BMC Cancer最新文献

{"title":"Synergistic potential of CDH3 in targeting CRC metastasis and enhancing immunotherapy.","authors":"Chen Fu, Jia Fu, Chaoyue Liu, Zhaojin Yu","doi":"10.1186/s12885-025-13845-2","DOIUrl":"https://doi.org/10.1186/s12885-025-13845-2","url":null,"abstract":"<p><strong>Background: </strong>Colorectal cancer (CRC) remains a leading cause of cancer-related mortality, particularly due to advanced-stage metastasis. P-cadherin (CDH3), a potential therapeutic target, is highly expressed in CRC tissues and associated with poor prognosis and metastasis. However, the mechanisms underlying its role in CRC progression and its translational potential remain poorly understood.</p><p><strong>Materials and methods: </strong>This study integrated multiple public databases (TCGA, HCMDB, UALCAN, HPA, UniProt, cBioPortal, and GEO) to evaluate CDH3 expression, construct a prognostic model, and perform functional and translational analyses. Immunohistochemistry was used to validate CDH3 protein expression in clinical samples. Additional analyses included correlations with clinicopathological parameters, immune infiltration (TIDE, TISIDB), functional enrichment (KEGG, GSEA), drug sensitivity (GSCA), and molecular docking (MOE). Single-cell sequencing (CancerSEA, HPA) was also conducted to explore CDH3's role at the single-cell level.</p><p><strong>Results: </strong>CDH3 expression was significantly elevated in CRC tissues and correlated with poor prognosis, recurrence, and metastasis. CDH3 expression was associated with the infiltration of resting immune cells, particularly dendritic cells, and enrichment analysis revealed its critical role in CRC metastasis through extracellular matrix (ECM) and local adhesion pathways. Notably, afatinib emerged as a promising candidate for targeting CDH3 via \"drug repositioning,\" a process involving the repurposing of existing drugs for new therapeutic applications.</p><p><strong>Conclusion: </strong>This study provides novel insights into CDH3's role in CRC metastasis and its potential as a therapeutic target. The translational potential of CDH3, including its integration with immunotherapy and drug repositioning strategies, offers a promising avenue for the treatment of metastatic CRC.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"560"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951682/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742315","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identification of testicular cancer with T2-weighted MRI-based radiomics and automatic machine learning.","authors":"Liang Wang, PeiPei Zhang, Yanhui Feng, Wenzhi Lv, Xiangde Min, Zhiyong Liu, Jin Li, Zhaoyan Feng","doi":"10.1186/s12885-025-13844-3","DOIUrl":"https://doi.org/10.1186/s12885-025-13844-3","url":null,"abstract":"<p><strong>Background: </strong>Distinguishing between benign and malignant testicular lesions on clinical magnetic resonance imaging (MRI) is crucial for guiding treatment planning. However, conventional MRI-based radiomics to identify testicular cancer requires expert machine learning knowledge. This study aims to investigate the potential of utilizing automatic machine learning (AutoML) based on MRI to diagnose testicular lesions without the need for expert algorithm optimization.</p><p><strong>Methods: </strong>Retrospective preoperative MRI scans from 115 patients diagnosed with testicular disease through pathology were obtained. A total of 1781 radiomics features were extracted from each lesion on the T2-weighted images. Intraclass and interclass correlation coefficients were used to evaluate the intra-observer and interobserver agreements for each radiomics feature. We developed an AutoML method based on the tree-based pipeline optimization tool (TPOT) algorithm to construct a discriminant model. The best pipeline was determined through 100 repeated operations using a 5-fold cross-validation algorithm in TPOT. The model was evaluated for accuracy, sensitivity, and specificity using the area under the curve (AUC) value of the receiver operating characteristic (ROC) curve. Shapley Additive exPlanations were used to illustrate the optimization results.</p><p><strong>Results: </strong>Utilizing the TPOT method, 100 diagnostic models were developed to identify testicular lesions. The best model was determined based on the highest AUC in the training cohort. The prediction model yielded AUC values of 0.989 (95% confidence interval [CI]: 0.985-0.993) and 0.909 (95% CI: 0.893-0.923) in the training and testing cohorts, respectively.</p><p><strong>Conclusions: </strong>AutoML, based on the TPOT algorithm, holds potential as a noninvasive method for effectively discriminating between benign and malignant testicular lesions.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"563"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951623/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Heterogeneity of T cells regulates tumor immunity mediated by Helicobacter pylori infection in gastric cancer.","authors":"Zhisheng Wu, Xinya Wang, Shujing Shi, Deyuan Kong, Chuanli Ren, Lijun Bian, Yuanliang Gu, Fangmei An, Qiang Zhan, Caiwang Yan, Chupeng Hu, Yun Chen, Runqiu Jiang, Jinfei Chen","doi":"10.1186/s12885-025-13957-9","DOIUrl":"https://doi.org/10.1186/s12885-025-13957-9","url":null,"abstract":"<p><p>The impact of Helicobacter pylori (H. pylori) status on gastric cancer survival remains unclear. In this study, we conducted a prognostic analysis of 488 gastric cancer patients and performed single-cell RNA sequencing (scRNA-seq) on 18,717 T cells from six tumor samples with varying H. pylori statuses. Our findings revealed that gastric cancer patients with H. pylori infection had significantly longer survival times compared to those with negative H. pylori status. After unsupervised re-clustering of T cells based on scRNA-seq data, we identified ten CD4⁺ and twelve CD8⁺ clusters. Among them, four CD8⁺ T cell clusters exhibited distinct distributions based on H. pylori infection status. One cluster, marked by CXCL13, showed high levels of IFNG and GZMB in H. pylori-infected patients, while another cluster, which expressed immune suppression related genes like AREG and PTGER2, was predominantly comprised of cells from non-infected patients. High PTGER2 expression was significantly associated with worse prognosis in patients with high CD8 expression. These insights advance our understanding of H. pylori's influence on T cell responses in gastric cancer, aiding in treatment and prognostic strategies.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"567"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954285/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742294","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identification and validation of molecular subtypes and prognostic models in patients with kidney cancer based on differential genes based on B cells: a multiomics analysis.","authors":"Jiaao Sun, Shiyan Song, Qiancheng Ma, Feng Chen, Xiaochi Chen, Guangzhen Wu","doi":"10.1186/s12885-025-13923-5","DOIUrl":"https://doi.org/10.1186/s12885-025-13923-5","url":null,"abstract":"<p><strong>Background: </strong>B cells play a variety of complex roles in cancer, both promoting cancer progression and enhancing anti-tumor immune responses, but their mechanism of action in kidney cancer has not been elucidated.</p><p><strong>Results: </strong>We collected kidney cancer sample data from the GEO database and TCGA database, mapped the single-cell landscape inside kidney cancer tissue, identified 25 B-cell-related genes, and based on this, identified related molecular subtypes of kidney cancer patients, and explored their internal microenvironment characteristics. Finally, we constructed a 6-gene biological prognostic model that can be used to predict survival in patients with renal cancer, and we further validated the predictive performance of the model based on imaging omics. It is worth mentioning that the structural patterns and functional sites of 6 model gene transcription proteins were also mined.</p><p><strong>Conclusions: </strong>Overall, we explored for the first time the profound role of B cells in kidney cancer and developed a bio-predictive model based on B cell-related genes, providing scientific guidance for personalized treatment of kidney cancer patients.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"559"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951520/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742297","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A single centre experience of patients with rare cancers referred for early phase clinical trials.","authors":"Angelos Angelakas, Natalie Cook, Donna M Graham, Matthew Krebs, Fiona Thistlethwaite, Louise Carter","doi":"10.1186/s12885-025-13934-2","DOIUrl":"10.1186/s12885-025-13934-2","url":null,"abstract":"<p><strong>Background: </strong>Cancers affecting < 6/100,000/year are classified as rare, but they account for up to 25% of all cancers and are associated with worse 5-year survival than common cancers. Early-phase clinical trials (EPCTs) may represent a viable treatment option for patients with rare cancers as they have evolved significantly with novel designs and the increasing use of precision medicine.</p><p><strong>Methods: </strong>A retrospective study of patients with rare cancers referred to a large EPCT team at a UK specialist centre over 5 years (2016-2020) was conducted. Patient demographics, medical and oncological history, genomic variants, EPCT participation, responses and survival outcomes were analysed.</p><p><strong>Results: </strong>In total, 240 patients with rare cancers were included. The mean age at diagnosis was 51.7 years (range 16-84), 54.2% of the patients were female. The most frequent rare cancers originated from the digestive system (27.1%), female genital tract (20%) and head and neck (H + N) (18.3%). Molecular profiling was offered to 45.5% of the population, median number of gene alterations was 3 per patient (range 1-20) while actionable gene alterations were reported in 60.2% (n = 50) of those with identified gene aberrations. Fifty-one patients participated in EPCTs, with 39.2% achieving SD and 11.8% PR. Median PFS for trial participants was three months (95% CI 1.12 - 4.88) while median OS in the trial patients was 16 months (95% CI 9.10 - 22.90) compared to 7 months for non-trial participants (95% CI 5.50 - 8.51). Finally, poor Royal Marsden Hospital (RMH) prognostic score (2-3) was correlated with worse survival when controlling for age and sex (HR 1.714, 95% CI 1.19 - 2.46, p = 0.004).</p><p><strong>Conclusions: </strong>Participation of patients with rare cancers in EPCTs may be associated with a survival benefit and lead to the development of new treatments for these patients. Moreover, expanded use of precision medicine is paramount as it can inform targeted treatment selection in this heterogenous group.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"558"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951660/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143728605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association of HLA-A*02:01 type with efficacy and toxicity of immune checkpoint inhibitor therapy in melanoma patients: a retrospective cohort study.","authors":"Isabel Manger, Christina Schmitt, Carola Berking, Lars E French, Julio Vera-Gonzalez, Lucie Heinzerling","doi":"10.1186/s12885-025-13857-y","DOIUrl":"https://doi.org/10.1186/s12885-025-13857-y","url":null,"abstract":"<p><strong>Background: </strong>Immune checkpoint inhibitors (ICI) are highly effective but may induce severe or even fatal and unpredictable immune-related adverse events (irAEs). It is unclear whether human leukocyte antigen (HLA) genes contribute to the susceptibility of developing irAEs during ICI therapy.</p><p><strong>Methods: </strong>This multicentre retrospective study investigated the association of irAE and outcome with HLA-A*02:01 status in a cohort of 97 patients with metastatic melanoma undergoing ICI therapy. Organ-specific irAEs and therapy outcome as assessed by response rate, progression-free survival (PFS) and overall survival (OS) were analysed depending on HLA type HLA-A*02:01. For the outcome only patients with cutaneous melanoma were analysed. Chi square test, exact fisher test, Kruskal Wallis test and log rank test were employed for statistical analysis (p ≤ 0.05).</p><p><strong>Results: </strong>The cohort included 38 HLA-A*02:01 positive (39.2%) and 59 HLA-A*02:01 negative (60.8%) patients. Data showed no evidence of an association of HLA-A*02:01 with organ-specific irAEs except for a numerical difference in immune-related colitis. Furthermore, response rates of the subgroup of patients with metastatic cutaneous melanoma did not differ between the two cohorts. The median PFS was 5 months and 8 months in HLA-A*02:01 positive and negative patients with cutaneous melanoma, respectively.</p><p><strong>Conclusion: </strong>HLA-A*02:01 was not associated with specific checkpoint inhibitor-induced organ toxicity in this cohort of HLA-A-typed melanoma patients. Interestingly, in the relatively small subgroup of patients with cutaneous melanoma an earlier progression in HLA-A*02:01 positive patients was observed, however not in the long term. These findings are exploratory due to the limited sample size and require validation in larger, prospective cohorts.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"565"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954185/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742290","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"PD-1 inhibitors improve the efficacy of transcatheter arterial chemoembolization combined with apatinib in advanced hepatocellular carcinoma: a meta-analysis and trial sequential analysis.","authors":"Jiahui Yu, Jinxin Yu, Yimiao Chen, Yuting Yang, Pengsheng Yi","doi":"10.1186/s12885-025-13932-4","DOIUrl":"https://doi.org/10.1186/s12885-025-13932-4","url":null,"abstract":"<p><strong>Background: </strong>The efficacy of adding programmed death-1 (PD-1) inhibitors to transcatheter arterial chemoembolization (TACE) combined with apatinib for advanced hepatocellular carcinoma (HCC) remains controversial. This study aimed to evaluate the efficacy of incorporating PD-1 inhibitors into TACE combined with apatinib.</p><p><strong>Methods: </strong>Relevant literature on TACE combined with apatinib plus PD-1 inhibitors for advanced HCC was searched in PubMed, Cochrane Library, Embase, and Web of Science databases. Trial sequential analysis (TSA) was conducted to minimize randomization errors and assess whether the meta-analysis provided conclusive evidence.</p><p><strong>Results: </strong>Six studies involving 1,452 patients were included. Compared with the TACE combined with apatinib treatment group (T-A), TACE combined with apatinib plus PD-1 inhibitors (T-A-P) significantly prolonged overall survival (OS) (Hazard Ratio [HR] 2.22, 95% Confidence Interval [CI] 1.93-2.56; p < 0.001) and progression-free survival (PFS) (HR 2.36, 95% CI 2.01-2.77; p < 0.001), while also improving the objective response rate (ORR) (risk ratios [RR] 1.60, 95% CI 1.20-2.14; p < 0.001) and disease control rate (DCR) (RR 1.06, 95% CI 1.00-1.12; p < 0.001). TSA results indicated that additional studies were required to confirm the significance of DCR. Prognostic analysis identified treatment regimen and extrahepatic metastasis as common independent risk factors for OS and PFS. The incidence of adverse events in the T-A-P treatment group was comparable to that in the T-A treatment group.</p><p><strong>Conclusion: </strong>Adding PD-1 inhibitors to TACE combined with apatinib significantly prolonged OS and PFS, particularly in patients without extrahepatic metastases. It also improved ORR and DCR in patients with HCC.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"564"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951536/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742307","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-term outcomes and prognostic factors of eye-preserving treatment with particle beam radiotherapy for orbital malignancies.","authors":"Weixu Hu, Qiong Cai, Jing Gao, Jiyi Hu, Qingting Huang, Haojiong Zhang, Lin Kong","doi":"10.1186/s12885-025-13986-4","DOIUrl":"https://doi.org/10.1186/s12885-025-13986-4","url":null,"abstract":"<p><strong>Background: </strong>This retrospective study report the clinical experience of eye-preserving treatment follow by particle beam radiotherapy (IMPT or CIRT) for orbital malignancies. And to evaluate prognostic factors for orbital and lacrimal gland tumors.</p><p><strong>Methods: </strong>Sixty-two patients with orbital malignancies were identified in the records of a single center between 2015 and 2021. Sixty-one patients met inclusion criteria. All of the patients received eye-preserving treatment before PBRT. Majority of the patients (91.8%) were treatment with CIRT. Clinical data, treatment modality, local control, metastases and survivals and visual outcomes, as well as associated prognostic indicators were were assessed.</p><p><strong>Results: </strong>Sixty-one patients were followed with a median of 40.7 months (44.3 months for surviving patients). The 3- and 5-year DSS and LC rates were 88.1% and 69.9%, and the 3- and 5-year DMC rates were 77.5% and 74.2% for entire orbital malignancies. For lacrimal gland carcinoma, the 5-year DSS, LC, DMC, and PFS rates were 83.3%, 64.8%, 66.8%, and 53.4%. Tumor size, T stage, extraorbital invasion, and bone invasion influenced survivals. No grade 3 or higher acute toxicities were observed. A total of 8 patients experienced grade 3-4 visual impairment.</p><p><strong>Conclusions: </strong>Particle radiotherapy following eye-preserving treatment provided a favorable local control and survivals with moderate acute and late toxicities, even in patients with unresectable disease. Particle radiotherapy was a promising strategy for management of orbital tumors.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"569"},"PeriodicalIF":3.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954175/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742304","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analysis of extracellular vesicles of frequently used colorectal cancer cell lines.","authors":"Marie Boudná, Nicolas Blavet, Tetiana Samoilenko, Táňa Macháčková, Robin Jugas, Petra Vychytilová-Faltejsková, Miroslav Boudný, Renata Bartošová, Jan Kotouček, Vojtěch Bystrý, Kateřina Koželková, Ondřej Slabý, Kamila Součková","doi":"10.1186/s12885-025-13936-0","DOIUrl":"10.1186/s12885-025-13936-0","url":null,"abstract":"<p><p>Colorectal cancer (CRC) ranks as the second most prevalent malignancy globally, highlighting the urgent need for more effective diagnostic and therapeutic strategies, as well as a deeper understanding of its molecular basis. Extensive research has demonstrated that cells actively secrete extracellular vesicles (EVs) to mediate intercellular communication at both proximal and distal sites. In this study, we conducted a comprehensive analysis of the RNA content of small extracellular vesicles (sEVs) secreted into the culture media of five frequently utilised CRC cell lines (RKO, HCT116, HCT15, HT29, and DLD1). RNA sequencing data revealed significant insights into the RNA profiles of these sEVs, identifying nine protein-coding genes and fourteen long non-coding RNA (lncRNA) genes that consistently ranked among the top 30 most abundant across all cell lines. Notably, the genes found in sEVs were highly similar among the cell lines, indicating a conserved molecular signature. Several of these genes have been previously documented in the context of cancer biology, while others represent novel discoveries. These findings provide valuable insights into the molecular cargo of sEVs in CRC, potentially unveiling novel biomarkers and therapeutic targets.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"555"},"PeriodicalIF":3.4,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951637/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143728606","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness of bilateral tongue base mucosectomy by transoral robotic surgery or transoral laser microsurgery in combination with tonsillectomy in identifying head and neck primary cancer of unknown primary: a randomized phase 2 protocol (RoboCUP trial).","authors":"Vianney Bastit, Justine Lequesne, Alexandra Leconte, Sophie Deneuve, Francois Mouawad, Elske Quak, Corinne Jeanne, Bénédicte Clarisse, Juliette Thariat","doi":"10.1186/s12885-025-13913-7","DOIUrl":"10.1186/s12885-025-13913-7","url":null,"abstract":"<p><strong>Background: </strong>In cases of prevalent lymphadenopathy in the head and neck cancer region, identifying the primary tumor site allows for more precise radiotherapy targeting. This improves treatment by reducing the volumes of mucosal irradiation and potentially lowering morbidity. An extensive diagnostic workup, including FDG PET-CT imaging and bilateral tonsillectomy, has been shown to identify the primary cancer in 60% of cases. Mucosectomy of the tongue base holds promise for detecting additional primary sites. When performed using minimally invasive endoscopic techniques such as transoral robotic surgery (TORS) or transoral laser microsurgery (TLM), mucosectomy results in minimal morbidity. However, the effectiveness of tongue base mucosectomy in detecting primary tumors has yet to be evaluated in a randomized trial involving patients with lymphadenopathy of unknown primary.</p><p><strong>Methods: </strong>The RoboCUP trial is a multicentre, open-label, randomized, non-comparative phase 2 trial aiming to evaluate the benefit of bilateral TORS or TLM-assisted tongue base mucosectomy in association to tonsillectomy in the assessment of prevalent cervical lymphadenopathy with a negative exhaustive diagnostic workup. The main endpoint is the proportion of patients with detection of a primary cancer. Surgery will consist in tongue base mucosectomy plus tonsillectomy in the experimental arm, and the standard of care, i.e. tonsillectomy alone in the control arm. Patients will then be treated by intensity modulated radiotherapy, possibly with chemotherapy as radiosensitizer, per current guidelines. Using a single-stage Fleming design, 36 patients will be enrolled in the experimental arm, and 36 patients in the control arm.</p><p><strong>Discussion: </strong>This trial aims to improve the diagnostic performances, i.e. detection of primary tumor, in patients with head and neck carcinoma of unknown primary. It is expected that the subsequent therapeutic changes could enhance radiotherapy accuracy, and could improve the prognosis, toxicity profiles and quality-of-life of patients.</p><p><strong>Trial registration: </strong>NCT04767048, registered February, 19, 2021.</p>","PeriodicalId":9131,"journal":{"name":"BMC Cancer","volume":"25 1","pages":"551"},"PeriodicalIF":3.4,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11948765/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143728622","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}