BACKGROUND
Immunotherapy is a new promising treatment for patients with advanced hepatocellular carcinoma (HCC), but is costly and potentially associated with considerable side effects.
OBJECTIVE
This study aimed to evaluate the role of machine learning (ML) models in predicting the one-year cancer-related mortality in advanced HCC patients treated with immunotherapy
METHODS
395 HCC patients who had received immunotherapy (including nivolumab, pembrolizumab or ipilimumab) in 2014 - 2019 in Hong Kong were included. The whole data set were randomly divided into training (n=316) and validation (n=79) set. The data set, including 45 clinical variables, was used to construct six different ML models in predicting the risk of one-year mortality. The performances of ML models were measured by the area under receiver operating characteristic curve (AUC) and the mean absolute error (MAE) using calibration analysis.
RESULTS
The overall one-year cancer-related mortality was 51.1%. Of the six ML models, the random forest (RF) has the highest AUC of 0.93 (95%CI: 0.86-0.98), which was better than logistic regression (0.82, p=0.01) and XGBoost (0.86, p=0.04). RF also had the lowest false positive (6.7%) and false negative rate (2.8%). High baseline AFP, bilirubin and alkaline phosphatase were three common risk factors identified by all ML models.
CONCLUSIONS
ML models could predict one-year cancer-related mortality of HCC patients treated with immunotherapy, which may help to select patients who would most benefit from this new treatment option.