TY - JOUR
T1 - Regorafenib for Hepatocellular Carcinoma in Real-World Practice (REFINE)
T2 - A Prospective, Observational Study
AU - Kim, Yoon Jun
AU - Merle, Philippe
AU - Finn, Richard S.
AU - Kudo, Masatoshi
AU - Klümpen, Heinz Josef
AU - Lim, Ho Yeong
AU - Ikeda, Masafumi
AU - Granito, Alessandro
AU - Masi, Gianluca
AU - Gerolami, René
AU - Cho, Sung Bum
AU - Hsu, Chih Hung
AU - Huang, Yi Hsiang
AU - Jeng, Long Bin
AU - Kim, Do Young
AU - Lin, Shi Ming
AU - Pinter, Matthias
AU - Shao, Guoliang
AU - Kato, Naoya
AU - Kurosaki, Masayuki
AU - Numata, Kazushi
AU - Kuo, Kung Kai
AU - Mao, Yilei
AU - Lin, Yih Jyh
AU - Zhu, Kangshun
AU - Twumasi-Ankrah, Philip
AU - Khan, Javeed
AU - Awan, Maria
AU - Ozgurdal, Kirhan
AU - Qin, Shukui
N1 - Publisher Copyright:
© 2024 The Author(s). Published by S. Karger AG, Basel.
PY - 2024
Y1 - 2024
N2 - Introduction: In the phase 3 RESORCE trial, regorafenib prolonged overall survival (OS) in patients with unresectable hepatocellular carcinoma (uHCC) whose disease progressed on prior sorafenib. The prospective, observational REFINE study aimed to evaluate the safety and effectiveness of regorafenib in a broader population of patients in real-world clinical practice, including patients with Eastern Cooperative Oncology Group performance status (ECOG PS) .2, Child- Pugh B liver status, and sorafenib intolerance. Methods: This international, prospective, multicenter study (NCT03289273) enrolled patients with uHCC for whom the decision to treat with regorafenib was made by their physician before enrollment, according to the local health authority-approved label. The primary aim was to evaluate the safety of regorafenib, including the incidence of treatment-emergent adverse events (TEAEs) and dose modifications due to TEAEs. Results: Of the 1,028 patients enrolled, 1,005 initiated regorafenib and were eligible for analysis. Median age was 66 years (range 21.94); most patients were male (83%), Child-Pugh A (61%), and had an ECOG PS of 0 or 1 (82%) at study entry. Overall, 47%, 11%, and 40% of patients initiated regorafenib at 160, 120, and 80 mg/day, respectively. Median treatment duration was 3.7 months (range 1 day to 38.9 months). Dose modifications and permanent discontinuation of regorafenib due to TEAEs occurred in 45% and 31% of patients, respectively. The most common drug-related TEAEs were hand-foot skin reaction (31%), diarrhea (26%), and fatigue (15%). Median OS was 13.2 months (95% confidence interval 11.6, 14.8). Conclusion: The results of the real-world REFINE study confirmed the safety and effectiveness of regorafenib in a broad population of patients with uHCC. Of patients who received standard regorafenib dosing in REFINE, safety and efficacy findings were consistent with those reported in the RESORCE trial.
AB - Introduction: In the phase 3 RESORCE trial, regorafenib prolonged overall survival (OS) in patients with unresectable hepatocellular carcinoma (uHCC) whose disease progressed on prior sorafenib. The prospective, observational REFINE study aimed to evaluate the safety and effectiveness of regorafenib in a broader population of patients in real-world clinical practice, including patients with Eastern Cooperative Oncology Group performance status (ECOG PS) .2, Child- Pugh B liver status, and sorafenib intolerance. Methods: This international, prospective, multicenter study (NCT03289273) enrolled patients with uHCC for whom the decision to treat with regorafenib was made by their physician before enrollment, according to the local health authority-approved label. The primary aim was to evaluate the safety of regorafenib, including the incidence of treatment-emergent adverse events (TEAEs) and dose modifications due to TEAEs. Results: Of the 1,028 patients enrolled, 1,005 initiated regorafenib and were eligible for analysis. Median age was 66 years (range 21.94); most patients were male (83%), Child-Pugh A (61%), and had an ECOG PS of 0 or 1 (82%) at study entry. Overall, 47%, 11%, and 40% of patients initiated regorafenib at 160, 120, and 80 mg/day, respectively. Median treatment duration was 3.7 months (range 1 day to 38.9 months). Dose modifications and permanent discontinuation of regorafenib due to TEAEs occurred in 45% and 31% of patients, respectively. The most common drug-related TEAEs were hand-foot skin reaction (31%), diarrhea (26%), and fatigue (15%). Median OS was 13.2 months (95% confidence interval 11.6, 14.8). Conclusion: The results of the real-world REFINE study confirmed the safety and effectiveness of regorafenib in a broad population of patients with uHCC. Of patients who received standard regorafenib dosing in REFINE, safety and efficacy findings were consistent with those reported in the RESORCE trial.
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U2 - 10.1159/000542285
DO - 10.1159/000542285
M3 - Article
AN - SCOPUS:85216814110
SN - 2235-1795
JO - Liver Cancer
JF - Liver Cancer
ER -
