Smith Lynette M, Mahoney Douglas W, Bamlet William R, Yu Fang, Liu Suyu, Goggins Michael G, Darabi Sourat, Majumder Shounak, Wang Qiao-Li, Coté Gregory A, Demeure Michael J, Zhang Zhen, Srivastava Sudhir, Chawla Akhil, Izmirlian Grant, Olson Janet E, Wolpin Brian M, Genkinger Jeanine M, Zaret Kenneth S, Brand Randall, Koay Eugene J, Oberg Ann L
Department of Biostatistics, University of Nebraska Medical Center, Omaha, NE, USA.
Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN, USA.
Pancreatology. 2024 Dec;24(8):1265-1279. doi: 10.1016/j.pan.2024.10.012. Epub 2024 Oct 29.
Pancreatic ductal adenocarcinoma (PDAC) is a highly lethal disease that is challenging to detect at an early stage. Biomarkers are needed that can detect PDAC early in the course of disease when interventions lead to the best outcomes. We highlight study design and statistical considerations that inform pancreatic cancer early detection biomarker evaluation.
We describe experimental design strategies in this setting useful for streamlining biomarker evaluation at each Early Detection Research Network (EDRN) phase of biomarker development. We break the early EDRN phases into sub-phases, proposing objectives, study design strategies, and biomarker performance benchmarks.
The goal of early detection in populations at high-risk of PDAC is described. Evaluating biomarker behavior in patients under surveillance without disease can winnow candidate biomarkers. Potential resources for biomarker validation studies are described.
Multisite and multidisciplinary collaboration can facilitate study design strategies in this lethal but low incidence disease and streamline the path from biomarker discovery to clinical use. Improvements in analytical and experimental design methods could help accelerate biomarker evaluation through the phases of biomarker development.
胰腺导管腺癌(PDAC)是一种致死率很高的疾病,早期检测具有挑战性。需要能够在疾病进程早期检测出PDAC的生物标志物,此时进行干预可带来最佳治疗效果。我们重点介绍有助于胰腺癌早期检测生物标志物评估的研究设计和统计学考量因素。
我们描述了在此背景下的实验设计策略,这些策略有助于在生物标志物开发的每个早期检测研究网络(EDRN)阶段简化生物标志物评估。我们将EDRN早期阶段细分为子阶段,提出目标、研究设计策略和生物标志物性能基准。
描述了在PDAC高危人群中进行早期检测的目标。在无疾病的受监测患者中评估生物标志物行为可以筛选出候选生物标志物。介绍了生物标志物验证研究的潜在资源。
多中心和多学科合作可以促进针对这种致死率高但发病率低的疾病的研究设计策略,并简化从生物标志物发现到临床应用的流程。分析和实验设计方法的改进有助于在生物标志物开发阶段加速生物标志物评估。