Predictive Biomarkers for Early Diagnosis of Pancreatic Ductal Adenocarcinoma (PDAC) (B-PDAC)
研究概览
地位
详细说明
Pancreatic ductal adenocarcinoma (PDAC) is currently one of the leading causes of cancer-related mortality worldwide and is characterized by late diagnosis, high biological aggressiveness, and marked resistance to systemic therapies. Epidemiologic projections indicate that, by 2030, PDAC will become the second most common cause of cancer death in Western countries, highlighting the need for improved early-diagnosis strategies and therapeutic interventions. Most PDACs arise from non-invasive precursor lesions through a multistep neoplastic transformation process. Key precursor entities include pancreatic intraepithelial neoplasia (PanIN) and mucinous cystic lesions, particularly intraductal papillary mucinous neoplasms (IPMN), which are clinically detectable by imaging, biologically heterogeneous, and associated with variable malignant potential. The widespread use of high-resolution imaging has increased incidental detection of pancreatic cysts, but only a subset of these lesions will progress to invasive carcinoma, making clinical management challenging.
In this context, the identification of reliable molecular biomarkers capable of predicting progression to invasive carcinoma is a clinical and scientific priority. This single-center, prospective observational translational study will include three arms: patients with IPMN, patients with PanIN, and patients with PDAC confirmed by histopathologic examination after pancreatic resection. Residual tissue not needed for diagnostic histopathology will be processed following standardized procedures to ensure high-quality biospecimens and full traceability. Fresh samples will be used for isolation of tumor stem-like cells and establishment of three-dimensional cultures (tumorspheres and organoids); additional material will be fixed in formalin and embedded in paraffin (FFPE) for histopathologic and immunohistochemical analyses.
Molecular analyses will comprise next-generation sequencing (NGS) to identify somatic mutations, genomic instability, tumor mutational burden (TMB), and alterations in key oncogenic genes and pathways; RNA-sequencing (RNA-seq) to characterize transcriptional programs associated with neoplastic transformation; protein expression studies by immunofluorescence, immunohistochemistry, and Western blot focusing on proliferation, apoptosis, and tumor stemness markers; advanced cellular imaging to study proliferation, survival, invasiveness, and metabolic adaptation under stress; and in-vitro pharmacologic assays to assess sensitivity of patient-derived models to inhibitors targeting the identified biomarkers. Statistical analyses will include Monte Carlo-based sample-size estimation, group comparisons using parametric and non-parametric tests, logistic regression models, ROC curve analyses, and high-dimensional bioinformatic pipelines for differential expression, clustering, and multivariate pattern recognition. The ultimate aim is to define and validate molecular signatures predictive of progression toward PDAC, correlate them with clinical and pathological features, and generate organoid models for preclinical studies in precision oncology.
研究类型
注册 (估计的)
联系人和位置
学习联系方式
- 姓名:Raffaele Armentano, MD
- 电话号码:+390804994185
- 邮箱:raffaele.armentano@irccsdebellis.it
研究联系人备份
- 姓名:Martina Lepore Signorile, Biologist
- 邮箱:martina.lepore@irccsdebellis.it
参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
取样方法
研究人群
描述
Inclusion Criteria:
- Age ≥ 18 years.
- Patients undergoing pancreatic surgical resection for suspected pancreatic neoplasm.
- Histopathologic diagnosis of intraductal papillary mucinous neoplasm (IPMN), pancreatic intraepithelial neoplasia (PanIN), or pancreatic ductal adenocarcinoma (PDAC).
- Availability of residual tumor tissue from the surgical procedure not required for diagnostic purposes.
- Signed written informed consent for the use of biological samples for research purposes
Exclusion Criteria:
- Age < 18 years.
- Surgical procedure performed for neoplasms other than IPMN, PanIN, or PDAC.
- Absence of written informed consent.
学习计划
研究是如何设计的?
设计细节
队列和干预
团体/队列 |
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IPMN Cohort
Adult patients undergoing pancreatic resection with histopathologic diagnosis of intraductal papillary mucinous neoplasm (IPMN); residual tumor tissue and, when available, adjacent non neoplastic tissue will be collected for molecular analyses and organoid generation
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PanIN Cohort
Adult patients undergoing pancreatic resection with histopathologic diagnosis of pancreatic intraepithelial neoplasia (PanIN); residual tissue will be used for genomic, transcriptomic, proteomic, and functional studies in patient derived models
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PDAC Cohort
Adult patients undergoing pancreatic resection with histopathologic diagnosis of pancreatic ductal adenocarcinoma (PDAC); biospecimens will be processed for comprehensive molecular characterization and comparison with precursor lesions
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Molecular biomarkers predictive of progression to pancreatic ductal adenocarcinoma (PDAC
大体时间:Up to 36 months from surgery
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Identification of genomic, transcriptomic, and proteomic biomarkers associated with progression from pancreatic precursor lesions (IPMN, PanIN) to PDAC, based on NGS, RNA seq, protein expression assays, and advanced imaging in patient derived tumorspheres and organoids.
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Up to 36 months from surgery
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Protein level validation of candidate biomarkers
大体时间:Up to 36 months from surgery
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Validation of selected predictive biomarkers at the protein level by immunohistochemistry, immunofluorescence, and Western blot in tissue and organoid samples from IPMN, PanIN, and PDAC patients.
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Up to 36 months from surgery
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Association between biomarker profiles and tumor progression
大体时间:Up to 36 months
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Evaluation of correlations between biomarker expression profiles and clinical, histopathologic, and imaging indicators of tumor progression, using logistic regression and ROC curve analyses.
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Up to 36 months
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Establishment of patient derived organoid models for preclinical studies
大体时间:Up to 36 months
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Generation and characterization of stable patient derived tumorsphere and organoid models from IPMN, PanIN, and PDAC samples suitable for preclinical drug testing and precision medicine applications
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Up to 36 months
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合作者和调查者
调查人员
- 首席研究员:Raffaele Armentano, MD、IRCCS "Saverio de Bellis"
出版物和有用的链接
一般刊物
- Siegel RL, Miller KD, Fuchs HE, Jemal A. Cancer statistics, 2022. CA Cancer J Clin. 2022 Jan;72(1):7-33. doi: 10.3322/caac.21708. Epub 2022 Jan 12.
- Rahib L, Coffin T, Kenner B. Factors Driving Pancreatic Cancer Survival Rates. Pancreas. 2025 Jul 1;54(6):e530-e536. doi: 10.1097/MPA.0000000000002489.
- Marsoner K, Haybaeck J, Csengeri D, Waha JE, Schagerl J, Langeder R, Mischinger HJ, Kornprat P. Pancreatic resection for intraductal papillary mucinous neoplasm- a thirteen-year single center experience. BMC Cancer. 2016 Nov 4;16(1):844. doi: 10.1186/s12885-016-2887-8.
- Muraki T, Jang KT, Reid MD, Pehlivanoglu B, Memis B, Basturk O, Mittal P, Kooby D, Maithel SK, Sarmiento JM, Christians K, Tsai S, Evans D, Adsay V. Pancreatic ductal adenocarcinomas associated with intraductal papillary mucinous neoplasms (IPMNs) versus pseudo-IPMNs: relative frequency, clinicopathologic characteristics and differential diagnosis. Mod Pathol. 2022 Jan;35(1):96-105. doi: 10.1038/s41379-021-00902-x. Epub 2021 Sep 13.
- Taherian M, Wang H, Wang H. Pancreatic Ductal Adenocarcinoma: Molecular Pathology and Predictive Biomarkers. Cells. 2022 Sep 29;11(19):3068. doi: 10.3390/cells11193068.
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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