Opportunities and Approaches to Optimising Advanced Cholangiocarcinoma Outcomes in the Era of Targeted Therapies: A Narrative Review.

IF 3.4 Q2 ONCOLOGY
Oncology and Therapy Pub Date : 2025-12-01 Epub Date: 2025-10-08 DOI:10.1007/s40487-025-00370-2
Teresa Macarulla, Cindy Neuzillet, Gerald W Prager, Lorenza Rimassa, John Bridgewater
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引用次数: 0

Abstract

Cholangiocarcinoma (CCA) represents a diverse group of malignancies. It is often identified at a late stage after the opportunity for curable resection has passed. An international educational meeting on CCA was held in Barcelona in September 2024. The meeting described the challenges with obtaining accurate epidemiological estimates for CCA because of misdiagnosis and marked regional differences, reflecting the prevalence of socioeconomic risk factors. Early-stage CCA is usually asymptomatic, and when symptoms appear, they are nonspecific. Diagnosis and treatment planning should involve a multidisciplinary team (MDT) from the outset. The European Society for Medical Oncology (ESMO) guidelines recommend molecular testing using next-generation sequencing when advanced disease is diagnosed. Biopsy sampling is technically challenging; if insufficient quality tissue is collected for molecular testing, liquid biopsy can be used. If the tumour is unresectable, the recommended first-line treatment is cisplatin + gemcitabine + durvalumab a programmed cell death ligand 1 [PD-L1] inhibitor or pembrolizumab a programmed cell death protein 1 [PD-1] inhibitor. The development of targeted therapies has led to these treatments being recommended as second- or third-line therapy for patients with actionable gene alterations, while 5-fluorouracil-based chemotherapy is recommended for those without. Robust data support the use of ivosidenib in patients with IDH1 mutations (phase 3), and phase 2 trials showed efficacy of pemigatinib and futibatinib for patients with FGFR2 gene fusions, trastuzumab deruxtecan and zanidatamab for patients with HER2 overexpression/amplification, and dabrafenib + trametinib for patients with BRAFV600E mutations. It is hoped that wider dissemination of the content from this meeting will improve outcomes of patients with CCA by encouraging earlier referral, increasing the use of early molecular testing, an MDT approach, and maximising the use of targeted therapies. Continued efforts to raise awareness, implement education outreach opportunities, and involve patient advocacy groups are encouraged to improve CCA outcomes.

Abstract Image

Abstract Image

Abstract Image

在靶向治疗时代优化晚期胆管癌预后的机会和方法:叙述性回顾。
胆管癌(CCA)是一种不同类型的恶性肿瘤。它通常在可治愈的切除机会过去后的晚期才被发现。2024年9月在巴塞罗那举行了CCA国际教育会议。会议描述了由于误诊和明显的区域差异,反映了社会经济风险因素的普遍存在,在获得准确的CCA流行病学估计方面所面临的挑战。早期CCA通常无症状,当症状出现时,它们是非特异性的。诊断和治疗计划从一开始就应该包括一个多学科小组(MDT)。欧洲肿瘤医学学会(ESMO)指南建议在诊断出晚期疾病时使用新一代测序进行分子检测。活检取样在技术上具有挑战性;如果收集到的质量不足的组织用于分子检测,可以使用液体活检。如果肿瘤不可切除,推荐的一线治疗是顺铂+吉西他滨+ durvalumab(程序性细胞死亡配体1 [PD-L1]抑制剂)或pembrolizumab(程序性细胞死亡蛋白1 [PD-1]抑制剂)。靶向治疗的发展导致这些治疗被推荐为具有可操作基因改变的患者的二线或三线治疗,而基于5-氟尿嘧啶的化疗被推荐用于无可操作基因改变的患者。强有力的数据支持IDH1突变患者使用ivosidenib(3期),2期试验显示pemigatinib和futibatinib对FGFR2基因融合患者有效,曲妥珠单抗deruxtecan和zanidatamab对HER2过表达/扩增患者有效,dabrafenib + trametinib对BRAFV600E突变患者有效。希望此次会议内容的广泛传播将通过鼓励早期转诊、增加早期分子检测、MDT方法的使用和最大化靶向治疗的使用来改善CCA患者的预后。鼓励继续努力提高认识,实施教育推广机会,并让患者倡导团体参与进来,以改善CCA结果。
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来源期刊
CiteScore
3.40
自引率
0.00%
发文量
31
审稿时长
6 weeks
期刊介绍: Now indexed in PubMed Aims and Scope Oncology and Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality pre-clinical, clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Oncology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model this allows for the rapid and efficient communication of the latest research and reviews, allowing the advancement of clinical therapies. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning that authors will always have a personal point of contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE, GPP and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. We also encourage pre-submission enquiries and are always happy to provide a confidential assessment of manuscripts. Digital features and plain language summaries Oncology and Therapy offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by key summary points, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article. The journal also provides the option to include various types of digital features including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations. All additional features are peer reviewed to the same high standard as the article itself. If you consider that your paper would benefit from the inclusion of a digital feature, please let us know. Our editorial team are able to create high-quality slide decks and infographics in-house, and video abstracts through our partner Research Square, and would be happy to assist in any way we can. For further information about digital features, please contact the journal editor (see ‘Contact the Journal’ for email address), and see the ‘Guidelines for digital features and plain language summaries’ document under ‘Submission guidelines’. Preprints We encourage posting of preprints of primary research manuscripts on preprint servers, authors'' or institutional websites, and open communications between researchers whether on community preprint servers or preprint commenting platforms. Posting of preprints is not considered prior publication and will not jeopardize consideration in our journals. Please see here for further information on preprint sharing: https://www.springer.com/gp/authors-editors/journal-author/journal-author-helpdesk/submission/1302#c16721550 Peer Review Process Upon submission, manuscripts are assessed by the editorial team to ensure they fit within the aims and scope of the journal and are also checked for plagiarism. All suitable submissions are then subject to a comprehensive single-blind peer review. Reviewers are selected based on their relevant expertise and publication history in the subject area. The journal has an extensive pool of editorial and advisory board members who have been selected to assist with peer review based on the afore-mentioned criteria. At least two extensive reviews are required to make the editorial decision, with the exception of some article types such as Commentaries, Editorials and Letters which are generally reviewed by one member of the Editorial Board. Where reviewer recommendations are conflicted, the editorial board will be contacted for further advice and a presiding decision. Manuscripts are then either accepted, rejected or authors are required to make major or minor revisions (both reviewer comments and editorial comments may need to be addressed). Once a revised manuscript is re-submitted, it is assessed along with the responses to reviewer comments and if it has been adequately revised it will be accepted for publication. Accepted manuscripts are then copyedited and typeset by the production team before online publication. Appeals against decisions following peer review are considered on a case by case basis and should be sent to the journal editor. Copyright Oncology and Therapy''s content is published open access under the Creative Commons Attribution-Noncommercial License, which allows users to read, copy, distribute, and make derivative works for non-commercial purposes from the material, as long as the author of the original work is cited. The author assigns the exclusive right to any commercial use of the article to Springer. For more information about the Creative Commons Attribution-Noncommercial License, click here: http://creativecommons.org/licenses/by-nc/4.0 Publication Fees Upon acceptance of an article, authors will be required to pay the mandatory Rapid Service Fee of £3650/€4500/$5100. The journal will consider fee discounts for developing countries and this is decided on a case by case basis. Open Access All articles published by Oncology and Therapy are published open access Contact For more information about the journal, including pre-submission enquiries, please contact managing editor Lydia Alborn at lydia.alborn@springer.com.
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