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How to foster new treatment development in ultra-rare tumours? Joint EMA-EORTC multi-stakeholder workshops on ultra-rare sarcomas as a model for rare cancers 如何促进超罕见肿瘤的新疗法发展?EMA-EORTC多方利益相关者联合研讨会:超罕见肉瘤作为罕见癌症的模型
IF 10.5 1区 医学 Q1 ONCOLOGY Pub Date : 2025-08-06 DOI: 10.1016/j.ctrv.2025.103003
Silvia Stacchiotti , Pan Pantziarka , Hugh Leonard , Caroline Voltz , Laura Abatedaga , Gauthier Bouche , Christelle Bouygues , Judith V.M.G. Bovee , Winette T.A. Van der Graaf , Teresa De Rojas , Lorenzo D’Ambrosio , Martha Donoghue , Harald Enzmann , Gerry Feeney , Paolo Foggi , Anna Maria Frezza , Ralf Herold , Robin L. Jones , Bernd Kasper , Kit Roes , Pierre Demolis
Ultra-rare sarcomas (URS) and ultra-rare cancers (URC) represent a unique challenge in oncology due to their rarity, heterogeneity, and the severe unmet clinical needs of affected patients. In 2024, the European Medicines Agency (EMA) and the European Organisation for Research and Treatment of Cancer (EORTC) convened two multi-stakeholder workshops, bringing together regulators, clinicians, researchers, and patient advocates. These workshops aimed to explore innovative strategies for treatment development and establish a framework for future collaboration.
Key issues were discussed, including the scarcity of biological and clinical data, major barriers in conducting randomized trials, and limited pharmaceutical investment. A key outcome was the unanimous commitment of all stakeholders, including regulatory agencies such as EMA and the U.S. FDA, to work together towards pragmatic solutions. Participants recognized the necessity of flexible regulatory approaches, alternative trial designs, and meaningful endpoints tailored to ultra-rare conditions. The workshops also highlighted the importance of global collaboration, early regulatory engagement, and leveraging existing mechanisms like orphan drug designation and conditional approvals.
The discussions emphasized that while scientific rigor must be upheld, regulatory frameworks must adapt to the specific challenges posed by URS. Stakeholders pledged to maintain open dialogue, share expertise, and develop innovative infrastructures to accelerate progress.
This collaborative commitment marks a critical step forward in addressing the high unmet needs of URS. By fostering a unified effort among diverse stakeholders, the workshops established a model for advancing treatments in other URC, prioritizing patient outcomes while navigating the complexities of drug development for these challenging diseases.
超罕见肉瘤(URS)和超罕见癌症(URC)由于其罕见性、异质性和患者严重未满足的临床需求,在肿瘤学中代表了一个独特的挑战。2024年,欧洲药品管理局(EMA)和欧洲癌症研究与治疗组织(EORTC)召集了两次多方利益相关者研讨会,汇集了监管机构、临床医生、研究人员和患者倡导者。这些研讨会旨在探索治疗发展的创新战略,并建立未来合作的框架。讨论了关键问题,包括生物和临床数据的缺乏,进行随机试验的主要障碍,以及有限的制药投资。一个关键的结果是所有利益相关者的一致承诺,包括EMA和美国FDA等监管机构,共同努力寻求务实的解决方案。与会者认识到灵活的监管方法、替代试验设计和针对超罕见疾病量身定制的有意义的终点的必要性。研讨会还强调了全球合作、早期监管参与以及利用孤儿药指定和有条件批准等现有机制的重要性。讨论强调,虽然必须坚持科学严谨性,但监管框架必须适应URS带来的具体挑战。利益攸关方承诺保持公开对话,分享专业知识,开发创新基础设施,以加快进展。这一合作承诺标志着在解决URS未满足的高需求方面向前迈出了关键一步。通过促进不同利益相关者之间的统一努力,研讨会建立了在其他URC中推进治疗的模式,优先考虑患者的结果,同时引导这些具有挑战性的疾病的药物开发的复杂性。
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引用次数: 0
The emerging role of PARP inhibitors in prostate cancer: A narrative review PARP抑制剂在前列腺癌中的新作用:综述
IF 10.5 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-25 DOI: 10.1016/j.ctrv.2025.103000
Lorenzo Lobianco , Gabriele Calvanese , Debora D’Ausilio , Sabrina Rossetti , Sabrina Chiara Cecere , Jole Ventriglia , Carmela Pisano , Rosa Tambaro , Marilena Di Napoli , Anna Passarelli , Cristin Roma , Antonella De Luca , Filippo Sepe , Silvana Cozzolino , Erica Perri , Maria Rosaria Lamia , Carlo Moccia , Sandro Pignata
Prostate cancer (PC) is the second most common cancer and the fifth leading cause of cancer mortality in men, worldwide. Genomic analysis identified alterations in DNA damage repair (DDR) pathways, in up to 30% of metastatic castration-resistant prostate cancer (mCRPC). Homologous recombination repair (HRR) mutations in BRCA1/2 emerged as a relevant biomarker in PC, linked to aggressive behavior, unfavorable outcomes and notable responses to poly-ADP ribose polymerase inhibitors (PARPi). While PARPi efficacy in BRCA-mutated mCRPC is well established, their role in earlier disease stages is currently under investigation. This non-systematic narrative review aims to summarize current evidence on PARPi in PC, outlining approved indications, ongoing clinical trials, and emerging therapeutic strategies across different disease settings.
前列腺癌(PC)是全球第二大常见癌症,也是男性癌症死亡的第五大原因。基因组分析发现,在高达30%的转移性去势抵抗性前列腺癌(mCRPC)中,DNA损伤修复(DDR)途径发生了改变。BRCA1/2的同源重组修复(HRR)突变成为PC的相关生物标志物,与侵袭行为、不良结局和对多adp核糖聚合酶抑制剂(PARPi)的显着反应有关。虽然PARPi对brca突变的mCRPC的疗效已经得到了很好的证实,但它们在早期疾病阶段的作用目前还在研究中。这篇非系统的叙述性综述旨在总结目前PARPi在PC中的证据,概述已批准的适应症、正在进行的临床试验和不同疾病背景下新出现的治疗策略。
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引用次数: 0
The importance of education and training in neuroendocrine neoplasms: challenges and opportunities for multidisciplinary management 神经内分泌肿瘤教育和培训的重要性:多学科管理的挑战和机遇
IF 9.6 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-24 DOI: 10.1016/j.ctrv.2025.102998
Francesco Panzuto , Simona Barbi , Annalisa Trama , Nicola Fazio
Neuroendocrine neoplasms (NENs) exemplify the challenges and opportunities inherent in managing rare cancers. Their rarity, biological heterogeneity, and diagnostic complexity necessitate a highly structured and multidisciplinary approach to patient care. In this context, education and training emerge as central pillars for improving clinical outcomes.
This review highlights how integrating multidisciplinary teams of diverse medical specialties enhances diagnostic accuracy, refines therapeutic strategies, and ensures adherence to evolving best practices. Establishing dedicated training pathways—both through traditional educational models and innovative digital platforms—is crucial to address the unique learning needs posed by NENs.
Scientific societies play a pivotal role by producing guidelines and fostering continuous professional development, although notable variability across international recommendations emphasizes the need for clinicians to harmonize and interpret critically. Patient advocacy groups, meanwhile, have become essential actors in the educational ecosystem, bridging informational gaps and advocating for patient-centered research and policy initiatives.
Emerging technologies, particularly artificial intelligence, offer promising tools to support clinical education and decision-making, provided that their implementation is cautious, validated, and integrated under healthcare professionals’ guidance.
By analyzing the NEN model, this review underscores that the future of rare cancer management relies on building strong collaborative networks, promoting standardized yet flexible educational programs, and embracing technological innovation, always focusing on quality, safety, and patient-centered care.
神经内分泌肿瘤(NENs)体现了管理罕见癌症固有的挑战和机遇。它们的罕见性、生物学异质性和诊断复杂性需要高度结构化和多学科的方法来治疗患者。在这种情况下,教育和培训成为改善临床结果的中心支柱。这篇综述强调了如何整合不同医学专业的多学科团队提高诊断准确性,完善治疗策略,并确保坚持不断发展的最佳实践。通过传统的教育模式和创新的数字平台建立专门的培训途径,对于满足新一代的独特学习需求至关重要。科学学会在制定指南和促进持续的专业发展方面发挥着关键作用,尽管国际建议的显著差异强调了临床医生需要协调和批判性地解释。与此同时,患者权益团体已成为教育生态系统中的重要参与者,弥合信息差距,倡导以患者为中心的研究和政策举措。新兴技术,特别是人工智能,为支持临床教育和决策提供了有前途的工具,前提是它们的实施是谨慎的、经过验证的,并在医疗保健专业人员的指导下进行整合。通过分析NEN模式,本综述强调了罕见癌症管理的未来依赖于建立强大的合作网络,促进标准化但灵活的教育计划,拥抱技术创新,始终关注质量,安全和以患者为中心的护理。
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引用次数: 0
Circulating tumor DNA as prognostic marker in patients with metastatic colorectal cancer undergoing systemic therapy: A systematic review and meta-analysis 循环肿瘤DNA作为转移性结直肠癌患者接受全身治疗的预后标志物:一项系统回顾和荟萃分析
IF 10.5 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-24 DOI: 10.1016/j.ctrv.2025.102999
Anja Holz , Bidisha Paul , Antonia Zapf , Klaus Pantel , Simon A. Joosse

Background

The response to systemic therapy against metastatic colorectal cancer (mCRC) is currently assessed by radiologic imaging. However, an increasing number of studies have shown that circulating tumor DNA (ctDNA) as liquid biopsy can be used as an alternative method to assess therapy efficacy. We conducted a systematic review with subsequent meta-analysis of primary studies to assess the prognostic value of sequential liquid biopsies in patients with metastatic colorectal cancer treated with systemic therapy.

Methods

Randomized, non-randomized, and prospective observational studies, reporting on the change in ctDNA concentration in total cell-free DNA over the course of systemic therapy of patients treated for metastatic colorectal cancer to predict treatment response according to RECIST criteria were included.

Results

Fifty-six studies involving 3735 evaluable patients with metastatic colorectal cancer were included in the meta-analysis. ctDNA increase during systemic therapy as compared to baseline was strongly associated with progression-free survival (HR: 2.44, 95% CI: 2.02–2.95) and overall survival (HR: 2.53, 95% CI: 2.01–3.18), which were reported in 39 and 33 studies, respectively.

Conclusion

Our analyses underscore the strong prognostic value of longitudinal plasma-based ctDNA analysis through liquid biopsy in mCRC patients. Subsequent research should evaluate the role of ctDNA in guiding therapy decisions, particularly in identifying patients likely to benefit from continuation or change of systemic therapy. While further standardization of ctDNA testing remains necessary, current evidence supports integrating serial ctDNA monitoring into upcoming clinical mCRC intervention trials, to lay the groundwork for its inclusion into future RECIST versions.

Protocol registration

The protocol for this review was registered on PROSPERO (CRD42023420012).
背景:目前,对转移性结直肠癌(mCRC)的全身治疗的反应是通过放射影像学来评估的。然而,越来越多的研究表明,循环肿瘤DNA (ctDNA)作为液体活检可以作为评估治疗效果的替代方法。我们进行了一项系统综述,并对初步研究进行了meta分析,以评估连续液体活检对接受全身治疗的转移性结直肠癌患者的预后价值。方法纳入随机、非随机和前瞻性观察性研究,这些研究报告了转移性结直肠癌患者在全身治疗过程中总游离DNA中ctDNA浓度的变化,以根据RECIST标准预测治疗反应。结果共纳入56项研究,涉及3735例可评估的转移性结直肠癌患者。与基线相比,ctDNA在全身治疗期间的增加与无进展生存期(HR: 2.44, 95% CI: 2.02-2.95)和总生存期(HR: 2.53, 95% CI: 2.01-3.18)密切相关,分别在39项和33项研究中报道。结论我们的分析强调了通过液体活检纵向血浆ctDNA分析对mCRC患者的预后有很强的价值。后续研究应评估ctDNA在指导治疗决策中的作用,特别是在识别可能从继续或改变全身治疗中获益的患者时。虽然ctDNA检测的进一步标准化仍然是必要的,但目前的证据支持将连续ctDNA监测整合到即将到来的临床mCRC干预试验中,为其纳入未来的RECIST版本奠定基础。方案注册本综述的方案已在PROSPERO上注册(CRD42023420012)。
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引用次数: 0
Management changes and survival outcomes for cancer patients after multidisciplinary team discussion; a systematic review and meta-analysis 多学科小组讨论后癌症患者的管理改变和生存结局系统回顾和荟萃分析
IF 9.6 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-23 DOI: 10.1016/j.ctrv.2025.102997
Gabrielle J. Williams , John F. Thompson

Objective

Multidisciplinary team (MDT) review of cancer patients has become routine in many institutions worldwide. The process seeks to ensure correct diagnosis and staging, optimise treatment decisions and improve outcomes. The aim of this study was to measure any differences in management and survival of cancer patients reviewed at an MDT meeting compared to patients not reviewed or with management plans made prior to MDT review.

Methods and Analysis

A PRISMA-compliant systematic review was undertaken with searches of Medline, Embase and the Cochrane Trials Register to 7 June 2024, with no restrictions on language or era.

Results

From 2832 titles, 179 comparative studies were identified. Hazard ratios (HRs) for death, generated from multivariable analyses, were reported in 37 studies that included 56,187 patients but estimates were highly variable (I2 87 %), ranging from HR 0.1 to 0.96. While the magnitude of the benefit for patients after MDT review was variable, all 37 studies reported a reduced risk of death for MDT-reviewed patients compared to non-reviewed patients. Sub-group analyses based on the patient’s cancer type reduced heterogeneity in patients with breast and hepatocellular carcinomas and suggested a reduced risk of death in MDT-reviewed patients compared to those not reviewed (HR 0.86, 95 %CI 0.79–0.93, I2 56 %, p = 0.0001 and HR 0.82, 95 %CI 0.76–0.88, I2 36 %, p < 0.00001, respectively).

Conclusion

Extensive evidence shows a survival benefit for cancer patients discussed at an MDT meeting but there is considerable variation in the reported magnitude of that benefit, ranging from a 4% to a 90% reduction in the risk of death.
目的:多学科小组(MDT)对癌症患者的复查已成为世界范围内许多机构的常规工作。该过程旨在确保正确的诊断和分期,优化治疗决策并改善结果。本研究的目的是衡量在MDT会议上接受审查的癌症患者与未接受审查的患者或在MDT审查之前制定了管理计划的患者相比,在管理和生存方面的任何差异。方法和分析采用符合prisma标准的系统评价,检索Medline、Embase和Cochrane Trials Register至2024年6月7日,无语言和时代限制。结果从2832篇文献中筛选出179篇比较研究。由多变量分析得出的死亡风险比(HRs)在37项研究中报告,其中包括56,187例患者,但估计值变化很大(i287%),风险比范围为0.1至0.96。虽然MDT审查后患者获益的幅度是可变的,但所有37项研究都报告了MDT审查的患者与未审查的患者相比死亡风险降低。基于患者癌症类型的亚组分析降低了乳腺癌和肝细胞癌患者的异质性,并表明与未接受mdt审查的患者相比,接受mdt审查的患者的死亡风险降低(HR 0.86, 95% CI 0.79-0.93, I2 56%, p = 0.0001; HR 0.82, 95% CI 0.76-0.88, I2 36%, p <;分别为0.00001)。结论:广泛的证据表明,在MDT会议上讨论了癌症患者的生存获益,但报告的获益幅度差异很大,从4%到90%的死亡风险降低不等。
{"title":"Management changes and survival outcomes for cancer patients after multidisciplinary team discussion; a systematic review and meta-analysis","authors":"Gabrielle J. Williams ,&nbsp;John F. Thompson","doi":"10.1016/j.ctrv.2025.102997","DOIUrl":"10.1016/j.ctrv.2025.102997","url":null,"abstract":"<div><h3>Objective</h3><div>Multidisciplinary team (MDT) review of cancer patients has become routine in many institutions worldwide. The process seeks to ensure correct diagnosis and staging, optimise treatment decisions and improve outcomes. The aim of this study was to measure any differences in management and survival of cancer patients reviewed at an MDT meeting compared to patients not reviewed or with management plans made prior to MDT review.</div></div><div><h3>Methods and Analysis</h3><div>A PRISMA-compliant systematic review was undertaken with searches of Medline, Embase and the Cochrane Trials Register to 7 June 2024, with no restrictions on language or era.</div></div><div><h3>Results</h3><div>From 2832 titles, 179 comparative studies were identified. Hazard ratios (HRs) for death, generated from multivariable analyses, were reported in 37 studies that included 56,187 patients but estimates were highly variable (I<sup>2</sup> 87 %), ranging from HR 0.1 to 0.96. While the magnitude of the benefit for patients after MDT review was variable, all 37 studies reported a reduced risk of death for MDT-reviewed patients compared to non-reviewed patients. Sub-group analyses based on the patient’s cancer type reduced heterogeneity in patients with breast and hepatocellular carcinomas and suggested a reduced risk of death in MDT-reviewed patients compared to those not reviewed (HR 0.86, 95 %CI 0.79–0.93, I<sup>2</sup> 56 %, p = 0.0001 and HR 0.82, 95 %CI 0.76–0.88, I<sup>2</sup> 36 %, p &lt; 0.00001, respectively).</div></div><div><h3>Conclusion</h3><div>Extensive evidence shows a survival benefit for cancer patients discussed at an MDT meeting but there is considerable variation in the reported magnitude of that benefit, ranging from a 4% to a 90% reduction in the risk of death.</div></div>","PeriodicalId":9537,"journal":{"name":"Cancer treatment reviews","volume":"139 ","pages":"Article 102997"},"PeriodicalIF":9.6,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144703597","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Exercise in patients with metastatic prostate cancer: A comprehensive review 运动对转移性前列腺癌患者的影响:一项综合综述
IF 9.6 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-18 DOI: 10.1016/j.ctrv.2025.102996
F. Javier Muñoz-Carrillo , Marta Garcia de Herreros , David Carrillo , Olenka Peralta , Caterina Aversa , Laia Fernández-Mañas , Laura Ferrer-Mileo , Mariana Altamirano , Òscar Reig Torras , Natalia Jimenez , Begoña Mellado
Prostate cancer (PC) is one of the most prevalent malignancies worldwide. Metastatic PC remains an incurable disease, with the androgen receptor (AR) pathway being the primary driver of tumor progression and the main target for therapeutic strategies. Thus, patients usually undergo long-term treatments, particularly androgen deprivation therapy (ADT), which can worsen the intrinsic deterioration in quality of life (QoL) caused by the disease burden. Increasing evidence supports the use of physical activity (PA) and structured exercise (EX) as complementary measures to mitigate treatment-related adverse effects and improve clinical outcomes across tumor types. EX has shown benefits across multiple systems and plays a significant role in modulating tumor progression through several cellular pathways. Furthermore, it has confirmed potential to alleviate cancer-related symptoms while enhancing functional capacity and tolerability of treatment. This review gathers the current evidence regarding the impact of PA and EX on patients with metastatic PC, integrating both epidemiological and interventional studies. Despite promising findings, most of the available evidence is documented on non-metastatic populations, highlighting the need for directed studies in advanced disease settings. Future research is needed in metastatic PC patients, in order to assess long-term impacts of EX in this population.
前列腺癌是世界上最常见的恶性肿瘤之一。转移性前列腺癌仍然是一种无法治愈的疾病,雄激素受体(AR)途径是肿瘤进展的主要驱动因素,也是治疗策略的主要目标。因此,患者通常接受长期治疗,特别是雄激素剥夺治疗(ADT),这可能加剧疾病负担引起的生活质量(QoL)的内在恶化。越来越多的证据支持使用身体活动(PA)和结构化运动(EX)作为辅助措施,以减轻治疗相关的不良反应并改善各种肿瘤类型的临床结果。EX在多个系统中显示出益处,并通过几种细胞途径在调节肿瘤进展中发挥重要作用。此外,它已证实有可能减轻癌症相关症状,同时增强功能能力和治疗耐受性。本综述收集了目前关于PA和EX对转移性PC患者影响的证据,整合了流行病学和介入性研究。尽管有令人鼓舞的发现,但大多数现有证据都是在非转移性人群中记录的,这突出了在晚期疾病环境中进行定向研究的必要性。为了评估EX对这一人群的长期影响,需要对转移性PC患者进行进一步的研究。
{"title":"Exercise in patients with metastatic prostate cancer: A comprehensive review","authors":"F. Javier Muñoz-Carrillo ,&nbsp;Marta Garcia de Herreros ,&nbsp;David Carrillo ,&nbsp;Olenka Peralta ,&nbsp;Caterina Aversa ,&nbsp;Laia Fernández-Mañas ,&nbsp;Laura Ferrer-Mileo ,&nbsp;Mariana Altamirano ,&nbsp;Òscar Reig Torras ,&nbsp;Natalia Jimenez ,&nbsp;Begoña Mellado","doi":"10.1016/j.ctrv.2025.102996","DOIUrl":"10.1016/j.ctrv.2025.102996","url":null,"abstract":"<div><div>Prostate cancer (PC) is one of the most prevalent malignancies worldwide. Metastatic PC remains an incurable disease, with the androgen receptor (AR) pathway being the primary driver of tumor progression and the main target for therapeutic strategies. Thus, patients usually undergo long-term treatments, particularly androgen deprivation therapy (ADT), which can worsen the intrinsic deterioration in quality of life (QoL) caused by the disease burden. Increasing evidence supports the use of physical activity (PA) and structured exercise (EX) as complementary measures to mitigate treatment-related adverse effects and improve clinical outcomes across tumor types. EX has shown benefits across multiple systems and plays a significant role in modulating tumor progression through several cellular pathways. Furthermore, it has confirmed potential to alleviate cancer-related symptoms while enhancing functional capacity and tolerability of treatment. This review gathers the current evidence regarding the impact of PA and EX on patients with metastatic PC, integrating both epidemiological and interventional studies. Despite promising findings, most of the available evidence is documented on non-metastatic populations, highlighting the need for directed studies in advanced disease settings. Future research is needed in metastatic PC patients, in order to assess long-term impacts of EX in this population.</div></div>","PeriodicalId":9537,"journal":{"name":"Cancer treatment reviews","volume":"139 ","pages":"Article 102996"},"PeriodicalIF":9.6,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144694744","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
De-escalation strategies with targeted therapies in non-small cell lung cancer 非小细胞肺癌靶向治疗的降级策略
IF 9.6 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-17 DOI: 10.1016/j.ctrv.2025.102995
Martina Bortolot , Jordi Remon , Paolo Bironzo , Francesco Cortiula , Jessica Menis , Sze Wai Chan , Robin van Geel , Noemi Reguart , Oscar Arrieta , Giannis Mountzios , Anne-Marie C. Dingemans , Benjamin Besse , Lizza E.L. Hendriks
Targeted therapies (TT) for non-small cell lung cancer (NSCLC) with actionable genomic alterations (AGA), particularly EGFR-mutant and ALK-rearranged tumors, have become the standard of care across nearly all stages of the disease. However, the arbitrarily defined dose and treatment duration of TT, as well as the financial cost of these drugs, reduce their availability worldwide. Pharmacokinetic and pharmacodynamic properties of TT suggest that doses of some TT are overestimated as there is limited evidence supporting a direct relationship between therapeutic intensity and outcomes. This can lead to overtreatment of patients, resulting in an increased risk of toxicity without enhanced efficacy. Some academic initiatives have been launched aiming to explore de-escalating strategies with TT, either reducing the dose or the duration of these drugs. These approaches can decrease the risk of adverse events positively impacting patients’ quality of life, without compromising efficacy, while reducing economic impact. In this review, we summarize current data regarding de-escalating strategies with TT, ongoing trials and challenges of this approach.
针对具有可操作基因组改变(AGA)的非小细胞肺癌(NSCLC),特别是egfr突变和alk重排肿瘤的靶向治疗(TT)已成为几乎所有疾病阶段的标准治疗方法。然而,TT的剂量和治疗时间的任意定义,以及这些药物的财务成本,减少了它们在世界范围内的可用性。TT的药代动力学和药效学特性表明,由于有限的证据支持治疗强度与结果之间的直接关系,某些TT的剂量被高估了。这可能导致对患者的过度治疗,导致毒性风险增加,而疗效却没有提高。一些学术倡议已经启动,旨在探索TT的降级策略,要么减少这些药物的剂量,要么减少这些药物的持续时间。这些方法可以降低不良事件的风险,对患者的生活质量产生积极影响,而不影响疗效,同时减少经济影响。在这篇综述中,我们总结了目前关于TT降压策略的数据,正在进行的试验和该方法的挑战。
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引用次数: 0
Corrigendum to “Trastuzumab deruxtecan in human epidermal growth factor receptor 2-positive breast cancer brain metastases: A systematic review and updated meta-analysis” [Cancer Treat. Rev. 133 (2025) 102882] 《曲妥珠单抗德鲁西替康治疗人表皮生长因子受体2阳性乳腺癌脑转移:系统回顾和最新荟萃分析》的更正。Rev. 133(2025) 102882]。
IF 10.5 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-15 DOI: 10.1016/j.ctrv.2025.102994
Isabella Michelon , Caio E.R. Castro , Thiago Madeira , Maria Inez Dacoregio , Carlos Stecca , Leonardo R. Soares , Anwaar Saeed , Maysa Vilbert , Ludimila Cavalcante
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引用次数: 0
Palliative systemic therapy for locally advanced or metastatic salivary duct carcinoma: A comprehensive review 局部晚期或转移性唾液管癌的姑息性全身治疗:全面回顾
IF 9.6 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-11 DOI: 10.1016/j.ctrv.2025.102993
Masafumi Kanno , Satoshi Kano , Yoshinori Imamura , Daisuke Kawakita , Norihiko Narita , Yuichiro Tada , Yumi Ito , Taiyo Morikawa , Yoshimasa Imoto , Yukinori Kato , Tetsuji Takabayashi , Shigeharu Fujieda
Salivary duct carcinoma (SDC) is a rare and highly aggressive malignancy of the salivary glands. For patients ineligible for curative surgery or definitive radiotherapy, treatment options remain limited owing to the absence of standardized therapeutic protocols. This review draws upon major salivary gland cancer guidelines and integrates molecular profiles with clinical response data to propose an evidence-based treatment algorithm that stratifies patients into four groups according to the expression status of human epidermal growth factor receptor 2 (HER2) and androgen receptor (AR).
In HER2-positive tumors, HER2-targeted therapy constitutes the standard of care, with trastuzumab plus docetaxel providing clinical benefit. Antibody-drug conjugates, such as trastuzumab deruxtecan, have demonstrated efficacy even in patients with disease progression during trastuzumab-based therapy. In AR-positive tumors, androgen deprivation therapy, particularly combined androgen blockade, has demonstrated clinical activity, though concurrent molecular characteristics may influence treatment outcomes.
In HER2-positive/AR-positive tumors, HER2-targeted therapy is generally prioritized, although the absence of validated predictive biomarkers and defined thresholds remains a clinical challenge. For HER2-negative and AR-negative tumors, or those refractory to either or both targeted approaches, cytotoxic chemotherapy remains a viable option. Immune checkpoint inhibitors may offer benefits in tumors with high PD-L1 expression, although data on their role in SDC remain limited. Next-generation sequencing is recommended to identify actionable alterations (e.g., NTRK, BRAF, FGFR, HRAS) that may guide targeted therapy or clinical trial enrollment. Integrating comprehensive molecular profiling into treatment decision-making is essential to optimizing outcomes in advanced SDC.
摘要唾液腺导管癌是一种罕见且高度侵袭性的恶性肿瘤。由于缺乏标准化的治疗方案,对于不符合治疗性手术或明确放疗条件的患者,治疗选择仍然有限。本综述借鉴了主要的唾液腺癌指南,结合分子图谱和临床反应数据,提出了一种基于证据的治疗算法,该算法根据人表皮生长因子受体2 (HER2)和雄激素受体(AR)的表达状态将患者分为四组。在her2阳性肿瘤中,her2靶向治疗构成了标准治疗,曲妥珠单抗加多西他赛提供了临床益处。抗体-药物结合物,如曲妥珠单抗德鲁西替康,即使在曲妥珠单抗治疗期间疾病进展的患者中也已证明有效。在ar阳性肿瘤中,雄激素剥夺疗法,特别是联合雄激素阻断疗法,已显示出临床活性,尽管同时存在的分子特征可能会影响治疗结果。在her2阳性/ ar阳性肿瘤中,通常优先考虑her2靶向治疗,尽管缺乏经过验证的预测性生物标志物和定义的阈值仍然是临床挑战。对于her2阴性和ar阴性肿瘤,或对其中一种或两种靶向方法都难治的肿瘤,细胞毒性化疗仍然是一种可行的选择。免疫检查点抑制剂可能在PD-L1高表达的肿瘤中提供益处,尽管关于其在SDC中的作用的数据仍然有限。新一代测序被推荐用于识别可操作的改变(例如,NTRK、BRAF、FGFR、HRAS),这些改变可能指导靶向治疗或临床试验的入组。将全面的分子谱分析整合到治疗决策中对于优化晚期SDC的预后至关重要。
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引用次数: 0
Long-term surveillance recommendations for young adult cancer survivors 对年轻成年癌症幸存者的长期监测建议
IF 9.6 1区 医学 Q1 ONCOLOGY Pub Date : 2025-07-04 DOI: 10.1016/j.ctrv.2025.102992
Katharina Egger-Heidrich , Franziska Wolters , Mareike Frick , Teresa Halbsguth , Theresa Müller , Hannah Woopen , Kristin Tausche , Diana Richter , Judith Gebauer

Background

Advancements in cancer treatment have led to increased survival rates among young adult cancer survivors (YACS). However, these individuals face unique long-term health risks, including secondary malignancies, cardiovascular disease, and psychosocial challenges. Effective long-term surveillance strategies are critical to mitigating these risks and improving health outcomes. This scoping review aims to summarize existing recommendations for long-term surveillance of YACS, identify gaps in current guidelines, and highlight areas for future research.

Methods

A scoping review was conducted using Pubmed focusing on peer-reviewed literature published between January 2015 and January 2025 that addresses post-treatment (>5 years after diagnosis) follow-up strategies for YACS. The review synthesizes recommendations across various cancer types, treatment modalities, and long-term effects.

Results

The review identified 32 recommendations. Of all eligible articles initially retrieved, 169 different articles were included after screening and eligibility. Findings indicate a lack of standardized, age-specific surveillance guidelines, with most recommendations adapted from pediatric or adult oncology frameworks. Emerging evidence suggests that risk-based, personalized surveillance approaches—incorporating genetic predisposition, treatment history, and lifestyle factors—may optimize long-term health outcomes.

Discussion

This review underscores the need for age-appropriate, evidence-based surveillance guidelines tailored to YACS and highlights the importance of multidisciplinary care models to support survivorship. Future research should focus on developing standardized, risk-stratified surveillance protocols and evaluating their impact on health outcomes.
癌症治疗的进步导致年轻成年癌症幸存者(YACS)的生存率增加。然而,这些个体面临独特的长期健康风险,包括继发性恶性肿瘤、心血管疾病和社会心理挑战。有效的长期监测战略对于减轻这些风险和改善健康结果至关重要。本次范围审查的目的是总结现有的长期监测YACS的建议,确定当前指南中的差距,并强调未来研究的领域。方法使用Pubmed对2015年1月至2025年1月间发表的同行评议文献进行范围综述,这些文献涉及YACS治疗后(诊断后5年)随访策略。该综述综合了各种癌症类型、治疗方式和长期影响的建议。结果本综述确定了32项建议。在最初检索到的所有符合条件的文章中,经过筛选和合格后纳入了169篇不同的文章。研究结果表明,缺乏标准化的、针对特定年龄的监测指南,大多数建议改编自儿科或成人肿瘤框架。新出现的证据表明,基于风险的个性化监测方法——结合遗传易感性、治疗史和生活方式因素——可能优化长期健康结果。本综述强调了为YACS量身定制适合年龄的循证监测指南的必要性,并强调了多学科护理模式对支持生存的重要性。未来的研究应侧重于制定标准化、风险分层的监测方案,并评估其对健康结果的影响。
{"title":"Long-term surveillance recommendations for young adult cancer survivors","authors":"Katharina Egger-Heidrich ,&nbsp;Franziska Wolters ,&nbsp;Mareike Frick ,&nbsp;Teresa Halbsguth ,&nbsp;Theresa Müller ,&nbsp;Hannah Woopen ,&nbsp;Kristin Tausche ,&nbsp;Diana Richter ,&nbsp;Judith Gebauer","doi":"10.1016/j.ctrv.2025.102992","DOIUrl":"10.1016/j.ctrv.2025.102992","url":null,"abstract":"<div><h3>Background</h3><div>Advancements in cancer treatment have led to increased survival rates among young adult cancer survivors (YACS). However, these individuals face unique long-term health risks, including secondary malignancies, cardiovascular disease, and psychosocial challenges. Effective long-term surveillance strategies are critical to mitigating these risks and improving health outcomes. This scoping review aims to summarize existing recommendations for long-term surveillance of YACS, identify gaps in current guidelines, and highlight areas for future research.</div></div><div><h3>Methods</h3><div>A scoping review was conducted using Pubmed focusing on peer-reviewed literature published between January 2015 and January 2025 that addresses post-treatment (&gt;5 years after diagnosis) follow-up strategies for YACS. The review synthesizes recommendations across various cancer types, treatment modalities, and long-term effects.</div></div><div><h3>Results</h3><div>The review identified 32 recommendations. Of all eligible articles initially retrieved, 169 different articles were included after screening and eligibility. Findings indicate a lack of standardized, age-specific surveillance guidelines, with most recommendations adapted from pediatric or adult oncology frameworks. Emerging evidence suggests that risk-based, personalized surveillance approaches—incorporating genetic predisposition, treatment history, and lifestyle factors—may optimize long-term health outcomes.</div></div><div><h3>Discussion</h3><div>This review underscores the need for age-appropriate, evidence-based surveillance guidelines tailored to YACS and highlights the importance of multidisciplinary care models to support survivorship. Future research should focus on developing standardized, risk-stratified surveillance protocols and evaluating their impact on health outcomes.</div></div>","PeriodicalId":9537,"journal":{"name":"Cancer treatment reviews","volume":"139 ","pages":"Article 102992"},"PeriodicalIF":9.6,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144606174","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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Cancer treatment reviews
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