Ruan Qiang, Wang Xiaohui, Li Qiang, Tao Yu, Xun Yang, Yang Hua, Zhao Han, Lu Jinfeng, Yang Dazhi, Chen Yifan, Wei Li
Department of the Second Area of Gastrointestinal Surgery, Guangzhou Institute of Cancer Research, The Affiliated Cancer Hospital, Guangzhou Medical University, Guangzhou, China.
Department of Hepatobiliary Surgery, Hunan Provincial People's Hospital (The First Affiliated Hospital of Hunan Normal University), Changsha, China.
Sci Rep. 2025 Aug 12;15(1):29608. doi: 10.1038/s41598-025-13799-2.
There is no well-established model to predict the outcomes of patients with unresectable hepatocellular carcinoma (u-HCC) receiving targeted therapy. The goal of this study was to develop and validate a prediction model that accurately predicts outcomes of patients who received targeted therapy for u-HCC. We retrospectively analyzed data from patients with u-HCC who had received targeted therapy (sorafenib or lenvatinib) between 2011 and 2023 across three centers. The clinical data from two centers were divided in a 7:3 ratio to create training and internal validation sets, respectively. While the data from the third center was used as the external validation dataset. In the training set, the variables independently associated with overall survival (OS) or progression-free survival (PFS) in multivariable analysis were alpha-fetoprotein level ≥ 20 ng/mL and macrovascular invasion (MVI). The variables were then used to develop the targeted therapy for unresectable hepatocellular carcinoma prognosis (TUHP) model. In the validation set, the TUHP model was tested and compared with other prognostic model. The results showed that the TUHP model was also significantly associated with OS and PFS and exhibited greater discriminative ability than the existing prognostic models. The TUHP model accurately predicted OS and PFS among patients with u-HCC who received targeted therapy in both training and validation cohorts. The TUHP model may help optimize outcomes of patients who receive targeted therapy for u-HCC.
目前尚无成熟的模型可用于预测接受靶向治疗的不可切除肝细胞癌（u-HCC）患者的预后。本研究的目的是开发并验证一种能准确预测接受u-HCC靶向治疗患者预后的预测模型。我们回顾性分析了2011年至2023年间在三个中心接受靶向治疗（索拉非尼或仑伐替尼）的u-HCC患者的数据。将两个中心的临床数据按7:3的比例分别划分为训练集和内部验证集。而将第三个中心的数据用作外部验证数据集。在训练集中，多变量分析中与总生存期（OS）或无进展生存期（PFS）独立相关的变量为甲胎蛋白水平≥20 ng/mL和大血管侵犯（MVI）。然后使用这些变量开发了不可切除肝细胞癌靶向治疗预后（TUHP）模型。在验证集中，对TUHP模型进行了测试，并与其他预后模型进行了比较。结果显示，TUHP模型也与OS和PFS显著相关，并且比现有的预后模型具有更强的判别能力。TUHP模型在训练队列和验证队列中均准确预测了接受靶向治疗的u-HCC患者的OS和PFS。TUHP模型可能有助于优化接受u-HCC靶向治疗患者的预后。
