BRAF 抑制剂被批准用于治疗 BRAFV600 突变的转移性黑色素瘤、非小细胞肺癌 (NSCLC)、埃尔德海姆-切斯特病 (ECD) 和甲状腺癌。我们在此报告 AcSé 维罗非尼篮子研究中对除 BRAFV600 突变黑色素瘤和 NSCLC 之外的各种 BRAF 突变晚期肿瘤患者给予单药维罗非尼的有效性、安全性和长期结果。除 BRAFV600E 以外的晚期肿瘤患者黑色素瘤和标准治疗后进展的患者有资格纳入九个队列（包括一个杂项队列），并接受每日两次口服维莫非尼 960 mg。主要终点是通过贝叶斯设计估计的客观缓解率 (ORR)。次要结局是疾病控制率、缓解持续时间、无进展生存期 (PFS)、总生存期 (OS) 和维莫非尼安全性。 共有 98 名患有各种实体癌或血液癌的晚期患者，其中 88 名患有 BRAFV600 突变，10 名患有 BRAFV600 突变。 BRAFnonV600 突变也包括在内。中位随访时间为 47.7 个月。贝叶斯估计的 ORR 在毛细胞白血病 (HCL) 中为 89.7%，在胶质母细胞瘤队列中为 33.3%，在胆管癌中为 18.2%，在 ECD 中为 80.0%，在卵巢癌中为 50.0%，在黄色星形细胞瘤中为 50.0%，在神经节胶质瘤中为 66.7%，在肉瘤中 60.0%。整个系列的中位 PFS 为 8.8 个月。 12、24 和 36 个月的 PFS 率分别为 42.2%、23.8% 和 17.9%。总体而言，54 名患者死亡，中位 OS 为 25.9 个月，预计 4 年 OS 为 40%。不良事件与之前报道的维莫非尼不良事件类似。在许多具有 BRAF 突变的肿瘤中观察到缓解和延长的 PFS，包括 HCL、ECD、卵巢癌、神经胶质瘤、神经节胶质瘤和肉瘤。尽管并非所有癌症类型都有反应，但维莫非尼是一种不可知的癌症基因疗法。版权所有 © 2023 作者。由爱思唯尔有限公司出版。保留所有权利。

BRAF inhibitors are approved in BRAFV600-mutated metastatic melanoma, non-small-cell lung cancer (NSCLC), Erdheim-Chester disease (ECD), and thyroid cancer. We report here the efficacy, safety, and long-term results of single-agent vemurafenib given in the AcSé vemurafenib basket study to patients with various BRAF-mutated advanced tumours other than BRAFV600-mutated melanoma and NSCLC.Patients with advanced tumours other than BRAFV600E melanoma and progressing after standard treatment were eligible for inclusion in nine cohorts (including a miscellaneous cohort) and received oral vemurafenib 960 mg two times daily. The primary endpoint was the objective response rate (ORR) estimated with a Bayesian design. The secondary outcomes were disease control rate, duration of response, progression-free survival (PFS), overall survival (OS), and vemurafenib safety.A total of 98 advanced patients with various solid or haematological cancers, 88 with BRAFV600 mutations and 10 with BRAFnonV600 mutations, were included. The median follow-up duration was 47.7 months. The Bayesian estimate of ORR was 89.7% in hairy cell leukaemias (HCLs), 33.3% in the glioblastomas cohort, 18.2% in cholangiocarcinomas, 80.0% in ECD, 50.0% in ovarian cancers, 50.0% in xanthoastrocytomas, 66.7% in gangliogliomas, and 60.0% in sarcomas. The median PFS of the whole series was 8.8 months. The 12-, 24-, and 36-month PFS rates were 42.2%, 23.8%, and 17.9%, respectively. Overall, 54 patients died with a median OS of 25.9 months, with a projected 4-year OS of 40%. Adverse events were similar to those previously reported with vemurafenib.Responses and prolonged PFS were observed in many tumours with BRAF mutations, including HCL, ECD, ovarian carcinoma, gliomas, ganglioglioma, and sarcomas. Although not all cancer types responded, vemurafenib is an agnostic oncogene therapy of cancers.Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved.