Department of Gastrointestinal Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Oncologist. 2023 Apr 6;28(4):327-332. doi: 10.1093/oncolo/oyac273.
Limited data from small series have suggested that brain metastases from biliary tract cancers (BrM-BTC) affect ≤2% of patients with BTC. We sought to review our experience with patients with BrM-BTC and to identify associations of tumor-related molecular alterations with outcomes.
A retrospective review of patients with BTC seen at a tertiary referral center from 2005 to 2021 was performed; patients with BrM-BTC were identified, and clinical and molecular data were collected.
Twenty-one of 823 patients with BTC (2.6%) developed BrM. For patients with BrM-BTC, median follow-up time was 27.9 months after primary BTC diagnosis and 3.1 months after BrM diagnosis. Median time from primary diagnosis to diagnosis of BrM was 14.4 [range, 1.1-66.0] months. Median overall survival (OS) from primary diagnosis was 31.5 [2.9-99.8] months and median OS from BrM diagnosis was 4.2 [0.2-33.8] months. Patients who underwent BrM-directed therapy trended toward longer OS following BrM diagnosis than patients receiving supportive care only (median 6.5 vs 0.8 months, P = .060). The BrM-BTC cohort was enriched for BRAF (30%), PIK3CA (25%), and GNAS (20%) mutations. patients with BrM-BTC with BRAF mutations trended toward longer OS following BrM diagnosis (median 13.1 vs 4.2 months, P = .131).
This is the largest series of patients with BrM-BTC to date and provides molecular characterization of this rare subgroup of patients with BTC. Patients with BrM-BTC may be more likely to have BRAF mutations. With advances in targeted therapy for patients with BTC with actionable mutations, continued examination of shifting patterns of failure, with emphasis on BrM, is warranted.
来自小系列的有限数据表明，胆道癌（BTC）的脑转移（BrM-BTC）影响≤2%的 BTC 患者。我们试图回顾我们在 BrM-BTC 患者中的经验，并确定肿瘤相关分子改变与结局的关联。
对 2005 年至 2021 年在三级转诊中心就诊的 BTC 患者进行回顾性研究；确定了 BrM-BTC 患者，并收集了临床和分子数据。
823 例 BTC 患者中有 21 例（2.6%）发生 BrM-BTC。对于 BrM-BTC 患者，从原发性 BTC 诊断后中位随访时间为 27.9 个月，从 BrM 诊断后中位随访时间为 3.1 个月。从原发性诊断到 BrM 诊断的中位时间为 14.4 [范围，1.1-66.0] 个月。从原发性诊断的中位总生存期（OS）为 31.5 [2.9-99.8] 个月，从 BrM 诊断的中位 OS 为 4.2 [0.2-33.8] 个月。与仅接受支持性治疗的患者相比，接受 BrM 定向治疗的患者在 BrM 诊断后的 OS 趋势更长（中位 6.5 与 0.8 个月，P =.060）。BrM-BTC 队列中富含 BRAF（30%）、PIK3CA（25%）和 GNAS（20%）突变。BrM-BTC 患者中具有 BRAF 突变的患者在 BrM 诊断后的 OS 趋势更长（中位 13.1 与 4.2 个月，P =.131）。
这是迄今为止最大的 BrM-BTC 患者系列，提供了 BTC 这一罕见亚组患者的分子特征。BrM-BTC 患者可能更有可能发生 BRAF 突变。随着针对具有可操作突变的 BTC 患者的靶向治疗进展，需要继续检查失败模式的变化，重点是 BrM。
