The PPRD score stratifies patients with hepatocellular carcinoma and portal vein tumor thrombus treated with sorafenib plus transarterial chemoembolization.

Objectives: To identify clinical prognostic and predictive factors in patients with hepatocellular carcinoma (HCC) and portal vein tumor thrombus (PVTT) undergoing sorafenib plus transarterial chemoembolization (TACE) and establish a prognostic score for these patients.

Methods: Between January 2012 and December 2017, 184 consecutive patients with HCC and PVTT were concurrently treated with sorafenib and TACE. Univariate and multivariate analyses were performed to explore the clinical factors independently correlated with overall survival (OS). A prognostic score was then developed to identify different prognoses in an initial cohort and validated in an external cohort (n = 72).

Results: In the multivariate analysis, performance status, extension of PVTT, initial radiological response, and sorafenib-related dermatologic toxicity were identified as predictors associated with OS. These factors were used to develop a prognostic score (PPRD score, range from 0 to 11). The median survival was found to decrease as the PPRD score increased, and patients were stratified into a favorable group (0 points), intermediate group (1-4 points), and dismal group (> 4 points). The median survival of patients in the three groups was 34.0 months, 20.0 months, and 7.0 months, respectively (p < 0.001). Additionally, the time to progression (TTP) (p < 0.001) was stratified along the same prognostic groups. The external validation cohort confirmed the prognostic scores.

Conclusions: The proposed score system can accurately stratify the outcomes of patients with HCC and PVTT treated with sorafenib plus TACE to help identify which group of patients may benefit from treatment.

Key Points: • The survival benefits of patients with advanced HCC treated with sorafenib plus TACE remains controversial. • The independent factors associated with survival were identified to develop a prognostic score, called the PPRD score (standing for performance status, PVTT grade, radiological response, and sorafenib-related dermatologic toxicity); the median survival decreases as the score increases. • The scoring system can accurately stratify the survival benefits of patients with HCC and PVTT treated with combination therapy and help to identify which group of patients may benefit from the treatment. Graphical abstract.