ASCO GI 2023 | REFINE: a real-world analysis of regorafenib in patients with unresectable HCC
Richard Finn, MD, University of California, Los Angeles, CA, presents the final analysis of the prospective, observational REFINE trial (NCT03289273), which evaluated regorafenib in patients with unresectable hepatocellular carcinoma in routine clinical practice. Regorafenib was routinely administered in the second-line setting, often after patients progress on sorafenib, where survival was higher in the second-line setting. Reported safety and overall survival were additionally comparable to the data from the RESORCE trial (NCT01774344). This interview took place at the American Society of Clinical Oncology (ASCO) 2023 Gastrointestinal Cancers (GI) Symposium in San Francisco, CA.
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Transcript (edited for clarity)
So the REFINE study is an observational study looking at regorafenib use in the real world. Regorafenib was really only the second drug to be approved in the systemic management of advanced liver cancer. There had been many failures after sorafenib, and the resource study provided evidence that regorafenib improved survival in the second line. Since 2017, there’s been a lot of changes in the landscape of advanced liver cancer...
So the REFINE study is an observational study looking at regorafenib use in the real world. Regorafenib was really only the second drug to be approved in the systemic management of advanced liver cancer. There had been many failures after sorafenib, and the resource study provided evidence that regorafenib improved survival in the second line. Since 2017, there’s been a lot of changes in the landscape of advanced liver cancer. However, still, the REFINE study was launched right after the approval of regorafenib, and so it allowed us to gain some real world experience with the drug in terms of its primary endpoint of safety in a real world population, as well as clinical readout such as survival and PFS. And this study was global, over a thousand patients, and some interesting observations were made. One key observation is that regorafenib use occurred in patients who had not only had sorafenib, but other frontline regimens, which is a knowledge gap for us. And we saw that regorafenib performed similarly, regardless of what agent it got before. Obviously, survival was better if it was used earlier in the course, as a second line versus third line. Safety with regorafenib was very similar to what we saw in the Phase III RESOURCE clinical trial. And keep in mind, in real world studies, there aren’t as rigid inclusion/exclusion criteria. So the safety profile looked very similar. Also, we saw that in the real world, not everybody starts with the 160 milligram daily dose. But those who started at the lower dose seemed to have tolerated a lower dose of sorafenib. So maybe the clinicians started them on a lower dose of regorafenib subsequently. But they also had less compensated liver function. But at the end, the side effect profile was very similar.
And we saw that the survival for this overall population was over 13 months, which is very comparable to the Phase III study. And I think in some, this large dataset should give clinicians the confidence that regorafenib is an important agent in our armamentarium for managing patients with advanced liver cancer. And even though the landscape has changed, immunotherapy is now frontline for most patients. Having this data set gives us confidence that this drug is tolerable and likely plays a role in controlling disease.