Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies

Lynette M. Smith; Douglas W. Mahoney; William R. Bamlet; Fang Yu; Suyu Liu; Michael G. Goggins; Sourat Darabi; Shounak Majumder; Qiao Li Wang; Gregory A. Coté; Michael J. Demeure; Zhen Zhang; Sudhir Srivastava; Akhil Chawla; Grant Izmirlian; Janet E. Olson; Brian M. Wolpin; Jeanine M. Genkinger; Kenneth S. Zaret; Randall Brand; Eugene J. Koay; Ann L. Oberg

doi:10.1016/j.pan.2024.10.012

Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies

Lynette M. Smith, Douglas W. Mahoney, William R. Bamlet, Fang Yu, Suyu Liu, Michael G. Goggins, Sourat Darabi, Shounak Majumder, Qiao Li Wang, Gregory A. Coté, Michael J. Demeure, Zhen Zhang, Sudhir Srivastava, Akhil Chawla, Grant Izmirlian, Janet E. Olson, Brian M. Wolpin, Jeanine M. Genkinger, Kenneth S. Zaret, Randall BrandEugene J. Koay, Ann L. Oberg

Research output: Contribution to journal › Review article › peer-review

1 Scopus citations

Abstract

Objectives: 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. Methods: 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. Results: 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. Conclusions: 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.

Original language	English (US)
Pages (from-to)	1265-1279
Number of pages	15
Journal	Pancreatology
Volume	24
Issue number	8
DOIs	https://doi.org/10.1016/j.pan.2024.10.012
State	Published - Dec 2024

Keywords

Biomarker development
Early detection
Experimental design
Pancreatic cancer
Validation

ASJC Scopus subject areas

Endocrinology, Diabetes and Metabolism
Hepatology
Endocrinology

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10.1016/j.pan.2024.10.012

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Smith, L. M., Mahoney, D. W., Bamlet, W. R., Yu, F., Liu, S., Goggins, M. G., Darabi, S., Majumder, S., Wang, Q. L., Coté, G. A., Demeure, M. J., Zhang, Z., Srivastava, S., Chawla, A., Izmirlian, G., Olson, J. E., Wolpin, B. M., Genkinger, J. M., Zaret, K. S., ... Oberg, A. L. (2024). Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies. Pancreatology, 24(8), 1265-1279. https://doi.org/10.1016/j.pan.2024.10.012

Smith, LM, Mahoney, DW, Bamlet, WR, Yu, F, Liu, S, Goggins, MG, Darabi, S, Majumder, S, Wang, QL, Coté, GA, Demeure, MJ, Zhang, Z, Srivastava, S, Chawla, A, Izmirlian, G, Olson, JE, Wolpin, BM, Genkinger, JM, Zaret, KS, Brand, R, Koay, EJ & Oberg, AL 2024, 'Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies', Pancreatology, vol. 24, no. 8, pp. 1265-1279. https://doi.org/10.1016/j.pan.2024.10.012

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title = "Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies",

abstract = "Objectives: 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. Methods: 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. Results: 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. Conclusions: 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.",

keywords = "Biomarker development, Early detection, Experimental design, Pancreatic cancer, Validation",

author = "Smith, {Lynette M.} and Mahoney, {Douglas W.} and Bamlet, {William R.} and Fang Yu and Suyu Liu and Goggins, {Michael G.} and Sourat Darabi and Shounak Majumder and Wang, {Qiao Li} and Cot{\'e}, {Gregory A.} and Demeure, {Michael J.} and Zhen Zhang and Sudhir Srivastava and Akhil Chawla and Grant Izmirlian and Olson, {Janet E.} and Wolpin, {Brian M.} and Genkinger, {Jeanine M.} and Zaret, {Kenneth S.} and Randall Brand and Koay, {Eugene J.} and Oberg, {Ann L.}",

note = "Publisher Copyright: {\textcopyright} 2024 The Authors",

year = "2024",

month = dec,

doi = "10.1016/j.pan.2024.10.012",

language = "English (US)",

volume = "24",

pages = "1265--1279",

journal = "Pancreatology",

issn = "1424-3903",

publisher = "Elsevier B.V.",

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}

TY - JOUR

T1 - Early detection of pancreatic cancer

T2 - Study design and analytical considerations in biomarker discovery and early phase validation studies

AU - Smith, Lynette M.

AU - Mahoney, Douglas W.

AU - Bamlet, William R.

AU - Yu, Fang

AU - Liu, Suyu

AU - Goggins, Michael G.

AU - Darabi, Sourat

AU - Majumder, Shounak

AU - Wang, Qiao Li

AU - Coté, Gregory A.

AU - Demeure, Michael J.

AU - Zhang, Zhen

AU - Srivastava, Sudhir

AU - Chawla, Akhil

AU - Izmirlian, Grant

AU - Olson, Janet E.

AU - Wolpin, Brian M.

AU - Genkinger, Jeanine M.

AU - Zaret, Kenneth S.

AU - Brand, Randall

AU - Koay, Eugene J.

AU - Oberg, Ann L.

PY - 2024/12

Y1 - 2024/12

N2 - Objectives: 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. Methods: 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. Results: 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. Conclusions: 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.

AB - Objectives: 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. Methods: 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. Results: 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. Conclusions: 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.

KW - Biomarker development

KW - Early detection

KW - Experimental design

KW - Pancreatic cancer

KW - Validation

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DO - 10.1016/j.pan.2024.10.012

M3 - Review article

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AN - SCOPUS:85208483083

SN - 1424-3903

VL - 24

SP - 1265

EP - 1279

JO - Pancreatology

JF - Pancreatology

IS - 8

ER -

Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies

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Keywords

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