C-index of 0.77 (95% confidence interval [CI], 0.73-0.81), and the predictors were age, sex, smoking, alcohol use, body mass index, etiology, α-fetoprotein, albumin, alanine aminotransferase, and platelet levels. The AUROCs were 0.75 (95% CI, 0.65-0.85) at 1 year and 0.77 (95% CI, 0.71-0.83) at 2 years, and the model was well calibrated. In the external validation cohort, the AUROC at 2 years was 0.70 with excellent calibration.
The risk index, including objective and routinely available risk factors, can differentiate patients with cirrhosis who will develop HCC and help guide discussions regarding HCC surveillance and prevention. Future studies are needed for additional external validation and refinement of risk stratification.
现有的肝细胞癌(HCC)风险分层指标的适用性有限。我们在美国肝硬化患者队列中开发并验证了一种 HCC 风险分层指标。
我们使用来自 2 个美国前瞻性队列的数据来开发风险指数。从 8 个中心招募肝硬化患者,并随访至 HCC 发生、死亡或 2021 年 12 月 31 日。我们确定了一组最优的预测因子,这些预测因子具有最高的 HCC 区分能力(C 指数)。使用竞争风险回归对预测因子进行重新拟合,并使用接受者操作特征曲线下的面积(AUROC)评估其预测性能。外部验证在 2018 年至 2019 年期间在美国退伍军人事务系统中观察到的 21550 例肝硬化患者队列中进行,随访至 2021 年。
我们在 2431 例患者中建立了模型(平均年龄 60 岁,31%为女性,24%治愈丙型肝炎,16%为酒精性肝病,29%为非酒精性脂肪性肝病)。所选模型的 C 指数为 0.77(95%置信区间 [CI],0.73-0.81),预测因子为年龄、性别、吸烟、饮酒、体重指数、病因、甲胎蛋白、白蛋白、丙氨酸氨基转移酶和血小板水平。1 年时的 AUROC 为 0.75(95%CI,0.65-0.85),2 年时为 0.77(95%CI,0.71-0.83),模型校准良好。在外部验证队列中,2 年时的 AUROC 为 0.70,且校准效果良好。
该风险指数包括客观且常规可获得的风险因素,可以区分出会发生 HCC 的肝硬化患者,并有助于指导 HCC 监测和预防的讨论。未来还需要进行更多的外部验证和风险分层的细化研究。