依维莫司和来曲唑联合或不联合瑞博西尼治疗复发性子宫内膜癌的疗效：一项2期随机临床试验。
完整摘要 · 中文译文
在这项II期试验中，子宫内膜癌（EC）患者被随机分配接受依维莫司/来曲唑联合瑞博西尼（试验组）或不联合瑞博西尼（对照组）治疗（ClinicalTrials.gov 标识号：NCT03008408）。主要终点为无进展生存期（PFS）。次要终点包括毒性、总生存期（OS）、客观缓解率（ORR）和临床获益率（CBR）。对于全人群，PFS（HR 0.78，95% CI 0.4-1.4，p = 0.38）、OS（HR 0.8，95% CI 0.4-1.5，p = 0.46）、ORR（44.1% vs 25.7%，p = 0.11）或毒性相关治疗中止（4例 [8.6%] vs 3例 [11.4%]，p = 0.22）均无差异。在既往接受过内分泌治疗的患者中，试验组的ORR更高（61.5% vs 0.0%，p < 0.001）。在试验组内，与非CTNNB1突变肿瘤相比，CTNNB1突变肿瘤与改善的PFS（HR 0.27，95% CI 0.1-0.7，p = 0.006）和OS（HR 0.24，95% CI 0.1-0.7，p = 0.004）相关。尽管全人群无生存改善，但在依维莫司/来曲唑基础上加用瑞博西尼可能提高既往接受过内分泌治疗患者的疗效，且CTNNB1突变肿瘤病例可能获得更持久的缓解。
完整摘要 · 英文原文
In this phase II trial, patients with endometrial cancer (EC) were randomized to everolimus/letrozole with (experimental arm) or without ribociclib (control arm) (ClinicalTrials.gov Identifier: NCT03008408). The primary endpoint was progression-free survival (PFS). Secondary endpoints included toxicity, overall survival (OS), objective response rate (ORR), and clinical benefit rate (CBR). For all-comers, there was no difference in PFS (HR 0.78, 95% CI 0.4-1.4, p = 0.38), OS (HR 0.8, 95% CI 0.4-1.5, p = 0.46), ORR (44.1% vs 25.7%, p = 0.11), or toxicity-related treatment discontinuation (4 [8.6%] vs 3 [11.4%], p = 0.22). Among those with prior endocrine therapy, ORR was higher in the experimental arm (61.5% vs 0.0%, p < 0.001). Within the experimental arm, CTNNB1mut tumors were associated with improved PFS (HR 0.27, 95% CI 0.1-0.7, p = 0.006) and OS (HR 0.24, 95% CI 0.1-0.7, p = 0.004) compared to those with non-CTNNB1mut tumors. Despite no survival improvement in all-comers, addition of ribociclib to everolimus/letrozole may improve efficacy in patients with prior endocrine therapy and cases with CTNNB1mut tumors may have prolonged response.