73MO - Regorafenib in patients (pts) with unresectable hepatocellular carcinoma (uHCC) in real-world practice in Asia: Final analysis of the prospective, observational REFINE study

Background

In an interim analysis of Asian pts with uHCC in the observational REFINE study of regorafenib (NCT03289273), treatment-emergent adverse events (TEAEs) were consistent with those reported in the global, phase 3 RESORCE trial. Here, we present the final analysis of Asian pts with uHCC in REFINE.

Methods

REFINE is an international, prospective, multicenter study that enrolled pts with uHCC for whom a decision to treat with regorafenib was made by the treating physician prior to enrollment, according to the local health authority approved label. The primary objective is safety, including the incidences of TEAEs and dose modifications due to TEAEs (NCI-CTCAE v4.03). Secondary endpoints include overall survival, progression-free survival, and treatment duration.

Results

Of the 1005 evaluable pts, 557 (55%) were from Asia (Korea [31%], Japan [26%], Taiwan [24%], China [18%], Thailand [1%]) and 82% were male. At baseline, median age was 65 years (range 21–94) and the most common HCC etiology in Asian pts was hepatitis B (60%) and in non-Asian pts was alcohol use (36%; Table). More Asian pts (71%) had received prior transarterial chemoembolization vs non-Asian pts (42%). The initial daily regorafenib dose was 160/120/80/40 mg in 51%/12%/35%/3% of Asian pts and 42%/9%/45%/4% of non-Asian pts. The median treatment duration was 3.7 months (range 0–34.4) in Asian pts and 3.6 months (range 0–38.9) in non-Asian pts. The most common TEAEs in Asian pts were hand–foot skin reaction (40%), diarrhea (27%), and decreased appetite (17%). TEAEs led to dose modification in 44% of Asian pts. Table: 73MO

*Pts with baseline tumor assessment >16 weeks before the start of regorafenib are excluded and currently under review.

Conclusions

These final data from REFINE confirm the safety and effectiveness of regorafenib in Asian pts with uHCC from a broad population in real-world practice. Final analyses from REFINE are ongoing and will be presented at the conference.

Clinical trial identification

NCT03289273.

Editorial acknowledgement

Editorial assistance in the preparation of this manuscript was provided by Matthew Reynolds of OPEN Health Communications (London, UK), with financial support from Bayer.

Legal entity responsible for the study

Bayer.

Funding

Bayer.

Disclosure

Y.J. Kim: Financial Interests, Personal, Advisory Role: Bayer, Bristol Myers Squibb, Samil, PharmaKing, Celltrion, Bukwang; Financial Interests, Personal, Invited Speaker: Roche, AbbVie, Eisai, Ipsen, Boston Scientific, Bristol Myers Squibb, BTG, Bayer, MSD, Gilead Sciences, Novo Nordisk, Green Cross Cell, Boehringer Ingelheim, AstraZeneca; Financial Interests, Personal, Funding: BTG, Bayer, Boston Scientific, AstraZeneca, Gilead Sciences, Samjin, BL&H. M. Kurosaki: Financial Interests, Personal, Speaker’s Bureau: Gilead Sciences, AbbVie, Eisai, Chugai, Lilly, Takeda. H.Y. Lim: Financial Interests, Personal, Advisory Role: Bayer, Eisai, Roche, Ipsen. M. Ikeda: Financial Interests, Personal, Advisory Board: AstraZeneca, Chugai, Eli Lilly Japan, Eisai, Nihon Servier, Novartis, Ono, Takeda, GlaxoSmithKline; Financial Interests, Personal, Invited Speaker: AstraZeneca, Bayer, Bristol Myers Squibb, Chugai, Eli Lilly Japan, Eisai, Nihon Servier, Novartis, Taiho, Yakult, Teijin Pharma, AbbVie, Abbott Japan, Fujifilm Toyama Chemical, Incyte Biosciences Japan, ASLAN, Chugai, Nihon Servier, Takeda; Financial Interests, Institutional, Invited Speaker: Bayer, Bristol Myers Squibb, Eisai, AstraZeneca, Eli Lilly Japan, Chugai Pharmaceutical, Merck Serono, MSD, Ono, Yakult, Novartis, Takeda, J-Pharma, Pfizer, Chiome Bioscience, Nihon Servier, Delta-Fly Pharma, Syneos Health, Merus.N.V. M. Kudo: Financial Interests, Personal, Invited Speaker: Eisai, Chugai, Eli Liiy, Bayer, Takeda, MSD; Financial Interests, Institutional, Research Grant: Otsuka, Sumitomo Dainippon Pharma, EA Pharma, Taiho, Eisai, AbbVie, Gilead Sciences, Takeda, GE Healthcare, Chugai. Y. Huang: Financial Interests, Personal, Advisory Role: Eisai, Bayer, BMS, Ono, Gilead, Lilly, AbbVie, Roche; Financial Interests, Personal, Invited Speaker: Eisai, Bayer, BMS, Ono, Gilead, Lilly, AbbVie, Roche; Financial Interests, Personal, Speaker’s Bureau: Eisai, Bayer, BMS, Ono, Gilead, Lilly, AbbVie, Roche; Financial Interests, Institutional, Funding: Gilead. N. Kato: Financial Interests, Personal, Invited Speaker: Gilead Sciences Inc., AbbVie G.K., Ohtsuka Pharmaceutical Co., Ltd., Bayer Yakuhin Ltd., Chugai Pharmaceutical Co., Ltd., AstraZeneca K.K., Sumitomo Dainippon Pharma Co., Ltd., Takeda Pharmaceutical Co., Ltd., Zeria Pharmaceutical Co., Ltd., Olympus Corporation, Eisai Co., Ltd., Aska Pharmaceutical Co., Ltd., Tsumura & Co., Mochida Pharmaceutical Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., Covidien Japan Inc., Eli Lilly Japan K.K., Nobelpharma Co., Ltd., Kowa Company, Ltd., Incyte Biosciences Japan GK, Yakult Honsha Co.,Ltd., Olympus Marketing, Inc., Taisho Pharmaceutical Co.,Ltd., Janssen Pharmaceutical K.K.; Financial Interests, Institutional, Research Grant: AbbVie G.K., Ohtsuka Pharmaceutical Co., Ltd., Bayer Yakuhin Ltd., Chugai Pharmaceutical Co., Ltd., Mitsubishi Tanabe Pharma Corporation, Sumitomo Dainippon Pharma Co., Ltd., Shionogi & Co., Ltd., Eisai Co., Ltd., Tsumura & Co., Nippon Kayaku Co., Ltd., JIMRO Co., Ltd., Kowa Company, Ltd. C. Hsu: Financial Interests, Personal, Speaker’s Bureau: Bristol Myers Squibb, Ono Pharmaceutical, Merck Sharp & Dohme, Roche, Eisai; Financial Interests, Institutional, Funding: Ono Pharmaceutical, AstraZeneca, MSD, Merck Serono, Taiho Pharmaceutical, Bristol Myers Squibb, BeiGene, NuCana BioMed, Johnson & Johnson, Roche/Genentech, BeiGene; Financial Interests, Personal, Advisory Role: Ono Pharmaceutical, MSD, Bristol Myers Squibb, Merck Serono, Roche/Genentech. B. Chewaskulyong: Financial Interests, Personal, Advisory Role: Pfizer, STADA; Financial Interests, Personal, Invited Speaker: AstraZeneca, Pfizer, DKSH, Janssen, BMS, MSD, Roche, TAIHO; Financial Interests, Personal, Speaker’s Bureau: AstraZeneca, Pfizer, DKSH, Janssen, BMS, MSD, Roche, TAIHO; Financial Interests, Institutional, Funding: Bayer. J. Khan: Financial Interests, Institutional, Full or part-time Employment: Bayer. K. Ozgurdal: Financial Interests, Institutional, Full or part-time Employment: Bayer; Financial Interests, Personal, Stocks/Shares: Bayer. All other authors have declared no conflicts of interest.