Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea.
Liverpool CR UK/NIHR Experimental Cancer Medicine Centre, and The Clatterbridge Cancer Centre, Liverpool, UK.
Clin Drug Investig. 2021 Sep;41(9):795-808. doi: 10.1007/s40261-021-01063-0. Epub 2021 Aug 5.
Hepatocellular carcinoma (HCC) is the fourth leading cause of cancer-related mortality worldwide. Despite recent advances, more effective therapeutic options for patients with advanced HCC are still required. The aim of this Phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study (NCT02528643) was to investigate the potential benefit of enzalutamide in the treatment of patients with advanced HCC.
Patients aged ≥ 18 years diagnosed with advanced HCC (Barcelona Clinic Liver Cancer stage B or C and Child-Pugh class A at screening who had progressed on, or were intolerant to, sorafenib or other anti-vascular endothelial growth factor therapies) were randomized 2:1 to receive either enzalutamide 160 mg daily or placebo. The primary endpoint was overall survival (OS); secondary endpoints included progression-free survival (PFS) and safety.
In total, 165 patients were randomized to enzalutamide (n = 110) or placebo (n = 55). The hazard ratio (HR) (95% confidence interval [CI]) for OS was 1.15 (0.774-1.696) and median OS was 7.8 months and 7.7 months for enzalutamide and placebo, respectively. The HR (95% CI) for PFS was 1.04 (0.732-1.474) and median PFS was 2.2 months and 1.9 months for enzalutamide and placebo, respectively. The overall frequency of treatment-emergent adverse events (TEAEs) was broadly similar between the groups: 105 (98.1%) enzalutamide patients experienced ≥1 TEAEs compared with 49 (89.1%) placebo patients.
The results of this study indicate that enzalutamide does not provide a benefit in patients with advanced HCC. No unexpected safety findings were observed in the trial. CLINICALTRIALS.
NCT02528643.
肝细胞癌（HCC）是全球癌症相关死亡的第四大原因。尽管最近取得了进展，但仍需要为晚期 HCC 患者提供更有效的治疗选择。这项 2 期、多中心、多国、随机、双盲、安慰剂对照研究（NCT02528643）的目的是研究恩扎鲁胺在治疗晚期 HCC 患者中的潜在益处。
年龄≥18 岁、诊断为晚期 HCC（巴塞罗那临床肝癌分期 B 或 C 期，筛选时丙氨酸氨基转移酶/天冬氨酸氨基转移酶[ALT/AST]正常，Child-Pugh 分级为 A 级，且索拉非尼或其他抗血管内皮生长因子治疗后进展或不耐受）的患者按 2:1 随机分组，分别接受恩扎鲁胺 160mg 每日或安慰剂治疗。主要终点是总生存期（OS）；次要终点包括无进展生存期（PFS）和安全性。
共有 165 例患者被随机分为恩扎鲁胺（n=110）或安慰剂（n=55）组。OS 的风险比（HR）（95%置信区间[CI]）为 1.15（0.774-1.696），恩扎鲁胺和安慰剂的中位 OS 分别为 7.8 个月和 7.7 个月。PFS 的 HR（95%CI）为 1.04（0.732-1.474），恩扎鲁胺和安慰剂的中位 PFS 分别为 2.2 个月和 1.9 个月。两组治疗中出现的不良事件（TEAEs）的总体发生率大致相似：105（98.1%）例恩扎鲁胺患者发生≥1 例 TEAEs，49（89.1%）例安慰剂患者发生≥1 例 TEAEs。试验中未观察到意外的安全性发现。
临床试验.gov 标识符：NCT02528643。
