Outcome prediction model for patients with unresectable hepatocellular carcinoma treated with targeted therapy.

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.