Real-world systemic sequential therapy with sorafenib and regorafenib for advanced hepatocellular carcinoma: a multicenter retrospective study in Korea
Min Jin Lee 1 , Sung Won Chang 1 , Ji Hoon Kim 2 3 , Young-Sun Lee 1 , Sung Bum Cho 4 , Yeon Seok Seo 1 , Hyung Joon Yim 1 , Sang Youn Hwang 5 , Hyun Woong Lee 6 , Young Chang 7 , Jae Young Jang 7
Affiliations
Affiliations
1
Department of Internal Medicine, Korea University College of Medicine, Seoul, Korea.
2
Department of Internal Medicine, Korea University College of Medicine, Seoul, Korea. [email protected].
3
Department of Internal Medicine, Division of Gastroenterology and Hepatology, Guro Hospital, Korea University College of Medicine, 97, Guro-Dong Gil, Guro-Dong, Guro-Ku, Seoul, 08308, Korea. [email protected].
4
Department of Gastroenterology and Hepatology, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, 264 Seoyang-ro, Hwasun-eup, Hwasun-gun, Jeollanam-do, Hwasun, 58128, Republic of Korea. [email protected].
5
Department of Internal Medicine and Gastrointestinal Cancer Center, Dongnam Institute of Radiological and Medical Sciences, Busan, Korea.
6
Department of Internal Medicine, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, Korea.
7
Department of Internal Medicine, Soonchunhyang University College of Medicine Seoul Hospital, Seoul, Korea.
PMID: 32749658 DOI: 10.1007/s10637-020-00977-4
Abstract
Background/Aims Regorafenib has been approved as a second-line systemic therapy for hepatocellular carcinoma (HCC) patients after the phase III RESORCE trial. This study analyzed real-world data to assess the clinical effectiveness and safety of regorafenib compared to the RESORCE trial. Methods This multicenter cohort study included HCC patients treated with regorafenib after sorafenib (n = 133). We evaluated the time to progression (TTP), progression-free survival (PFS), overall survival (OS), and safety in patients receiving regorafenib along with the predictors of prognosis. Results The median age was 60 years and 81.2% patients were men. Hepatitis B virus infection (68.4%) was the commonest etiology. Most patients were classified as Child-Pugh A (98.5%) and had extrahepatic metastasis (84%) and vascular invasion (45.1%). This study demonstrated similar characteristics apart from more frequent hepatitis B etiology and more vascular or extrahepatic involvement compared with the RESORCE trial. An objective response rate of 12.5% was obtained for response assessment (n = 112); the disease control rate was 34.8%. Thirty-eight patients died during follow-up. With regorafenib, the median OS, PFS, and TTP were 10.0, 2.7, and 2.6 months, respectively. In the exploratory analysis after sorafenib administration, the median OS was 25.8 months. The rate of response and survival were comparable to those in the RESORCE trial. Child-Pugh score > 5, alpha-fetoprotein > 400 ng/ml, and TTP for sorafenib ≥ median were independently associated with OS. Conclusions This real-word regorafenib study showed comparable effectiveness and safety to the RESORCE trial. Regorafenib improves the prognosis of patients with prolonged TTP during previous sorafenib therapy.