994P - Regorafenib (REG) in patients (pts) with unresectable hepatocellular carcinoma (uHCC) in real-world (RW) practice: Final analysis of the prospective, observational REFINE study by prior liver transplantation (PLT)

Background

In the final analysis of pts with uHCC from the observational REFINE study (NCT03289273), treatment-emergent adverse events (TEAEs) were consistent with the global, phase 3 RESORCE trial ( Kim YJ, ILCA 2022 ). Median overall survival (OS) in the overall cohort was 13.2 months. Pts with PLT are typically excluded from phase 3 clinical trials in HCC, including RESORCE. Here, we present the final analysis of pts with PLT in REFINE.

Methods

REFINE is an international, prospective, multicenter study that enrolled pts with uHCC for whom the decision to treat with REG was made by their physician before enrollment, according to the local health authority approved label. The primary aim was safety, including incidence of TEAEs (MedDRA v25) and dose modifications due to TEAEs. Secondary endpoints included OS and duration of treatment (DoT).

Results

Of the 1005 evaluable pts, 25 (2%) had PLT (median age, 60 [range 40–69] years; 60% Asian, 96% male). Most pts had ALBI grade 1 (60%), Child-Pugh A status (64%), ECOG performance status 0–1 (80%), and received prior sorafenib (92%); while none received prior immunotherapy. Furthermore, the most common HCC etiology was hepatitis B (68% vs 37% in pts with no PLT). Transarterial chemoembolization was the most common additional prior non-systemic treatment in the PLT subgroup (56%). Median DoT was numerically longer in the PLT vs non-PLT subgroup (5.5 [range 0.6–16.8] vs 3.7 [range <0.1–38.9] months). The most common TEAEs in the PLT subgroup were hand–foot skin reaction (52%), diarrhea (32%), and fatigue (24%). There were no grade 5 drug-related TEAEs in the PLT subgroup. Drug-related TEAEs leading to dose modification or permanent discontinuation were comparable in the PLT and non-PLT subgroups (44% vs 37% and 12% vs 16%, respectively). Median OS from start of REG treatment was similar in the PLT (15.8 months; 95% CI 10.1, 25.4) and non-PLT (12.8 months; 95% CI 11.4, 14.6) subgroups, and with the overall cohort.

Conclusions

Final data from the REFINE study confirm the safety and effectiveness of REG in pts with uHCC and PLT, consistent with findings from the overall cohort.

Clinical trial identification

Editorial acknowledgement

Editorial assistance in the preparation of this abstract was provided by Open Health Scientific Communications, London UK, with financial support from Bayer.

Legal entity responsible for the study

The authors.

Funding

Bayer.

Disclosure

M. Pinter: Financial Interests, Personal, Speaker, Consultant, Advisor: AstraZeneca, Bayer, Bristol Myers Squibb, Lilly, Eisai, Ipsen, MSD, Roche; Financial Interests, Personal, Speaker’s Bureau: Bayer, Bristol Myers Squibb, Eisai, Lilly, Roche. Y.J. Kim: Financial Interests, Institutional, Research Funding: BTG, Boston Scientific, AstraZeneca, Gilead Sciences, Samjin, BL&H, Bayer; Financial Interests, Personal, Speaker, Consultant, Advisor: Bayer, BMS, PharmaKing, Celltrion, Bukwang, Roche, AbbVie, Eisai, Boston Scientific, BMS, BTG, Bayer, MSD, Gilead, Novo Nordisk, Green Cross Cell, Boehringer Ingelheim. P. Merle: Financial Interests, Personal, Speaker, Consultant, Advisor: Roche, AstraZeneca, MSD, Eisai, Bayer, Ipsen; Financial Interests, Institutional, Research Funding: Ipsen. R.S. Finn: Financial Interests, Personal, Speaker, Consultant, Advisor: Pfizer, Bayer, Novartis, Bristol Myers Squibb, Merck, Eisai, Lilly, Genentech/Roche, AstraZeneca, Exelixis, CStone Pharmaceuticals; Financial Interests, Institutional, Research Funding: Pfizer, Bayer, Novartis, Eisai, Lilly, Merck, Bristol Myers Squibb, Roche/Genentech; Financial Interests, Personal, Expert Testimony: Bayer. J. Khan: Financial Interests, Personal, Full or part-time Employment: Bayer. K. Ozgurdal: Financial Interests, Personal, Stocks or ownership: Bayer; Financial Interests, Personal, Full or part-time Employment: Bayer. All other authors have declared no conflicts of interest.