Department of Medicine III and Comprehensive Cancer Center, University Hospital, Klinikum Grosshadern, Ludwig-Maximilians-University of Munich, Marchioninistr. 15, 81377, Munich, Germany.
German Cancer Consortium (DKTK), Partner Site Munich, Munich, Germany.
Target Oncol. 2023 Sep;18(5):767-776. doi: 10.1007/s11523-023-00985-3. Epub 2023 Aug 18.
With the rising importance of precision oncology in biliary tract cancer (BTC), the aim of this retrospective single-center analysis was to describe the clinical and molecular characteristics of patients with BTC who underwent comprehensive genomic profiling (CGP) and were discussed in the CCCMunich molecular tumor board (MTB).
In this single-center observational study, we included BTC patients with intrahepatic cholangiocarcinoma (iCCA), extrahepatic CCA (eCCA), and gallbladder cancer (GB), who had been discussed in the institutional MTB from May 29, 2017, to July 25, 2022. Patients were followed up until 31 January 2023. Data were retrospectively collected by review of medical charts, and MTB recommendation.
In total, 153 cases were registered to the MTB with a median follow-up of 15 months. Testing was successful in 81.7% of the patients. CGP detected targetable alterations in 35.3% of our BTC patients (most commonly ARID1A/ERBB2/IDH1/PIK3CA/BRAF-mutations and FGFR2-fusions). Recommendations for molecularly guided therapy were given in 46.4%. Of those, treatment implementation of targeted therapy followed in 19.4%. In patients receiving the recommended treatment, response rate was 57% and median overall survival was 19 months (vs 8 months in the untreated cohort). The progression-free survival ratio of 1.45 suggest a clinical benefit of molecularly guided treatment.
In line with previous work, our series demonstrates feasibility and clinical utility of comprehensive genomic profiling in BTC patients. With the growing number of targeted agents with clinical activity in BTC, CGP should become standard of care in the management of this group of patients.
随着精准肿瘤学在胆管癌（BTC）中的重要性不断提升，本回顾性单中心分析旨在描述在 CCCMunich 分子肿瘤委员会（MTB）中讨论并接受全面基因组分析（CGP）的 BTC 患者的临床和分子特征。
在这项单中心观察性研究中，我们纳入了在机构 MTB 中讨论的肝内胆管癌（iCCA）、肝外胆管癌（eCCA）和胆囊癌（GB）的 BTC 患者。这些患者的入组时间为 2017 年 5 月 29 日至 2022 年 7 月 25 日，随访截止日期为 2023 年 1 月 31 日。通过回顾病历和 MTB 建议收集数据。
共有 153 例患者在 MTB 中登记，中位随访时间为 15 个月。81.7%的患者检测成功。CGP 检测到 35.3%的 BTC 患者存在可靶向改变（最常见的是 ARID1A/ERBB2/IDH1/PIK3CA/BRAF 突变和 FGFR2 融合）。46.4%的患者推荐进行分子指导治疗。其中，19.4%的患者接受了靶向治疗。在接受推荐治疗的患者中，反应率为 57%，中位总生存期为 19 个月（未接受治疗的患者为 8 个月）。1.45 的无进展生存率提示分子指导治疗具有临床获益。
与既往研究一致，本研究系列证实了全面基因组分析在 BTC 患者中的可行性和临床应用价值。随着具有临床活性的靶向药物在 BTC 中的数量不断增加，CGP 应成为该类患者治疗的标准。
