Evidence map›Paper›PMID 24063748›Full record

ArticleJournal of proteome research2013

Statistical design for biospecimen cohort size in proteomics-based biomarker discovery and verification studies.

Steven J Skates, Michael A Gillette, Joshua LaBaer, Steven A Carr, Leigh Anderson, Daniel C Liebler, David Ransohoff, Nader Rifai, Marina Kondratovich, Živana Težak and 9 more

Registry-linked trialAbstract readConsensus Development Conference, NIH
In one paragraph

Article in Journal of proteome research, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03067142 ("Pilot Study), which is not on this map. Cited by 72 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
72citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03067142 completednot on this mapstarted 2017, after this paper: background citation

"Pilot Study: Proteomics of Primary Hyperoxaluria Type 1 (PH1): A Rare Calcium Oxalate Stone Disease"

TypeobservationalSponsorAnn & Robert H Lurie Children's Hospital of ChicagoRan2017 to 2019Enrolled93ConditionsPrimary Hyperoxaluria Type 1ArmsObservational
3 · Its place in the literature

Who cites it

72 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
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  15. Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies.Pancreatology : official journal of the International Association of Pancreatology (IAP) ... [et al.] · 2024
    Review
  16. Article
  17. Article
  18. Article
  19. Proteomics techniques in protein biomarker discovery.Quantitative biology (Beijing, China) · 2024
    Review
  20. Article

12 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors.

Steven J SkatesBiostatistics Center, Massachusetts General Hospital Cancer Center , Boston, Massachusetts 02114, United States.
Michael A Gillette
Joshua LaBaer
Steven A Carr
Leigh Anderson
Daniel C Liebler
David Ransohoff
Nader Rifai
Marina Kondratovich
Živana Težak
Elizabeth Mansfield
Ann L Oberg
Ian Wright
Grady Barnes
Mitchell Gail
Mehdi Mesri
Christopher R Kinsinger
Henry Rodriguez
Emily S Boja

Funding

Women's Cancer ProgramP30CA015083 · NCI · MAYO CLINIC ROCHESTER · PI Felicity Enders · 1985 to 2026
$151.3M
Use of microfluidic tumor cultures to enable clinical trials of therapies for ovarian cancerP50CA136393 · NCI · MAYO CLINIC ROCHESTER · PI Zhenkun Lou · 2009 to 2026
$37.0M
Proteo-genomic Discovery, Prioritization and Verification of Cancer BiomarkersU24CA160034 · NCI · BROAD INSTITUTE, INC. · PI CARR, STEVEN A, PAULOVICH, AMANDA G · 2011 to 2016
$16.2M
Vanderbilt Proteome Characterization CenterU24CA159988 · NCI · VANDERBILT UNIVERSITY MEDICAL CENTER · PI LIEBLER, DANIEL C · 2011 to 2016
$13.9M
Proteomic, Genomic, and Longitudinal Pathways to Ovarian Cancer Biomarker DiscoveryU01CA152990 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI BIRRER, MICHAEL, SKATES, STEVEN J · 2010 to 2021
$8.9M
Intramural NIH HHS Z99 CA999999NCI NIH HHS P30 CA015083NCI NIH HHS P50 CA136393NCI NIH HHS P50CA136393NCI NIH HHS U01 CA152990NCI NIH HHS U01CA152990NCI NIH HHS U24 CA159988NCI NIH HHS U24 CA160034NCI NIH HHS U24CA160034
6 · The paper itself

Abstract

Protein biomarkers are needed to deepen our understanding of cancer biology and to improve our ability to diagnose, monitor, and treat cancers. Important analytical and clinical hurdles must be overcome to allow the most promising protein biomarker candidates to advance into clinical validation studies. Although contemporary proteomics technologies support the measurement of large numbers of proteins in individual clinical specimens, sample throughput remains comparatively low. This problem is amplified in typical clinical proteomics research studies, which routinely suffer from a lack of proper experimental design, resulting in analysis of too few biospecimens to achieve adequate statistical power at each stage of a biomarker pipeline. To address this critical shortcoming, a joint workshop was held by the National Cancer Institute (NCI), National Heart, Lung, and Blood Institute (NHLBI), and American Association for Clinical Chemistry (AACC) with participation from the U.S. Food and Drug Administration (FDA). An important output from the workshop was a statistical framework for the design of biomarker discovery and verification studies. Herein, we describe the use of quantitative clinical judgments to set statistical criteria for clinical relevance and the development of an approach to calculate biospecimen sample size for proteomic studies in discovery and verification stages prior to clinical validation stage. This represents a first step toward building a consensus on quantitative criteria for statistical design of proteomics biomarker discovery and verification research.

Indexed as

Gene Expression Regulation, NeoplasticAlgorithmsBiomarkers, TumorBlood ProteinsCohort StudiesHumansNeoplasm ProteinsNeoplasmsProteomicsResearch DesignSample SizeSensitivity and SpecificitySpecimen HandlingBiomarkers, TumorBlood ProteinsNeoplasm Proteins

Identifiers

PMID24063748
PMCPMC4039197

What OpenQuestion holds

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Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.