Background: Immune checkpoint inhibitors (ICIs) have been increasingly used to treat hepatocellular carcinoma (HCC). Prognostic biomarkers are an unmet need. We aimed to develop a prognostic nomogram for patients with unresectable HCC receiving ICIs therapy. Methods: A total of 120 patients with unresectable HCC receiving ICIs treatment were enrolled in this study. Patients were randomly divided into a training set (n = 84) and a validation set (n = 36) in a 7:3 ratio. Clinical characteristics were retrospectively analyzed. Serum α-fetoprotein protein (AFP) response was defined as a decline of ≥20% in AFP levels within the initial eight weeks of treatment. Univariable and multivariable Cox analyses were used to select relevant variables and construct the nomogram. The areas under the receiver operating characteristic curves (AUCs) were used to determine the performance of the model. Kaplan–Meier analysis with the log-rank test was used to compare different risk groups. Results: The median progression-free survival (PFS) was 7.7 months. In the multivariate Cox analysis, the presence of extrahepatic metastasis (hazard ratio [HR] = 2.08, 95% confidence interval [CI]: 1.02–4.27,p< 0.05), white blood cell count (HR = 3.48, 95% CI: 1.02–11.88,p< 0.05) and AFP response (HR = 0.41, 95% CI: 0.18–0.95,p< 0.05) independently predicted PFS. A nomogram for PFS was established with AUCs of 0.79 and 0.70 in the training and validation sets, respectively. The median PFS of the high- and low-risk subgroups was 3.5 and 11.7 months, respectively (p< 0.05). Conclusion: The nomogram could predict PFS in patients with unresectable HCC receiving ICIs treatment and further help decision making in daily clinical practice.
背景：免疫检查点抑制剂（ICIs）在肝细胞癌（HCC）治疗中的应用日益广泛，但目前仍缺乏有效的预后生物标志物。本研究旨在构建一个用于预测接受ICIs治疗的不可切除HCC患者预后的列线图模型。 方法：本研究共纳入120例接受ICIs治疗的不可切除HCC患者。按7:3的比例将患者随机分为训练集（84例）和验证集（36例），回顾性分析其临床特征。血清甲胎蛋白（AFP）反应定义为治疗最初八周内AFP水平下降≥20%。采用单变量和多变量Cox分析筛选相关变量并构建列线图。通过受试者工作特征曲线下面积（AUC）评估模型性能，并采用Kaplan-Meier分析和log-rank检验比较不同风险组间的差异。 结果：患者的中位无进展生存期（PFS）为7.7个月。多变量Cox分析显示，肝外转移（风险比[HR]=2.08，95%置信区间[CI]:1.02–4.27，p<0.05）、白细胞计数（HR=3.48，95%CI:1.02–11.88，p<0.05）和AFP反应（HR=0.41，95%CI:0.18–0.95，p<0.05）是PFS的独立预测因素。基于此构建的PFS列线图在训练集和验证集中的AUC值分别为0.79和0.70。高风险组和低风险组的中位PFS分别为3.5个月和11.7个月（p<0.05）。 结论：该列线图能有效预测接受ICIs治疗的不可切除HCC患者的PFS，有助于临床治疗决策的制定。
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