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References


References

  1. Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamids treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018:379(11):1007-1016.
  2. Nativi-Nicolau J, Maurer MS. Amyloidosis cardiomyopathy: update in the diagnosis and treatment of the most common types. Curr Opin Cardiol. 2018;33(5):571-579.
  3. Narotsky DL, Castano A, Weinsaft JW, Bokhari S, Maurer MS. Wild-type transthyretin cardiac amyloidosis: novel insights from advanced imaging. Can J Cardiol. 2016;32(9):1166.e1-1166.e10.
  4. Sipe JD, Benson MD, Buxbaum JN, et al. Amyloid fibril proteins and amyloidosis: chemical identification and clinical classification International Society of Amyloidosis 2016 Nomenclature Guidelines. Amyloid. 2016;23(4):209-213.
  5. Maurer MS, Elliott P, Comenzo R, Semigran M, Rapezzi C. Addressing common questions encountered in the diagnosis and management of cardiac amyloidosis. Circulation. 2017;135(14):1357-1377.
  6. González-López E, Gallego-Delgado M, Guzzo-Merello G, et al. Wild-type transthyretin amyloidosis as a cause of heart failure with preserved ejection fraction. Eur Heart J. 2015;36(38):2585-2594.
  7. Mohammed SF, Mirzoyev SA, Edwards WD, et al. Left ventricular amyloid deposition in patients with heart failure and preserved ejection fraction. JACC Heart Fail. 2014;2(2):113-122.
  8. Castaño A, Narotsky D, Maurer MS, et al. Emerging therapies for transthyretin cardiac amyloidosis could herald a new era for the treatment of HFPEF. J Am Coll Cardiol. 2015. https://www.acc.org/latest-in-cardiology/articles/2015/10/13/08/35/emerg.... Accessed January 4, 2019.
  9. 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 U S A. 2012;109(24):9629-9634.
  10. VyndamaxTM (Tafamidis 61mg) Hong Kong Prescribing Information. July 2020.
  11. Tankisheva E. A phase 1, open-label, randomized, crossover, multiple dose, pivotal bioequivalence study to compare PF-06291826 4 × 20 mg tafamidis meglumine and 61 mg: tafamidis free acid soft gelatin capsules administered under fasted conditions to healthy volunteers. Full Clinical Study Report [protocol B3461056]. July 23, 2018.
  12. Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy [supplemental appendix]. N Engl J Med. 2018;379(11):1007-1016. https://nejm.org/doi/suppl/10.1056/NEJMoa1805689/suppl_file/nejmoa180568.... Accessed July 1, 2019.
  13. Maurer MS, Mann DL. The Tafamidis Drug Development Program: A Translational Tripumph. JACC Basic Transl Sci. 2018: 31;3(6):871-873.
  14. Barroso FA, Judge DP, Ebede B, et al. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017;24(3):194-204.
  15. Adams D, Suhr OB, Hund E, et al. European Network for TTR-FAP (ATTReuNET). First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy. Curr Opin Neurol. 2016 Feb;29(suppl 1):S14-S26.
  16. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
  17. Sekijima Y, Ueda M, Koike H, Misawa S, Ishii T, Ando Y. Diagnosis and management of transthyretin familial amyloid polyneuropathy in Japan: red-flag symptom clusters and treatment algorithm. Orphanet J Rare Dis. 2018;13(1):6.
  18. Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related amyloidosis and the heart: a clinical overview. Nat Rev Cardiol 2010;7(7):398-408.
  19. Damy T, Kristen AV, Suhr OB, et al. Transthyretin cardiac amyloidosis in continental Western Europe: An Insight Through the Transthyretin Amyloidosis Outcomes Survey (THAOS). Eur Heart J. 2019;0:1-10.
  20. Vyndaqel® (Tafamidis) Hong Kong Prescribing Information. December 2017.
  21. Wixner J, Mundayat R, Karayal ON, Anan I, Karling P, Suhr OB; THAOS investigators. THAOS: gastrointestinal manifestations of transthyretin amyloidosis – common complications of a rare disease. Orphanet J Rare Dis. 2014;9:61.
  22. Suhr OB, Conceição IM, Karayal ON, Mandel FS, Huertas PE, Ericzon BG. Post hoc analysis of nutritional status in patients with transthyretin familial amyloid polyneuropathy: impact of tafamidis. Neurol Ther. 2014;3(2):101-112.
  23. Coelho T, Maia LF, Martins do Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012:79(8):785-792.
  24. Merlini G, Plante-Bordeneuve V, Judge D, et al. Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. J Cardiovasc Transl Res. 2013;6(6):1011-1020.
  25. Clinical Report for Protocol Fx-005. New York, NY: Pfizer Inc.; May 2011.
  26. Clinical Study Report for Protocol Fx1A-201. New York, NY: Pfizer Inc.; May 2011.
  27. Gundapaneni BK, Sultan MB, Keohane DJ, Schwartz JH. Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy. Eur J Neurol. 2018;25(3):464-468.
  28. 2.7.4 Summary of Clinical Safety. New York, NY: Pfizer Inc., September 2017.
  29. Huber P, Flynn A, Sultan MB, et al. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019;27(7):1-7.
  30. Clinical Study Report for Protocol B3461023. New York, NY: Pfizer Inc., August 2015.
  31. Brunjes DL, Castano A, Clemons A, Rubin J, Maurer MS. Transthyretin cardiac amyloidosis in older Americans. J Card Fail. 2016;22(12):996-1003.
  32. Siddiqi OK, Ruberg FL. Cardiac amyloidosis: an update on pathophysiology, diagnosis, and treatment. Trends Cardiovasc Med. 2018;28(1):10-21.
  33. Castaño A, Narotsky DL, Hamid N, et al. Unveiling transthyretin cardiac amyloidosis and its predictors amongelderly patients with severe aortic stenosis undergoing transcatheter aortic valve replacement. Eur Heart J. 2017;38:2879–2887.
  34. Treibel TA, Fontana M, Gilbertson JA, et al. Occult transthyretin cardiac amyloid in severe calcific aortic stenosis: prevalence and prognosis in patients undergoing surgical aortic valve replacement. Circ Cardiovasc Imaging. 2016;9:54-63.
  35. Mints YY, Doros G, Berk JL, et al. Features of atrial fibrillation in wild‐type transthyretin cardiac amyloidosis: a systematic review and clinical experience. ESC Heart Failure. 2018;5:772-779.
  36. Maurer MS, Hanna M, Grogan M, et al. Genotype and phenotype of transthyretin cardiac amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016;63(2):161-172.
  37. Conceiçāo I, González-Duarte A, Obici L, et al. "Red-flag" symptom clusters in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2016;21(1):5-9.
  38. Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;36(4):411-423.
  39. Sekijima Y, Yoshida K, Tokuda T, Ikeda S. Familial transthyretin amyloidosis. In: Pagon RA, Adam MP, Ardinger HH, et al, eds. GeneReviews [Internet]. Seattle WA: University of Washington, 1993-2016.
  40. Plante-Bordeneuve V. Update in the diagnosis and management of transthyretin familial amyloid polyneuropathy. J Neurol. 2014;261(6):1227-1233.
  41. Roberts JR, Lan ML, Besa EC, et al. Transthyretin-related amyloidosis. Medscape website. http://emedicine.medscape.com/article/335301-overview. Accessed October 2, 2018.
  42. Conceição I, Coelho T, Rapezzi C, et al. Assessment of patients with hereditary transthyretin amyloidosis—understanding the impact of management and disease progression. Amyloid. 2019;26(3):103-111.
  43. Gillmore JD, Maurer MS, Falk RH, et al. Nonbiopsy diagnosis of cardiac transthyretin amyloidosis. Circulation. 2016;133(24):2404-2412.
  44. Bokhari S, Morgenstern R, Weinberg R, et al. Standardization of 99mTechnetium pyrophosphate, imaging methodology to diagnose TTR cardiac amyloidosis. J Nucl Cardiol. 2018;25(1):181-190.
  45. Bhambhvani P, Hage FG. Nuclear imaging of cardiac amyloidosis. "We've only just begun." J Nucl Cardiol. 2018;25(1):191-194.
  46. American Society of Nuclear Cardiology (ASNC). ASNC practice points: 99mTechnetium-pyrophosphate imaging for transthyretin cardiac amyloidosis. https://www.asnc.org/files/19110%20ASNC%20Amyloid%20Practice%20Points%20WEB(2).pdf Published February 2016. Updated February 2019. Accessed June 5, 2019.
  47. Adams D, Théaudin M, Cauquil C, Algalarrondo V, Slama M. FAP neuropathy and emerging treatments. Curr Neurol Neurosci Rep. 2014;14(3):435.