Reichinger Andreas, Essl Leo, Kerschner Paul, Burghofer Jonathan, Webersinke Gerald, Rumpold Holger, Doleschal Bernhard
Department of Internal Medicine I for Hematology with Stem Cell Transplantation, Hemostaseology, and Medical Oncology, Ordensklinikum Linz - Barmherzige Schwestern Site, Seilerstaette 4, 4020 Linz,Austria.
Medical Faculty, Johannes Kepler University Linz, Altenberger Strasse 69, 4040 Linz, Austria.
Oncologist. 2024 Dec 6;29(12):1090-1094. doi: 10.1093/oncolo/oyae265.
Biliary tract cancers (BTCs) are a diverse group of malignancies with varied genetic backgrounds. The prevalence of intrahepatic cholangiocarcinoma (iCC) is increasing, particularly in Western countries. Despite advancements in treatments, the prognosis for BTC remains poor. Recent molecular profiling has revealed that up to 40% of iCC cases have targetable genetic alterations. MET amplification, although rare, presents a significant target for therapy.
A 25-year-old female with a history of ulcerative colitis presented with shoulder pain and a positron emission tomography-computed tomography (PET-CT) scan revealed an enlarged liver and multiple metastases. Histopathological analysis diagnosed poorly differentiated adenocarcinoma. First-line therapy with Cisplatin, Gemcitabine, and Durvalumab resulted in disease progression. Molecular profiling identified a TP53 mutation and MET amplification. Based on these findings, Tepotinib was initiated. Tepotinib treatment led to a significant reduction in tumor size and normalization of CA 19-9 levels within 2 months, achieving a complete metabolic remission lasting up to 17 months. The treatment was well tolerated with minimal side effects.
MET-amplified BTCs are exceedingly rare, and evidence for targeted treatment is limited. This case demonstrates the efficacy of Tepotinib in a young patient with MET-amplified iCC, showing a long-term response and suggesting a potential new standard treatment option for this molecularly defined entity. This case also highlights the aggressive nature of MET-amplified tumors and the need for targeted second-line therapies.
Tepotinib showed remarkable efficacy in treating MET-amplified intrahepatic cholangiocarcinoma, underscoring the importance of molecular profiling in BTCs and suggesting a potential new therapeutic approach for this rare cancer subtype.
胆道癌（BTCs）是一组具有不同遗传背景的恶性肿瘤。肝内胆管癌（iCC）的患病率正在上升，尤其是在西方国家。尽管治疗取得了进展，但BTC的预后仍然很差。最近的分子分析显示，高达40%的iCC病例存在可靶向的基因改变。MET扩增虽然罕见，但却是一个重要的治疗靶点。
一名25岁有溃疡性结肠炎病史的女性出现肩部疼痛，正电子发射断层扫描-计算机断层扫描（PET-CT）显示肝脏肿大并伴有多处转移。组织病理学分析诊断为低分化腺癌。一线使用顺铂、吉西他滨和度伐利尤单抗治疗导致疾病进展。分子分析发现TP53突变和MET扩增。基于这些发现，开始使用替泊替尼治疗。替泊替尼治疗在2个月内使肿瘤大小显著缩小，CA 19-9水平恢复正常，实现了长达17个月的完全代谢缓解。该治疗耐受性良好，副作用极小。
MET扩增的BTC极其罕见，靶向治疗的证据有限。本病例证明了替泊替尼在一名MET扩增的iCC年轻患者中的疗效，显示出长期反应，并为这个分子定义的实体提出了一种潜在的新的标准治疗选择。本病例还突出了MET扩增肿瘤的侵袭性以及对靶向二线治疗的需求。
替泊替尼在治疗MET扩增的肝内胆管癌方面显示出显著疗效，强调了分子分析在BTCs中的重要性，并为这种罕见的癌症亚型提出了一种潜在的新治疗方法。
