Relationship of binding-site occupancy, transthyretin stabilisation and disease modification in patients with tafamidis-treated transthyretin amyloid cardiomyopathy

References

• Liz MA, Mar FM, Franquinho F, et al. Aboard transthyretin: from transport to cleavage. IUBMB Life. 2010;62(6):429–435.
   PubMed Web of Science ®Google Scholar
• Galant NJ, Westermark P, Higaki JN, et al. Transthyretin amyloidosis: an under-recognized neuropathy and cardiomyopathy. Clin Sci. 2017;131(5):395–409.
   PubMed Web of Science ®Google Scholar
• Ruberg FL, Grogan M, Hanna M, et al. Transthyretin amyloid cardiomyopathy: JACC state-of-the-art review. J Am Coll Cardiol. 2019;73(22):2872–2891.
   PubMed Web of Science ®Google Scholar
• Lane T, Fontana M, Martinez-Naharro A, et al. Natural history, quality of life, and outcome in cardiac transthyretin amyloidosis. Circulation 2019;140(1):16–26.
   PubMed Web of Science ®Google Scholar
• Grogan M, Scott CG, Kyle RA, 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):1014–1020.
   PubMed Web of Science ®Google Scholar
• Porcari A, Merlo M, Rapezzi C, et al. Transthyretin amyloid cardiomyopathy: an uncharted territory awaiting discovery. Eur J Intern Med. 2020;82:7–15.
   PubMed Web of Science ®Google Scholar
• Johnson SM, Connelly S, Fearns C, et al. The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug. J Mol Biol. 2012;421(2-3):185–203.
   PubMed Web of Science ®Google Scholar
• Maurer MS, Schwartz JH, Gundapaneni B, ATTR-ACT Study Investigators, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379(11):1007–1016.
   PubMed Web of Science ®Google Scholar
• Maurer MS, Grogan DR, Judge DP, et al. Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail. 2015;8(3):519–526.
   PubMed Web of Science ®Google Scholar
• Nelson LT, Paxman RJ, Xu J, et al. Blinded potency comparison of transthyretin kinetic stabilisers by subunit exchange in human plasma. Amyloid 2021;28(1):24–29.
   PubMed Web of Science ®Google Scholar
• Bulawa CE, Connelly S, DeVit M, et al. Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid Cascade. Proc Natl Acad Sci USA 2012;109(24):9629–9634.
   PubMed Web of Science ®Google Scholar
• Sekijima Y, Dendle MA, Kelly JW. Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid 2006;13(4):236–249.
   PubMed Web of Science ®Google Scholar
• Rappley I, Monteiro C, Novais M, et al. Quantification of transthyretin kinetic stability in human plasma using subunit exchange. Biochemistry 2014;53(12):1993–2006.
   PubMed Web of Science ®Google Scholar
• Kelly J, Powers E. Response. Amyloid 2021;28(2):140–141.
   PubMed Web of Science ®Google Scholar
• Wiseman RL, Green NS, Kelly JW. Kinetic stabilization of an oligomeric protein under physiological conditions demonstrated by a lack of subunit exchange: implications for transthyretin amyloidosis. Biochemistry 2005;44(25):9265–9274.
   PubMed Web of Science ®Google Scholar
• Petrassi HM, Klabunde T, Sacchettini J, et al. Structure-based design of N-phenyl phenoxazine transthyretin amyloid fibril inhibitors. J Am Chem Soc. 2000;122(10):2178–2192.
   Web of Science ®Google Scholar
• Freiburger L, Auclair K, Mittermaier A. Global ITC fitting methods in studies of protein allostery. Methods 2015;76:149–161.
   PubMed Web of Science ®Google Scholar
• Levitt DG, Levitt MD. Human serum albumin homeostasis: a new look at the roles of synthesis, catabolism, renal and gastrointestinal excretion, and the clinical value of serum albumin measurements. Int J Gen Med. 2016;9:229–255.
   PubMed Web of Science ®Google Scholar
• Banker MJ, Clark TH, Williams JA. Development and validation of a 96-well equilibrium dialysis apparatus for measuring plasma protein binding. J Pharm Sci. 2003;92(5):967–974.
   PubMed Web of Science ®Google Scholar
• Nilsen J, Trabjerg E, Grevys A, et al. An intact C-terminal end of albumin is required for its long half-life in humans. Commun Biol. 2020;3(1):181.
   PubMed Web of Science ®Google Scholar
• Socolow EL, Woeber KA, Purdy RH, et al. Preparation of I-131-labeled human serum prealbumin and its metabolism in normal and sick patients. J Clin Invest. 1965;44(10):1600–1609.
   PubMed Web of Science ®Google Scholar
• Huh Y, Riley S, Harnisch L, et al. Population pharmacokinetic modelling and simulation of tafamidis in healthy subjects and patients with transthyretin amyloidosis. Br J Clin Pharmacol. 2021;87(9):3574–3587.
   PubMed Web of Science ®Google Scholar
• Di L, Breen C, Chambers R, et al. Industry perspective on contemporary protein-binding methodologies: considerations for regulatory drug-drug interaction and related guidelines on highly bound drugs. J Pharm Sci. 2017;106(12):3442–3452.
   PubMed Web of Science ®Google Scholar
• Verona G, Mangione PP, Raimondi S, et al. Inhibition of the mechano-enzymatic amyloidogenesis of transthyretin: role of ligand affinity, binding cooperativity and occupancy of the inner channel. Sci Rep. 2017;7(1):182.
   PubMedGoogle Scholar