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First Edition
Clinical Chemistry and Toxicology

CLSI C64

Quantitative Measurement of Proteins and Peptides by Mass Spectrometry

CLSI C64 provides a comprehensive framework for developing and validating clinical protein and peptide assays using mass spectrometry (MS). Designed for laboratorians experienced in small-molecule LC-MS but new to protein and peptide analysis, this guideline addresses unique challenges, complex workflows, and clinically relevant validation strategies. It supports the accurate and reliable measurement of proteins and peptides in clinical diagnostics.

May 05, 2021
Cory Bystrom, PhD

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Abstract

Clinical and Laboratory Standards Institute guideline C64—Quantitative Measurement of Proteins and Peptides by Mass Spectrometry provides a framework for developing clinical protein and peptide assays from conception to validation. This guideline is intended for those who have experience with traditional small-molecule liquid chromatography–mass spectrometry (LC-MS) but not with protein and peptide analysis. Although closely related to traditional small-molecule analysis by LC-MS, protein and peptide analysis involves unique challenges and necessitates complex workflows, which are covered in detail. To enhance translation of assays to clinical use, this guideline focuses on method development aligned with clinically appropriate analytical validation.

Scope

This guideline provides broad recommendations for appropriately developing and validating quantitative protein and peptide assays for clinical applications using electrospray liquid chromatography–mass spectrometry (LC-MS) and liquid chromatography–tandem mass spectrometry (LC-MS/MS) techniques. CLSI C64 is practically focused and includes workflow overviews and experimental strategies for developing and validating quantitative assays for soluble proteins and peptides in biofluids (eg, serum, saliva, urine). It covers complex analyses, including measurement of proteins with post-translational modifications (PTMs). Although there are a diverse array of ionization modes and associated mass analyzers (eg, matrix-assisted laser desorption/ionization time-of-flight [MALDI-TOF] mass spectrometry [MS]), this guideline focuses on liquid chromatography (LC) and electrospray ionization (ESI) coupled with tandem mass spectrometry (MS/MS) because of the wide availability and proven utility of this method. 

A protein or peptide associated with a medical diagnosis or clinical outcome may exist in vivo as a single specified molecular composition or as a complex collection of related proteoforms that differ in molecular composition. Given the heterogenous nature of proteins and the desirability of facilitating results standardization among different assays, the need to appropriately define the measurand is a key difference between small-molecule analysis and protein analysis. In order to design a suitable workflow for measurand assessment, the assay developer needs to consider analyte properties, enrichment and fractionation strategy, and instrument performance characteristics. Subsequently, calibrators and internal standards (IS) are selected based on the chosen workflow and a precisely defined measurand. At the beginning of method development, performance criteria guide the conception and refinement of the path of workflow. Following this iterative process, the developer eventually prepares a candidate method sufficiently robust to pass validation. Finally, rigorous validation studies are performed to demonstrate suitability for routine clinical use. 

The intended users of this guideline are medical, research, and public health laboratories; in vitro diagnostic instrument manufacturers; and regulatory and accreditation organizations. 

Tissues and other nonbiofluid specimens are not within this guideline's scope. Enzyme activity assays are also considered out of scope, as are detailed discussions of software tools and data processing algorithms used for in silico analysis of protein and peptide sequences.

Product Details
C64Ed1E
978-1-68440-111-6
118
Authors
Cory Bystrom, PhD
Russell P. Grant, PhD
Lorin M. Bachmann, PhD, DABCC, MT(ASCP)
Nandkishor S. Chindarkar, PhD, DABCC
Mari DeMarco, PhD
Daniel T. Holmes, MD, FRCPC
Andrew N. Hoofnagle, MD, PhD
Daniel Intelmann, PhD
Doug Jeffery, PhD
Mark M. Kushnir, PhD
Paula Ladwig, MS, MT(ASCP)
Mark S. Lowenthal, PhD
Stephen R. Master, MD, PhD
Christopher M. Shuford, PhD
Stefani Thomas, PhD, NRCC
Jeffrey Whiteaker, PhD
Abstract

Clinical and Laboratory Standards Institute guideline C64—Quantitative Measurement of Proteins and Peptides by Mass Spectrometry provides a framework for developing clinical protein and peptide assays from conception to validation. This guideline is intended for those who have experience with traditional small-molecule liquid chromatography–mass spectrometry (LC-MS) but not with protein and peptide analysis. Although closely related to traditional small-molecule analysis by LC-MS, protein and peptide analysis involves unique challenges and necessitates complex workflows, which are covered in detail. To enhance translation of assays to clinical use, this guideline focuses on method development aligned with clinically appropriate analytical validation.

Scope

This guideline provides broad recommendations for appropriately developing and validating quantitative protein and peptide assays for clinical applications using electrospray liquid chromatography–mass spectrometry (LC-MS) and liquid chromatography–tandem mass spectrometry (LC-MS/MS) techniques. CLSI C64 is practically focused and includes workflow overviews and experimental strategies for developing and validating quantitative assays for soluble proteins and peptides in biofluids (eg, serum, saliva, urine). It covers complex analyses, including measurement of proteins with post-translational modifications (PTMs). Although there are a diverse array of ionization modes and associated mass analyzers (eg, matrix-assisted laser desorption/ionization time-of-flight [MALDI-TOF] mass spectrometry [MS]), this guideline focuses on liquid chromatography (LC) and electrospray ionization (ESI) coupled with tandem mass spectrometry (MS/MS) because of the wide availability and proven utility of this method. 

A protein or peptide associated with a medical diagnosis or clinical outcome may exist in vivo as a single specified molecular composition or as a complex collection of related proteoforms that differ in molecular composition. Given the heterogenous nature of proteins and the desirability of facilitating results standardization among different assays, the need to appropriately define the measurand is a key difference between small-molecule analysis and protein analysis. In order to design a suitable workflow for measurand assessment, the assay developer needs to consider analyte properties, enrichment and fractionation strategy, and instrument performance characteristics. Subsequently, calibrators and internal standards (IS) are selected based on the chosen workflow and a precisely defined measurand. At the beginning of method development, performance criteria guide the conception and refinement of the path of workflow. Following this iterative process, the developer eventually prepares a candidate method sufficiently robust to pass validation. Finally, rigorous validation studies are performed to demonstrate suitability for routine clinical use. 

The intended users of this guideline are medical, research, and public health laboratories; in vitro diagnostic instrument manufacturers; and regulatory and accreditation organizations. 

Tissues and other nonbiofluid specimens are not within this guideline's scope. Enzyme activity assays are also considered out of scope, as are detailed discussions of software tools and data processing algorithms used for in silico analysis of protein and peptide sequences.

C64Ed1E
978-1-68440-111-6
118
Authors
Cory Bystrom, PhD
Russell P. Grant, PhD
Lorin M. Bachmann, PhD, DABCC, MT(ASCP)
Nandkishor S. Chindarkar, PhD, DABCC
Mari DeMarco, PhD
Daniel T. Holmes, MD, FRCPC
Andrew N. Hoofnagle, MD, PhD
Daniel Intelmann, PhD
Doug Jeffery, PhD
Mark M. Kushnir, PhD
Paula Ladwig, MS, MT(ASCP)
Mark S. Lowenthal, PhD
Stephen R. Master, MD, PhD
Christopher M. Shuford, PhD
Stefani Thomas, PhD, NRCC
Jeffrey Whiteaker, PhD