Yoo Changhoon, Byeon Seonggyu, Bang Yeonghak, Cheon Jaekyung, Kim Jin W, Kim Jee H, Chon Hong J, Kang Beodeul, Kang Myoung J, Kim Ilhwan, Hwang Jun-Eul, Kang Jung H, Lee Myung A, Hong Jung Y, Lim Ho Y, Ryoo Baek-Yeol
Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Liver Int. 2020 Sep;40(9):2263-2271. doi: 10.1111/liv.14496. Epub 2020 May 25.
BACKGROUND & AIMS: Regorafenib demonstrated a clinical benefit for patients with unresectable hepatocellular carcinoma (uHCC) in the phase III RESORCE trial. Considering the heterogeneity of uHCC and discrepancies in its characteristics between prospective trials and daily practice, real-life evidence is necessary.
This multicentre, retrospective analysis was performed by the Korean Cancer Study Group. In total, 440 patients who received regorafenib between January 2017 and November 2019 were identified in nine tertiary referral hospitals in Korea.
All patients received prior sorafenib, and the median time-to-progression (TTP) on sorafenib was 3.9 months (range, 0.2-71.6). Regorafenib was used as the second, third and fourth to seventh lines of therapy in 305 (69.3%), 115 (26.1%) and 20 (4.5%) patients respectively. According to the RECIST v1.1, the overall response rate was 7.7% (n = 34), and the median progression-free survival (PFS) and overall survival (OS) were 3.2 (95% CI, 2.8-3.5) and 12.1 (95% CI, 9.7-14.5) months respectively. Immune checkpoint inhibitors (ICIs) were given in 115 patients (26.1%) prior to regorafenib. There were no differences in PFS and OS with regorafenib according to the prior use of ICIs (PFS, P = .61; OS, P = .63). The occurrence of hand-foot skin reaction (HFSR) was associated with a better OS (P < .001).
The real-life clinical outcomes of regorafenib for patients who progressed on prior systemic therapy including ICIs were consistent with the phase III trial results. HFSR was significantly associated with better OS with regorafenib.
在III期RESORCE试验中，瑞戈非尼对不可切除肝细胞癌（uHCC）患者显示出临床获益。鉴于uHCC的异质性以及前瞻性试验与日常实践中其特征的差异，现实生活中的证据很有必要。
这项多中心回顾性分析由韩国癌症研究组开展。2017年1月至2019年11月期间，在韩国9家三级转诊医院中，共确定了440例接受瑞戈非尼治疗的患者。
所有患者之前均接受过索拉非尼治疗，索拉非尼治疗的中位疾病进展时间（TTP）为3.9个月（范围0.2 - 71.6个月）。瑞戈非尼分别用于305例（69.3%）、115例（26.1%）和20例（4.5%）患者的二线、三线以及四线至七线治疗。根据RECIST v1.1标准，总缓解率为7.7%（n = 34），中位无进展生存期（PFS）和总生存期（OS）分别为3.2个月（95%CI，2.8 - 3.5）和12.1个月（95%CI，9.7 - 14.5）。115例（26.1%）患者在使用瑞戈非尼之前接受过免疫检查点抑制剂（ICI）治疗。根据ICI的既往使用情况，使用瑞戈非尼后的PFS和OS无差异（PFS，P = 0.61；OS，P = 0.63）。手足皮肤反应（HFSR）的发生与更好的OS相关（P < 0.001）。
瑞戈非尼用于包括ICI在内的既往全身治疗后疾病进展患者的现实生活临床结局与III期试验结果一致。HFSR与瑞戈非尼治疗更好的OS显著相关。
