索拉非尼和雷戈非尼治療晚期肝細胞癌的現實世界系統性序

StephenW

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.

Keywords: Effectiveness; Hepatocellular carcinoma; Real-world study; Safety.

StephenW

索拉非尼和雷戈非尼治疗晚期肝细胞癌的现实世界系统性序贯治疗：韩国的多中心回顾性研究
李敏珍1，宋元昌1，李智勋2 3，李英新1，宋范祖4，延锡瑞1，亨俊炎1，桑允黄5，玄雄利6，杨昌7 ，Jae Young Jang 7
隶属关系
隶属关系

    1个
    高丽大学医学院内科，韩国首尔。
    2
    高丽大学医学院内科，韩国首尔。 [email protected]。
    3
    高丽大学医学院九老医院消化内科，消化内科，韩国首尔九老区九老洞九老洞吉97。 [email protected]。
    4
    韩国国土省全顺市全南郡和顺郡和顺邑徐养路264号，和南国立大学医学院，朝南国立大学和顺医院消化内科和肝病科，韩国58128。 [email protected]。
    5
    韩国釜山市放射医学医学研究所内科和消化道癌症中心。
    6
    延世大学医学院江南遣散医院内科，韩国首尔。
    7
    韩国首尔市淳春市大学医学院首尔医院内科。

    PMID：32749658 DOI：10.1007 / s10637-020-00977-4

抽象

背景/目的雷戈非尼经过RESORCE III期临床试验已被批准为肝细胞癌（HCC）患者的二线全身治疗。这项研究分析了现实世界的数据，以评估雷戈非尼与RESORCE试验相比的临床有效性和安全性。方法这项多中心队列研究包括索拉非尼后接受regorafenib治疗的HCC患者（n = 133）。我们评估了接受雷戈非尼患者的进展时间（TTP），无进展生存期（PFS），总生存期（OS）和安全性以及预后指标。结果中位年龄为60岁，男性为81.2％。乙型肝炎病毒感染（68.4％）是最常见的病因。大多数患者被归类为Child-Pugh A（98.5％），并有肝外转移（84％）和血管浸润（45.1％）。与RESORCE试验相比，该研究显示了相似的特征，除了更常见的乙型肝炎病因和更多的血管或肝外受累。评估反应的客观反应率为12.5％（n = 112）；疾病控制率为34.8％。随访期间有38例患者死亡。使用雷戈非尼时，中位OS​​，PFS和TTP分别为10.0、2.7和2.6个月。在索拉非尼给药后的探索性分析中，中位OS​​为25.8个月。缓解率和生存率与RESORCE试验相当。 Child-Pugh评分> 5，甲胎蛋白> 400 ng / ml和索拉非尼≥中位数的TTP与OS独立相关。结论瑞戈非尼这一实词研究显示出与RESORCE试验相当的有效性和安全性。瑞戈非尼改善了先前索拉非尼治疗期间TTP延长的患者的预后。

关键字：有效性；肝细胞癌；现实世界的研究；安全。