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Amyloid
The Journal of Protein Folding Disorders
Volume 23, 2016 - Issue 1
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Original Article

Long-term outcome of patients with hereditary transthyretin V30M amyloidosis with polyneuropathy after liver transplantation

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Pages 39-45 | Received 28 Jul 2015, Accepted 18 Nov 2015, Published online: 13 Jan 2016

References

  • Andrade C. A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves. Brain 1952;75:408–27
  • Benson MD, Dwulet FE. Identification of carriers of a variant plasma prealbumin (transthyretin) associated with familial amyloidotic polyneuropathy type I. J Clin Invest 1985;75:71–75
  • Planté-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol 2011;10:1086–97
  • Ihse E, Rapezzi C, Merlini G, Benson MD, Ando Y, Suhr OB, Ikeda S, et al. Amyloid fibrils containing fragmented ATTR may be the standard fibril composition in ATTR amyloidosis. Amyloid 2013;20:142–50
  • Sipe JD, Benson MD, Buxbaum JN, Ikeda S, Merlini G, Saraiva MJ, Westermark P. Nomenclature 2014: amyloid fibril proteins and clinical classification of the amyloidosis. Amyloid 2014;21:221–4
  • Beirão JM, Malheiro J, Lemos C, Beirão I, Costa P, Torres P. Ophthalmological manifestations in hereditary transthyretin (ATTR V30M) carriers: a review of 513 cases. Amyloid 2015;22:117–22
  • Kristen AV, Aus dem Siepen F, Scherer K, Kammerer R, Andre F, Buss SJ, Bauer R, et al. Comparison of different types of cardiac amyloidosis by cardiac magnetic resonance imaging. Amyloid 2015;22:132–41
  • Holmgren G, Steen L, Ekstedt J, Groth CG, Ericzon BG, Eriksson S, Andersen O, et al. Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30). Clin Genet 1991;40:242–6
  • Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch Neurol 2005;62:1057–62
  • Yamamoto S, Wilczek HE, Nowak G, Larsson M, Oksanen A, Iwata T, Gjertsen H, et al. Liver transplantation for familial amyloidotic polyneuropathy (FAP): a single-center experience over 16 years. Am J Transplant 2007;7:2597–604
  • Okamoto S, Wixner J, Obayashi K, Ando Y, Ericzon BG, Friman S, Uchino M, et al. Liver transplantation for familial amyloidotic polyneuropathy: impact on Swedish patients’ survival. Liver Transpl 2009;15:1229–35
  • Yamashita T, Ando Y, Okamoto S, Misumi Y, Hirahara T, Ueda M, Obayashi K, et al. Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology 2012;78:637–43
  • Holmgren G, Ericzon BG, Groth CG, Steen L, Suhr O, Andersen O, Wallin BG, et al. Clinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosis. Lancet 1993;341:1113–16
  • Adams D, Samuel D, Goulon-Goeau C, Nakazato M, Costa PM, Feray C, Planté V, et al. The course and prognostic factors of familial amyloid polyneuropathy after liver transplantation. Brain 2000;123:1495–504
  • Ericzon BG, Wilczek HE, Larsson M, Wijayatunga P, Stangou A, Pena JR, Furtado E, et al. Liver transplantation for hereditary transthyretin amyloidosis: after 20 years still the best therapeutic alternative? Transplantation 2015;99:1847–54
  • Sakashita N, Ando Y, Haraoka K, Terazaki H, Yamashita T, Nakamura M, Takeya M. Severe congestive heart failure with cardiac liver cirrhosis 10 years after orthotopic liver transplantation for familial amyloidotic polyneuropathy. Pathol Int 2006;56:408–12
  • Yazaki M, Mitsuhashi S, Tokuda T, Kametani F, Takei YI, Koyama J, Kawamorita A, et al. Progressive wild-type transthyretin deposition after liver transplantation preferentially occurs onto myocardium in FAP patients. Am J Transplant 2007;7:235–42
  • Yamashita T, Ando Y, Bernt Suhr O, Nakamura M, Sakashita N, Ohlsson PI, Terazaki H, et al. A new diagnostic procedure to detect unknown transthyretin (TTR) mutations in familial amyloidotic polyneuropathy (FAP). J Neurol Sci 2000;173:154–9
  • Yamashita T, Ando Y, Katsuragi S, Nakamura M, Obayashi K, Haraoka K, Ueda M, et al. Muscular amyloid angiopathy with amyloidogenic transthyretin Ser50Ile and Tyr114Cys. Muscle Nerve 2005;31:41–5
  • Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve 2007;36:411–23
  • Russo M, Mazzeo A, Stancanelli C, Di Leo R, Gentile L, Di Bella G, Minutoli F, et al. Transthyretin-related familial amyloidotic polyneuropathy: description of a cohort of patients with Leu64 mutation and late onset. J Peripher Nerv Syst 2012;17:385–90
  • Tashima K, Ando Y, Terazaki H, Yoshimatsu S, Suhr OB, Obayashi K, Yamashita T, et al. Outcome of liver transplantation for transthyretin amyloidosis: follow-up of Japanese familial amyloidotic polyneuropathy patients. J Neurol Sci 1999;171:19–23
  • Ohya Y, Okamoto S, Tasaki M, Ueda M, Jono H, Obayashi K, Takeda K, et al. Manifestations of transthyretin-related familial amyloidotic polyneuropathy: long-term follow-up of Japanese patients after liver transplantation. Surg Today 2011;41:1211–18
  • Koike H, Tanaka F, Hashimoto R, Tomita M, Kawagashira Y, Iijima M, Fujitake J, et al. Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas. J Neurol Neurosurg Psychiatry 2012;83:152–8
  • Ando Y, Yonehara T, Tanaka Y, Tashima K, Uchino M, Ando M. Early pacemaker implantation in patients with familial amyloidotic polyneuropathy. Muscle Nerve 1996;19:1640–1
  • Okamoto S, Yamashita T, Ando Y, Ueda M, Misumi Y, Obayashi K, Horibata Y, et al. Evaluation of myocardial changes in familial amyloid polyneuropathy after liver transplantation. Intern Med 2008;47:2133–7
  • Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology 2010;75:324–7
  • Gustafsson S, Ihse E, Henein MY, Westermark P, Lindqvist P, Suhr OB. Amyloid fibril composition as a predictor of development of cardiomyopathy after liver transplantation for hereditary transthyretin amyloidosis. Transplantation 2012;93:1017–23
  • Berk JL, Suhr OB, Obici L, Sekijima Y, Zeldenrust SR, Yamashita T, Heneghan MA, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA 2013;310:2658–67
  • Vinik EJ, Vinik AI, Paulson JF, Merkies IS, Packman J, Grogan DR, Coelho T. Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst 2014;19:104–14
  • Cruz MW. Regional differences and similarities of familial amyloidotic polyneuropathy (FAP) presentation in Brazil. Amyloid 2012;19:65–7
  • Maia LF, Magalhães R, Freitas J, Taipa R, Pires MM, Osório H, Dias D, et al. CNS involvement in V30M transthyretin amyloidosis: clinical, neuropathological and biochemical findings. J Neurol Neurosurg Psychiatry 2015;86:159–67
  • Yamashita T, Ando Y, Ueda M, Nakamura M, Okamoto S, Zeledon ME, Hirahara T, et al. Effect of liver transplantation on transthyretin Tyr114Cys-related cerebral amyloid angiopathy. Neurology 2008;70:123–8
  • Suhr OB, Friman S, Ericzon BG. Early liver transplantation improves familial amyloidotic polyneuropathy patients’ survival. Amyloid 2005;12:233–8
  • Obayashi K, Ueda M, Oshima T, Kawahara S, Misumi Y, Yamashita T, Jono H, et al. Pathological changes long after liver transplantation in a familial amyloidotic polyneuropathy patient. BMJ Case Rep 2012;2012:piibcr2012006593. doi: 10.1136/bcr-2012-006593
  • Wilczek HE, Larsson M, Ericzon BG, FAPWTR. Long-term data from the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR). Amyloid 2011;18:193–5
  • Olanow CW, Hauser RA, Jankovic J, Langston W, Lang A, Poewe W, Tolosa E, et al. A randomized, double-blind, placebo-controlled, delayed start study to assess rasagiline as a disease modifying therapy in Parkinson’s disease (the ADAGIO study): rationale, design, and baseline characteristics. Mov Disord 2008;23:2194–201
  • Ando Y, Ueda M. Diagnosis and therapeutic approaches to transthyretin amyloidosis. Curr Med Chem 2012;19:2312–23
  • Coelho T, Maia LF, Martins da Silva A, Waddington Cruz M, Planté-Bordeneuve V, Lozeron P, Suhr OB, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology 2012;79:785–92
  • Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 2013;369:819–29

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