Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States
Richard S Finn,1 Renuka Iyer,2 Richard S Kalman,3 Neehar D Parikh,4 Roniel Cabrera,5 Svetlana Babajanyan,6 Ahmed O Kaseb7 1Department of Medicine, University of California, Los Angeles, CA, USA; 2Department of Medicine, Roswell Park Cancer Institute, Buffalo, NY, USA; 3Department of Medicine, Einste...
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Dove Medical Press
2025-02-01
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| Series: | Journal of Hepatocellular Carcinoma |
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| author | Finn RS Iyer R Kalman RS Parikh ND Cabrera R Babajanyan S Kaseb AO |
| author_facet | Finn RS Iyer R Kalman RS Parikh ND Cabrera R Babajanyan S Kaseb AO |
| author_sort | Finn RS |
| collection | DOAJ |
| description | Richard S Finn,1 Renuka Iyer,2 Richard S Kalman,3 Neehar D Parikh,4 Roniel Cabrera,5 Svetlana Babajanyan,6 Ahmed O Kaseb7 1Department of Medicine, University of California, Los Angeles, CA, USA; 2Department of Medicine, Roswell Park Cancer Institute, Buffalo, NY, USA; 3Department of Medicine, Einstein Medical Center, Philadelphia, PA, USA; 4Division of Gastroenterology and Hepatology, University of Michigan, Ann Arbor, MI, USA; 5Department of Medicine, University of Florida Health, Gainesville, FL, USA; 6US Medical Affairs, Oncology, Bayer Healthcare, Whippany, NJ, USA; 7Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USACorrespondence: Richard S Finn, Department of Medicine, University of California, Los Angeles, CA, USA, Tel +1-310-586-2091, Email rfinn@mednet.ucla.eduIntroduction: While several systemic therapies are available for unresectable hepatocellular carcinoma (uHCC), there is a lack of granular real-world evidence to support the efficacy and safety of these therapies. The REFINE study evaluated safety and effectiveness of regorafenib in a global population under real-world practice conditions. This sub-analysis describes the safety and effectiveness of regorafenib among the United States (US) subset of patients in the REFINE study relative to patients in the non-US subset.Materials and Methods: REFINE was an international, prospective, multicenter observational study. Eligible patients were those with uHCC for whom a decision to treat with regorafenib had already been made. The primary study endpoint was the frequency of documented treatment-emergent adverse events (TEAEs). Additional endpoints included overall survival and progression-free survival. Groups were compared descriptively.Results: Of 1005 patients, 65 were from the US and 940 were from other countries. 91% of patients in the US subset (n=59) and 92% in the non-US subset (n=862) experienced ≥ 1 TEAE. Common adverse events (AEs) included gastrointestinal disorders, fatigue, and hand-foot skin reaction. Median overall survival for patients in the US subset was 11.4 months (interquartile range [IQR]: 4.7– 25.4) and 13.2 months (IQR: 5.8– 26.3) in the non-US subset. Median progression-free survival was 3.4 months (IQR: 2.4– 6.1) for patients in the US subset and 3.9 months (IQR: 2.2– 8.5) in the non-US subset.Conclusion: Regorafenib was associated with similar safety and effectiveness outcomes for patients in the US and non-US subsets of the REFINE study. Differences in the incidence of certain AEs may be due to differences in treatment management between study sites or baseline disease status. These findings are consistent with the phase 3 RESORCE trial and corroborate the safety and effectiveness of regorafenib as a subsequent-line treatment in US patients with uHCC.Keywords: hepatocellular carcinoma, regorafenib, sorafenib, observational study |
| format | Article |
| id | doaj-art-d896497c09d74d198c1ae870328af097 |
| institution | Kabale University |
| issn | 2253-5969 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Dove Medical Press |
| record_format | Article |
| series | Journal of Hepatocellular Carcinoma |
| spelling | doaj-art-d896497c09d74d198c1ae870328af0972025-02-09T16:10:20ZengDove Medical PressJournal of Hepatocellular Carcinoma2253-59692025-02-01Volume 1223124699994Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United StatesFinn RSIyer RKalman RSParikh NDCabrera RBabajanyan SKaseb AORichard S Finn,1 Renuka Iyer,2 Richard S Kalman,3 Neehar D Parikh,4 Roniel Cabrera,5 Svetlana Babajanyan,6 Ahmed O Kaseb7 1Department of Medicine, University of California, Los Angeles, CA, USA; 2Department of Medicine, Roswell Park Cancer Institute, Buffalo, NY, USA; 3Department of Medicine, Einstein Medical Center, Philadelphia, PA, USA; 4Division of Gastroenterology and Hepatology, University of Michigan, Ann Arbor, MI, USA; 5Department of Medicine, University of Florida Health, Gainesville, FL, USA; 6US Medical Affairs, Oncology, Bayer Healthcare, Whippany, NJ, USA; 7Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USACorrespondence: Richard S Finn, Department of Medicine, University of California, Los Angeles, CA, USA, Tel +1-310-586-2091, Email rfinn@mednet.ucla.eduIntroduction: While several systemic therapies are available for unresectable hepatocellular carcinoma (uHCC), there is a lack of granular real-world evidence to support the efficacy and safety of these therapies. The REFINE study evaluated safety and effectiveness of regorafenib in a global population under real-world practice conditions. This sub-analysis describes the safety and effectiveness of regorafenib among the United States (US) subset of patients in the REFINE study relative to patients in the non-US subset.Materials and Methods: REFINE was an international, prospective, multicenter observational study. Eligible patients were those with uHCC for whom a decision to treat with regorafenib had already been made. The primary study endpoint was the frequency of documented treatment-emergent adverse events (TEAEs). Additional endpoints included overall survival and progression-free survival. Groups were compared descriptively.Results: Of 1005 patients, 65 were from the US and 940 were from other countries. 91% of patients in the US subset (n=59) and 92% in the non-US subset (n=862) experienced ≥ 1 TEAE. Common adverse events (AEs) included gastrointestinal disorders, fatigue, and hand-foot skin reaction. Median overall survival for patients in the US subset was 11.4 months (interquartile range [IQR]: 4.7– 25.4) and 13.2 months (IQR: 5.8– 26.3) in the non-US subset. Median progression-free survival was 3.4 months (IQR: 2.4– 6.1) for patients in the US subset and 3.9 months (IQR: 2.2– 8.5) in the non-US subset.Conclusion: Regorafenib was associated with similar safety and effectiveness outcomes for patients in the US and non-US subsets of the REFINE study. Differences in the incidence of certain AEs may be due to differences in treatment management between study sites or baseline disease status. These findings are consistent with the phase 3 RESORCE trial and corroborate the safety and effectiveness of regorafenib as a subsequent-line treatment in US patients with uHCC.Keywords: hepatocellular carcinoma, regorafenib, sorafenib, observational studyhttps://www.dovepress.com/tolerability-and-effectiveness-of-regorafenib-treatment-in-patients-wi-peer-reviewed-fulltext-article-JHChepatocellular carcinomaregorafenibsorafenibobservational study |
| spellingShingle | Finn RS Iyer R Kalman RS Parikh ND Cabrera R Babajanyan S Kaseb AO Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States Journal of Hepatocellular Carcinoma hepatocellular carcinoma regorafenib sorafenib observational study |
| title | Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States |
| title_full | Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States |
| title_fullStr | Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States |
| title_full_unstemmed | Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States |
| title_short | Tolerability and Effectiveness of Regorafenib Treatment in Patients with Unresectable Hepatocellular Carcinoma: Real-World Data from the United States |
| title_sort | tolerability and effectiveness of regorafenib treatment in patients with unresectable hepatocellular carcinoma real world data from the united states |
| topic | hepatocellular carcinoma regorafenib sorafenib observational study |
| url | https://www.dovepress.com/tolerability-and-effectiveness-of-regorafenib-treatment-in-patients-wi-peer-reviewed-fulltext-article-JHC |
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