1009P Regorafenib in patients (pts) with unresectable hepatocellular carcinoma (uHCC) in real-world practice in Asia: Interim results from the observational REFINE study

Abstract

Background: In the phase III RESORCE trial, regorafenib improved overall survival (OS) vs placebo in pts with uHCC who progressed on sorafenib. The international, prospective REFINE study was designed to evaluate regorafenib in pts with uHCC in routine practice. We present interim results for pts enrolled in REFINE in Korea, China, and Taiwan. Methods: REFINE is an ongoing observational study that recruited patients 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. A planned interim analysis was performed when the first 500 pts in the global cohort had been observed for 4 months. The primary aim is to assess treatment-emergent adverse events (TEAEs; NCI-CTCAE v4.03). Secondary endpoints include OS, progression-free survival, and tumor response. Tumor response and progression are assessed per investigator according to local standard. abstracts Annals of Oncology Volume 31 -Issue S4 -2020 S699

Real-world experience of safety and effectiveness of regorafenib for treatment of metastatic colorectal cancer, advanced gastrointestinal stromal tumors, and hepatocellular carcinoma: a post-marketing surveillance study in Korea

“…Although the PMS study should include all registry-approved indications, smaller numbers of patients with HCC and GIST were enrolled because GIST is a rare tumor, while regorafenib for HCC was the last to be approved by the Korean MFDS in 2017. However, it is not necessary to conduct further studies with more HCC patients regarding the effectiveness of regorafenib, because the effectiveness of regorafenib in HCC has been confirmed through the global large-scale prospective, observational REFINE study 14 , 15 and a larger real-world study on 440 HCC patients in Korea 16 . Another limitation is that a longer survival follow-up was not conducted after end of the study.…”

Evolving Treatment of Advanced Hepatocellular Carcinoma in the Asia–Pacific Region: A Review and Multidisciplinary Expert Opinion

“…It is the assessment of the authors that sorafenib and lenvatinib should both be offered to BCLC-B and BCLC-C HCC patients, and although supporting data are lacking, we believe, on the basis of our own clinical experience, that lenvatinib should not be withheld from patients with main portal vein invasion as long as they have good liver function (CP A). On the basis of real-world data from the observational REFINE study, sorafenib followed by regorafenib may be preferred for patients with CP-B (possibly even CP-C) liver function [ 44 , 45 , 46 ].…”