Survival in patients with unresectable hepatocellular carcinoma: TCC cocktail plus TACE vs TACE alone prospective randomized clinical trial.

[BACKGROUND] Transarterial chemoembolization (TACE) is commonly used to treat patients with unresectable hepatocellular carcinoma (HCC); however, TACE alone has demonstrated unsatisfactory survival benefits. Our previous studies suggested that TACE plus oral medication of thalidomide, carmofur and compound mylabris capsule (TCC cocktail) may be a better therapeutic option.

[METHODS] In this randomized, open-label, multicenter clinical trial, 72 treatment-naive HCC patients were randomly assigned to receive cTACE alone or cTACE plus oral TCC cocktail between July 2018 and October 2019. The primary endpoint of this trial was the 1-, 2- and 3-year overall survival (OS) rates. The second endpoints of this trial included 1-, 2- and 3-year progression-free survival (PFS) rates, objective response rates (ORR) according to the modified Response Evaluation Criteria in Solid Tumors (mRECIST), and safety with adverse events (AEs).

[RESULTS] The 1-, 2- and 3-year OS rates were significantly higher in the cTACE plus TCC group than in the cTACE group (83.2% vs 54.3%, 63.1% vs 30.1%, 37.7% vs 18.1%; p = 0.008), with a significantly longer median OS (29.0 vs 15.0 months; p < 0.001). Regarding the 1-, 2- and 3-year PFS rates, HCC patients in the cTACE plus TCC group also demonstrated significantly higher rates (66.3% vs 34.4%, 35.8% vs 18.8%, 31.8% vs 15.6%; p = 0.014) and had a longer median PFS (16.0 vs 8.0 months; p < 0.001) compared with cTACE group. All treatment-related AEs were tolerated.

[CONCLUSIONS] For patients with unresectable HCC, TACE combined with TCC cocktail was well tolerated and significantly improved clinical outcomes. Trial registration The trial was registered at https://www.chictr.org.cn/showproj.html?proj=27493 as ChiCTR1800016335 on 25th May 2018 named an open-label, multicenter, randomized, prospective clinical trial of thalidomide based triple oral regimen for low-dose maintenance therapy after TACE in advanced hepatocellular carcinoma.