Abstract
Objectives: To use baseline variables to predict one-year disease control for patients with hepatocellular carcinoma (HCC) treated with transarterial chemoembolization (TACE) combined with sorafenib as initial treatment by applying a machine learning approach based on the random survival forest (RF) model. Materials and Methods: The multicenter retrospective study included 496 patients with HCC treated with TACE combined with sorafenib between January 2014 and December 2018. The independent risk factors associated with one-year disease control (complete response, partial response, stable disease) were identified using the RF model, and their predictive importance was determined using the Gini index. Tumor response was assessed according to modified Response Evaluation Criteria in Solid Tumors. Results: The median overall survival was 15.5 months. A total of 186 (37.5%) patients achieved positive one-year disease control. The Barcelona Clinic Liver Cancer (BCLC) stage (Gini index: 20.0), tumor size (≤7 cm, >7 cm; Gini index: 9.0), number of lobes involved (unilobar, bilobar; Gini index: 6.4), alpha-fetoprotein level (≤200 ng/dl, >200 ng/dl; Gini index: 6.1), albumin–bilirubin grade (Gini index: 5.7), and number of lesions (1, >1; Gini index: 5.3) were identified as independent risk factors, with the BCLC stage as the most important variable. The RF model achieved a higher concordance index of 0.724 compared to that for the logistic regression model (0.709). Conclusions: The RF model is a simple and accurate approach for prediction of one-year disease control for patients with HCC treated with TACE combined with sorafenib.
Highlights
Despite improving surveillance programs, around 80% of hepatocellular carcinomas (HCCs) are first diagnosed at an intermediate or advanced stage according to the Barcelona Clinic Liver Cancer (BCLC) staging system (Bray et al, 2018; Forner et al, 2018; Villanueva, 2019)
The random survival forest (RF) model is a simple and accurate approach for prediction of one-year disease control for patients with HCC treated with transarterial chemoembolization (TACE) combined with sorafenib
The median overall survival (OS) was significantly longer for patients with positive one-year disease control (CR/partial response (PR)/stable disease (SD)) compared to that of patients with negative
Summary
Around 80% of hepatocellular carcinomas (HCCs) are first diagnosed at an intermediate or advanced stage according to the Barcelona Clinic Liver Cancer (BCLC) staging system (Bray et al, 2018; Forner et al, 2018; Villanueva, 2019). Three randomized controlled trials (RCTs) failed to identify significant treatment efficacy and safety for TACE combined with sorafenib compared to TACE monotherapy (Kudo et al, 2011; Lencioni et al, 2016b; Meyer et al, 2017). A longer time of disease control in order to achieve a longer sorafenib administration period is an important factor for patients achieving survival benefit from the combination treatment of TACE and sorafenib (Kudo and Arizumi, 2017)
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