Regorafenib in patients (pts) with unresectable hepatocellular carcinoma (uHCC) in real-world practice in the United States (US): Final analysis of the prospective, observational REFINE study.

• DOI: 10.1200/jco.2023.41.16_suppl.e16114
• Publication Date: 2023-06-04T15:38:35Z
• Authors: Ahmed Omar Kaseb; Roniel Cabrera; Renuka Iyer; Neehar Parikh; Richard Kalman; Kirhan Ozgurdal; Javeed Khan; Svetlana Babajanyan; Richard S. Finn
• Keywords: Regorafenib; Clinical endpoint

e16114 Background: In the observational REFINE study of regorafenib (NCT03289273) in pts with uHCC, treatment-emergent adverse events (TEAEs) were consistent those reported global, phase 3 RESORCE trial (YJ Kim, ILCA 2022). Median overall survival (OS) global cohort was 13.2 months; here, we present final analysis US pt cohort. Methods: an international, prospective, multicenter that enrolled uHCC for whom a decision to treat made by treating physician prior enrollment, according local health authority approved label. The primary objectives safety, including incidence TEAEs (MedDRA v25) and dose modifications due TEAEs. Secondary endpoints included OS, progression-free (PFS), duration treatment (DoT). Results: Of 1005 evaluable from cohort, 65 (6%) USA. 78% male, 69% had ECOG PS 0/1, population younger than (54% aged <65 years vs 45% cohort). most common HCC etiology hepatitis C (52% 24% cohort) second alcohol use (34% 25% A lower proportion extrahepatic spread compared (48% 59%), while similar vascular invasion (both 34%). Use immune checkpoint inhibitors more frequent (25% 10%). Most (88%) received sorafenib, (96%). However, relatively as second-line therapy (71% 84% Transarterial chemoembolization non-systemic (42%). DoT 3.1 months (range <0.1–24.6 months) 3.7 <0.1–38.9 91% apart diarrhea hand–foot skin reaction (HFSR) pts, incidences comparable addition, resulting modification (38% OS 11.4 (95% CI 8.4, 18.0), (median [95% 11.6, 14.8]). Conclusions: real-world confirmed safety effectiveness broader trial. findings remained uHCC. Clinical information: NCT03289273 . [Table: see text]