Multicenter retrospective analysis of the safety and efficacy of regorafenib after progression on sorafenib in Korean patients with hepatocellular carcinoma

Published on Jun 1, 2019in Investigational New Drugs2.663
· DOI :10.1007/s10637-018-0707-5
Changhoon Yoo16
Estimated H-index: 16
(UOU: University of Ulsan),
Joong Won Park24
Estimated H-index: 24
+ 6 AuthorsHo Yeong Lim25
Estimated H-index: 25
(SKKU: Sungkyunkwan University)
Background The pivotal RESORCE trial showed that regorafenib was effective as second-line therapy for patients with advanced HCC who progressed on first-line sorafenib. Real-world data are needed to assess clinical outcomes and adverse events in the setting of daily practice. Methods Between April 2017 and August 2017, the Named Patient Program (NPP) was activated to provide controlled, pre-approval access of regorafenib in Korea. This analysis is a multicenter retrospective study of patients who received regorafenib under the NPP. Results A total of 49 patients entered into this NPP, and 40 patients received regorafenib in five Korean institutions. All but one patient received regorafenib as second-line therapy after progression on sorafenib, and 36 (90%) and 34 (85%) patients were classified as Child-Pugh A and BCLC stage C, respectively. The response rate was 10% (n = 4). The median progression-free survival (PFS) was 3.7 months (95% CI, 2.5–4.9 months), and the median overall survival (OS) was not reached. The 1 year OS rate was 54.6%. The time-to-progression (TTP) on prior sorafenib was significantly associated with PFS and OS. The most common grade 3–4 toxicities were hand-foot skin reaction (n = 3, 8%), hypertension (n = 2, 5%), and increased aspartate aminotransferase (n = 2, 5%). Conclusion Regorafenib was well-tolerated and effective in patients with advanced HCC who progressed on first-line sorafenib, with efficacy and safety outcomes consistent with those of the previous RESORCE trial. TTP on first-line sorafenib may predict the efficacy of subsequent regorafenib.
  • References (15)
  • Citations (2)
#1Richard S. Finn (UCLA: University of California, Los Angeles)H-Index: 52
#2Philippe MerleH-Index: 28
Last. Jordi Bruix (University of Barcelona)H-Index: 93
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#2Tim Meyer (UCL: University College London)H-Index: 40
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#2Richard S. Finn (UCLA: University of California, Los Angeles)H-Index: 52
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#2Sandra BerndtH-Index: 1
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// Maria Kissel 1 , Sandra Berndt 2 , Lukas Fiebig 1 , Simon Kling 3 , Qunsheng Ji 4 , Qingyang Gu 4 , Tina Lang 5 , Frank-Thorsten Hafner 1 , Michael Teufel 6 and Dieter Zopf 2 1 Drug Discovery, Bayer AG, Wuppertal, Germany 2 Drug Discovery, Bayer AG, Berlin, Germany 3 Biochemistry, NMI Natural and Medicinal Sciences Institute, University of Tubingen, Reutlingen, Germany 4 Research Service Division, Oncology & Immunology Unit, WuXi AppTec Co. Ltd., Shanghai, China 5 Research & Clinical Sciences...
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#2Bruno Sangro (University of Navarra)H-Index: 56
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Summary Background For patients with advanced hepatocellular carcinoma, sorafenib is the only approved drug worldwide, and outcomes remain poor. We aimed to assess the safety and efficacy of nivolumab, a programmed cell death protein-1 (PD-1) immune checkpoint inhibitor, in patients with advanced hepatocellular carcinoma with or without chronic viral hepatitis. Methods We did a phase 1/2, open-label, non-comparative, dose escalation and expansion trial (CheckMate 040) of nivolumab in adults (≥18...
609 CitationsSource
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#2Meinhardt GeroldH-Index: 1
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#1Jordi Bruix (University of Barcelona)H-Index: 93
#2Shukui QinH-Index: 28
Last. Guohong Han (Fourth Military Medical University)H-Index: 30
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Summary Background There are no systemic treatments for patients with hepatocellular carcinoma (HCC) whose disease progresses during sorafenib treatment. We aimed to assess the efficacy and safety of regorafenib in patients with HCC who have progressed during sorafenib treatment. Methods In this randomised, double-blind, parallel-group, phase 3 trial done at 152 sites in 21 countries, adults with HCC who tolerated sorafenib (≥400 mg/day for ≥20 of last 28 days of treatment), progressed on sorafe...
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#1MasatoshiKudo (Kindai University)H-Index: 75
#2Riccardo Lencioni (UniPi: University of Pisa)H-Index: 62
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Background & Aims Treatment approaches for hepatocellular carcinoma (HCC) vary across countries, but these differences and their potential impact on outcomes have not been comprehensively assessed. Data from the multinational GIDEON (Global Investigation of therapeutic DEcisions in HCC and Of its treatment with sorafeNib) registry evaluated differences in patient characteristics, practice patterns and outcomes in HCC across geographical regions in patients who received sorafenib. Methods GIDEON ...
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#1Lotfi Abou-Elkacem (RWTH Aachen University)H-Index: 3
#2Susanne Arns (RWTH Aachen University)H-Index: 9
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The combination of target-specific drugs like bevacizumab with chemotherapeutics has improved treatment efficacy in advanced colorectal cancer (CRC). However, the clinical prognosis of metastatic CRCs is still poor, and novel drugs are currently assessed with respect to their efficacies in patients with CRCs. In a phase III study, the multikinase inhibitor regorafenib (BAY 73-4506) has recently been shown to prolong survival of patients with CRCs after standard therapies failed. In the present s...
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#1Sadahisa Ogasawara (Chiba University)H-Index: 16
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Regorafenib showed promising results as a second-line agent after sorafenib failure in hepatocellular carcinoma patients. The aim of this meta-analysis was to evaluate the efficacy and safety of regorafenib in hepatocarcinoma patients. A computerized bibliographic search was performed on the main databases. The primary outcome was overall survival. Secondary outcomes were progression-free survival, tumor response, and the adverse events rate. Outcomes were pooled through a random-effects model a...
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