Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial

References

• Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care. 2017;23:S107–S112.
   PubMed Web of Science ®Google Scholar
• Lousada I, Comenzo RL, Landau H, et al. Light chain amyloidosis: patient experience survey from the Amyloidosis Research Consortium. Adv Ther. 2015;32:920–928.
   PubMed Web of Science ®Google Scholar
• Sekijima Y, Kelly JW, Ikeda S. Pathogenesis of and therapeutic strategies to ameliorate the transthyretin amyloidoses. Curr Pharm Des. 2008;14:3219–3230.
   PubMed Web of Science ®Google Scholar
• Falk RH, Comenzo RL, Skinner M. The systemic amyloidoses. N Engl J Med. 1997;337:898–909.
   PubMed Web of Science ®Google Scholar
• Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch Neurol. 2005;62:1057–1062.
   PubMed Web of Science ®Google Scholar
• Castano A, Bokhari S, Maurer MS. Unveiling wild-type transthyretin cardiac amyloidosis as a significant and potentially modifiable cause of heart failure with preserved ejection fraction. Eur Heart J. 2015;36:2595–2597.
   PubMed Web of Science ®Google Scholar
• Lobato L, Beirao I, Silva M, et al. Familial ATTR amyloidosis: microalbuminuria as a predictor of symptomatic disease and clinical nephropathy. Nephrol Dial Transplant. 2003;18:532–538.
   PubMed Web of Science ®Google Scholar
• Lobato L, Rocha A. Transthyretin amyloidosis and the kidney. Clin J Am Soc Nephrol. 2012;7:1337–1346.
   PubMed Web of Science ®Google Scholar
• Dubrey SW, Hawkins PN, Falk RH. Amyloid diseases of the heart: assessment, diagnosis, and referral. Heart. 2011;97:75–84.
   PubMed Web of Science ®Google Scholar
• Coelho T, Ericzon B, Falk R, et al. A guide to transthyretin amyloidosis. Amyloidosis Foundation, 2016 [cited 2018 Jul 30]. Available at http://www.amyloidosis.org/wp-content/uploads/2017/05/2017-ATTR-guide.pdf.
   Google Scholar
• Parman Y, Adams D, Obici L, et al. Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe: where are we now? A European network approach to defining the epidemiology and management patterns for TTR-FAP. Curr Opin Neurol. 2016;29:S3–S13.
   PubMed Web of Science ®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;29:63–76.
   PubMed Web of Science ®Google Scholar
• Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;36:411–423.
   PubMed Web of Science ®Google Scholar
• Plante-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol. 2011;10:1086–1097.
   PubMed Web of Science ®Google Scholar
• Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
   PubMed Web of Science ®Google Scholar
• Galant NJ, Westermark P, Higaki JN, et al. Transthyretin amyloidosis: an under-recognized neuropathy and cardiomyopathy. Clin Sci (Lond). 2017;131:395–409.
   PubMed Web of Science ®Google Scholar
• Rapezzi C, Quarta CC, Obici L, et al. Disease profile and differential diagnosis of hereditary transthyretin-related amyloidosis with exclusively cardiac phenotype: an Italian perspective. Eur Heart J. 2013;34:520–528.
   PubMed Web of Science ®Google Scholar
• Mariani LL, Lozeron P, Theaudin M, et al. Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France. Ann Neurol. 2015;78:901–916.
   PubMed Web of Science ®Google Scholar
• Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379:22–31.
   PubMed Web of Science ®Google Scholar
• Ackermann EJ, Guo S, Benson MD, et al. Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides. Amyloid. 2016;23:148–157.
   PubMed Web of Science ®Google Scholar
• Coutinho P, da Silva AM, Lima JL, et al. Forty years of experience with type 1 amyloid neuropathy: review of 483 cases. In: Glenner GG, e Costa PP, de Freitas AF, editors. Amyloid and Amyloidosis: Proceedings of the Third International Symposium on Amyloidosis, 1979 Sep 23–28; Póvoa de Varzim, Portugal. Amsterdam: Excerpta Medica. 1980. p. 88–98.
   Google Scholar
• Dyck PJ, Kincaid JC, Dyck PJB, et al. Assessing mNIS +7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial. Muscle Nerve. 2017;56:901–911.
   PubMed Web of Science ®Google Scholar
• Dyck PJ, Zimmerman IR, O'Brien PC, et al. Introduction of automated systems to evaluate touch-pressure, vibration, and thermal cutaneous sensation in man. Ann Neurol. 1978;4:502–510.
   PubMed Web of Science ®Google Scholar
• Dyck PJ, Herrmann DN, Staff NP, et al. Assessing decreased sensation and increased sensory phenomena in diabetic polyneuropathies. Diabetes. 2013;62:3677–3686.
   PubMed Web of Science ®Google Scholar
• Dyck PJ, Argyros B, Russell JW, et al. Multicenter trial of the proficiency of smart quantitative sensation tests. Muscle Nerve. 2014;49:645–653.
   PubMed Web of Science ®Google Scholar
• Davies JL, Engelstad JK, Sr., Gove LE, et al. Somatotopic heat pain thresholds and intraepidermal nerve fibers in health. Muscle Nerve. 2018. DOI:10.1002/mus.26128
   PubMed Web of Science ®Google Scholar
• Vinik EJ, Vinik AI, Paulson JF, et al. Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2014;19:104–114.
   PubMed Web of Science ®Google Scholar
• Ware JE, Jr., Gandek B. Overview of the SF-36 health survey and the international quality of life assessment (IQOLA) project. J Clin Epidemiol. 1998;51:903–912.
   PubMed Web of Science ®Google Scholar
• Dyck PJ, Turner DW, Davies JL, et al. Electronic case-report forms of symptoms and impairments of peripheral neuropathy. Can J Neurol Sci. 2002;29:258–266.
   PubMed Web of Science ®Google Scholar
• Rapezzi C, Merlini G, Quarta CC, et al. Systemic cardiac amyloidoses: disease profiles and clinical courses of the 3 main types. Circulation. 2009;120:1203–1212.
   PubMed Web of Science ®Google Scholar
• Damy T, Deux JF, Moutereau S, et al. Role of natriuretic peptide to predict cardiac abnormalities in patients with hereditary transthyretin amyloidosis. Amyloid. 2013;20:212–220.
   PubMed Web of Science ®Google Scholar
• Swiecicki PL, Zhen DB, Mauermann ML, et al. Hereditary ATTR amyloidosis: a single-institution experience with 266 patients. Amyloid. 2015;22:123–131.
   PubMed Web of Science ®Google Scholar
• Cortese A, Vegezzi E, Lozza A, et al. Diagnostic challenges in hereditary transthyretin amyloidosis with polyneuropathy: avoiding misdiagnosis of a treatable hereditary neuropathy. J Neurol Neurosurg Psychiatry. 2017;88:457–458.
   PubMed Web of Science ®Google Scholar
• Adams D, Coelho T, Obici L, et al. Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study. Neurology. 2015;85:675–682.
   PubMed Web of Science ®Google Scholar
• Adams D, Lozeron P, Theaudin M, et al. Regional difference and similarity of familial amyloidosis with polyneuropathy in France. Amyloid. 2012;19:61–64.
   PubMed Web of Science ®Google Scholar
• Ando Y, Araki S, Ando M. Transthyretin and familial amyloidotic polyneuropathy. Intern Med. 1993;32:920–922.
   PubMed Web of Science ®Google Scholar
• 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;68:161–172.
   PubMed Web of Science ®Google Scholar