Context-of-use–Guided Development and Validation of a Transthyretin Immunoassay: A Framework for Biomarker Assay Design

Andrade C. A peculiar form of peripheral neuropathy: familiar atypical generalized amyloidosis with special involvement of the peripheral nerves. Brain. 1952;75(3):408–27.

Article  CAS  PubMed  Google Scholar 

Coelho T, Maurer MS, Suhr OB. THAOS – the transthyretin amyloidosis outcomes survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013;29(1):63–76.

Article  CAS  PubMed  Google Scholar 

Gonzalez-Lopez E, et al. Prognosis of transthyretin cardiac amyloidosis without heart failure symptoms. JACC CardioOncol. 2022;4(4):442–54.

Article  PubMed  PubMed Central  Google Scholar 

Grogan M, et al. Natural history of wild-type transthyretin cardiac amyloidosis and risk stratification using a novel staging system. J Am Coll Cardiol. 2016;68(10):1014–20.

Article  PubMed  Google Scholar 

Koike H, et al. Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas. J Neurol Neurosurg Psychiatry. 2012;83(2):152–8.

Article  PubMed  Google Scholar 

Mariani L-L, et al. Genotype–phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France. Ann Neurol. 2015;78(6):901–16.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Sattianayagam PT, et al. Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant. Eur Heart J. 2011;33(9):1120–7.

Article  PubMed  Google Scholar 

Robbins J. Transthyretin from discovery to now. Clin Chem Lab Med. 2002;40(12):1183–90.

Article  CAS  PubMed  Google Scholar 

Raghu P, Sivakumar B. Interactions amongst plasma retinol-binding protein, transthyretin and their ligands: implications in vitamin A homeostasis and transthyretin amyloidosis. Biochim Biophys Acta. 2004;1703(1):1–9.

Article  CAS  PubMed  Google Scholar 

Connelly S, et al. Structure-based design of kinetic stabilizers that ameliorate the transthyretin amyloidoses. Curr Opin Struct Biol. 2010;20(1):54–62.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Johnson SM, et al. Native state kinetic stabilization as a strategy to ameliorate protein misfolding diseases: a focus on the transthyretin amyloidoses. Acc Chem Res. 2005;38(12):911–21.

Article  CAS  PubMed  Google Scholar 

Coelho T, et al. Eplontersen for hereditary Transthyretin amyloidosis with polyneuropathy. JAMA. 2023;330(15):1448–58.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hanson JLS, et al. Use of serum transthyretin as a prognostic indicator and predictor of outcome in cardiac amyloid disease associated with wild-type transthyretin. Circ Heart Fail. 2018;11(2):e004000.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Chan GG, Koch CM, Connors LH. Blood proteomic profiling in inherited (ATTRm) and acquired (ATTRwt) forms of transthyretin-associated cardiac amyloidosis. J Proteome Res. 2017;16(4):1659–68.

Article  CAS  PubMed  Google Scholar 

Cowan KJ, et al. Recommendations for Selection and Characterization of Protein Biomarker Assay Calibrator Material. AAPS j. 2017;19(6):1550–63.

Article  CAS  PubMed  Google Scholar 

Leary BA, et al. Bioanalytical platform comparison using a generic human IgG PK assay format. J Immunol Methods. 2013;397(1):28–36.

Article  CAS  PubMed  Google Scholar 

Lee JW, et al. Fit-for-purpose method development and validation for successful biomarker measurement. Pharm Res. 2006;23(2):312–28.

Article  CAS  PubMed  Google Scholar 

Findlay JW, Dillard RF. Appropriate calibration curve fitting in ligand binding assays. AAPS J. 2007;9(2):E260–7.

Article  PubMed  PubMed Central  Google Scholar 

Xiang Y, et al. A simple approach to determine a curve fitting model with a correct weighting function for calibration curves in quantitative ligand binding assays. AAPS J. 2018;20(3):45.

Article  CAS  PubMed  Google Scholar 

Piccoli SP, Brad Ackermann JMS, Allinson J, Arnold M, Amur S. Points to Consider Document: Scientific and Regulatory Considerations for the Analytical Validation of Assays Used in the Qualification of Biomarkers in Biological Matrices. C – Path; 2019.

Hammarström P, et al. Sequence-dependent denaturation energetics: a major determinant in amyloid disease diversity. Proc Natl Acad Sci. 2002;99(suppl_4):16427–32.

Article  PubMed  PubMed Central  Google Scholar 

Benson MD, et al. Inotersen treatment for patients with hereditary Transthyretin amyloidosis. N Engl J Med. 2018;379(1):22–31.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Jiang X, et al. An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured. Biochemistry. 2001;40(38):11442–52.

Article  CAS  PubMed  Google Scholar 

Ni YG, et al. Why is biomarker assay validation different from that of pharmacokinetic assays? AAPS J. 2025;27(6):147.

Article  PubMed  Google Scholar 

van der Vorm LN, et al. Toward Worldwide Hepcidin Assay Harmonization: Identification of a Commutable Secondary Reference Material. Clin Chem. 2016;62(7):993–1001.

Article  PubMed  Google Scholar 

Mollenhauer B, et al. Antibody-based methods for the measurement of α-synuclein concentration in human cerebrospinal fluid - method comparison and round robin study. J Neurochem. 2019;149(1):126–38.

Article  CAS  PubMed  Google Scholar 

Schoonenboom NS, et al. Differences and similarities between two frequently used assays for amyloid beta 42 in cerebrospinal fluid. Clin Chem. 2005;51(6):1057–60.

Article  CAS  PubMed  Google Scholar 

Arslan B, et al. Method comparison of plasma and CSF GFAP immunoassays across multiple platforms. Clinical Chemistry and Laboratory Medicine (CCLM). 2025. https://doi.org/10.1515/cclm-2025-0667.

Article  PubMed  Google Scholar 

Keller S, Purushothama S. Biomarker assays: is it time for a standalone regulatory guidance? Bioanalysis. 2018;10(23):1893–5.

Article  CAS  PubMed  Google Scholar 

Goodman J, et al. Update to the European Bioanalysis Forum recommendation on biomarkers assays; bringing context of use into practice. Bioanalysis. 2020;12(20):1427–37.

Article  CAS  PubMed  Google Scholar 

Health NIO. FDA-NIH Biomarker Working Group. BEST (Biomarkers, EndpointS, and other Tools) Resource. Bethesda (MD). 2016.

Krueger K, et al. High quality performance of novel immunoassays for the sensitive quantification of soluble PD-1, PD-L1 and PD-L2 in blood. Biomedicines. 2022;10(10):2405.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Soderstrom CI, et al. Comparison of four distinct detection platforms using multiple ligand binding assay formats. J Immunol Methods. 2011;371(1):106–13.

Article  CAS  PubMed  Google Scholar 

Lee S, et al. Development of a Highly Sensitive Neurofilament Light Chain Assay on an Automated Immunoassay Platform. Front Neurol. 2022;13:935382.

Comments (0)

No login
gif