637
Views
14
CrossRef citations to date
0
Altmetric
Review

Progress in the treatment of small fiber peripheral neuropathy

, , , &

References

  • Devigili G, Tugnoli V, Penza P, et al. The diagnostic criteria for small fibre neuropathy: from symptoms to neuropathology. Brain 2008;131:1912-25
  • Brown MJ, Martin JR, Asbury AK. Painful diabetic neuropathy: a morphometric study. Arch Neurol 1976;33:164-71
  • Dyck P, Lambert EH. Dissociated sensation in amyloidosis: compound action potential, quantitative histologic and teased-fiber, and electron microscopic studies of sural nerve biopsies. Arch Neurol 1969;20:490-507
  • Koike H, Iijima M, Sugiura M, et al. Alcoholic neuropathy is clinicopathologically distinct from thiamine-deficiency neuropathy. Ann Neurol 2003;54:19-29
  • Mori K, Iijima M, Koike H, et al. The wide spectrum of clinical manifestations in Sjogren’s syndrome-associated neuropathy. Brain 2005;128:2518-34
  • Oki Y, Koike H, Iijima M, et al. Ataxic vs painful form of paraneoplastic neuropathy. Neurology 2007;69:564-72
  • Ohnishi A, Dyck P. Loss of small peripheral sensory neurons in Fabry disease: histologic and morphometric evaluation of cutaneous nerves, spinal ganglia, and posterior columns. Arch Neurol 1974;31:120-7
  • Lauria G, Merkies IS, Faber CG. Small fibre neuropathy. Curr Opin Neurol 2012;25:542-9
  • Tavakoli M, Petropoulos IN, Malik RA. Corneal confocal microscopy to assess diabetic neuropathy: an eye on the foot. J Diabetes Sci Technol 2013;7:1179-89
  • Papanas N, Ziegler D. Corneal confocal microscopy: a new technique for early detection of diabetic neuropathy. Curr Diab Rep 2013;13:488-99
  • Tavakoli M, Marshall A, Thompson L, et al. Corneal confocal microscopy: a novel noninvasive means to diagnose neuropathy in patients with Fabry disease. Muscle Nerve 2009;40:976-84
  • Nebuchennykh M, Loseth S, Lindal S, Mellgren S. The value of skin biopsy with recording of intraepidermal nerve fiber density and quantitative sensory testing in the assessment of small fiber involvement in patients with different causes of polyneuropathy. J Neurol 2009;256(7):1067-75
  • Maier C, Baron R, Tolle TR, et al. Quantitative sensory testing in the German Research Network on Neuropathic Pain (DFNS): somatosensory abnormalities in 1236 patients with different neuropathic pain syndromes. Pain 2010;150:439-50
  • Freeman R, Baron R, Bouhassira D, et al. Sensory profiles of patients with neuropathic pain based on the neuropathic pain symptoms and signs. Pain 2014;155:367-76
  • Casanova-Molla J, Grau-Junyent JM, Morales M, Valls-Sole J. On the relationship between nociceptive evoked potentials and intraepidermal nerve fiber density in painful sensory polyneuropathies. Pain 2011;152:410-18
  • Obermann M, Katsarava Z, Esser S, et al. Correlation of epidermal nerve fiber density with pain-related evoked potentials in HIV neuropathy. Pain 2008;138:79-86
  • Chao CC, Tseng MT, Lin YJ, et al. Pathophysiology of neuropathic pain in type 2 diabetes: skin denervation and contact heat evoked potentials. Diabetes Care 2010;33:2654-9
  • Chao CC, Hsieh SC, Tseng MT, et al. Patterns of contact heat evoked potential (CHEP) in neuropathy with skin denervation: correlation of CHEP amplitude with intraepidermal nerve fiber density. Clin Neurophysiol 2008;119:653-61
  • Chao CC, Wu VC, Tan CH, et al. Skin denervation and its clinical significance in late-stage chronic kidney disease. Arch Neurol 2011;68:200-6
  • Lin YH, Hsieh SC, Chao CC, et al. Influence of aging on thermal and vibratory thresholds of quantitative sensory testing. J Peripher Nerv Syst 2005;10:269-81
  • Lauria G, Hsieh ST, Johansson O, et al. European Federation of Neurological Societies/Peripheral Nerve Society Guideline on the use of skin biopsy in the diagnosis of small fiber neuropathy. Report of a joint task force of the European Federation of Neurological Societies and the Peripheral Nerve Society. Eur J Neurol 2010;17:903-12
  • England JD, Gronseth GS, Franklin G, et al. Practice parameter: evaluation of distal symmetric polyneuropathy: role of autonomic testing, nerve biopsy, and skin biopsy (an evidence-based review): report of the American Academy of Neurology, American Association of Neuromuscular and Electrodiagnostic Medicine, and American Academy of Physical Medicine and Rehabilitation. Neurology 2009;72:177-84
  • Lauria G, Lombardi R, Camozzi F, Devigili G. Skin biopsy for the diagnosis of peripheral neuropathy. Histopathology 2009;54:273-85
  • Wang N, Gibbons CH. Skin biopsies in the assessment of the autonomic nervous system. Handb Clin Neurol 2013;117:371-8
  • Herrmann DN, Griffin JW, Hauer P, et al. Epidermal nerve fiber density and sural nerve morphometry in peripheral neuropathies. Neurology 1999;53:1634-40
  • Scott LJC, Griffin JW, Luciano C, et al. Quantitative analysis of epidermal innervation in Fabry disease. Neurology 1999;52:1249
  • Holland NR, Crawford TO, Hauer P, et al. Small-fiber sensory neuropathies: clinical course and neuropathology of idiopathic cases. Ann Neurol 1998;44:47-59
  • Lauria G, Morbin M, Lombardi R, et al. Axonal swellings predict the degeneration of epidermal nerve fibers in painful neuropathies. Neurology 2003;61:631-6
  • Hsieh ST. Toxic neuropathies. In: Encyclopedia of Pain. Schmidt RF, Willis WD, Eds) Springer-Verlag; Berlin, Germany: 2013
  • Luo KR, Chao CC, Hsieh PC, Hsieh ST. Effect of glycemic control on sudomotor denervation in type 2 diabetes. Diabetes Care 2012;35:612-16
  • Luo KR, Chao CC, Chen YT, et al. Quantitation of sudomotor innervation on skin biopsies in diabetic neuropathy. J Neuropathol Exp Neurol 2011;70:930-8
  • Gibbons CH, Illigens BMW, Wang N, Freeman R. Quantification of sudomotor innervation: a comparison of three methods. Muscle Nerve 2010;42:112-19
  • Gibbons CH, Illigens BMW, Wang N, Freeman R. Quantification of sweat gland innervation: a clinical-pathologic correlation. Neurology 2009;72:1479-86
  • Nolano M, Provitera V, Caporaso G, et al. Quantification of pilomotor nerves: a new tool to evaluate autonomic involvement in diabetes. Neurology 2010;75:1089-97
  • Shun CT, Chang YC, Wu HP, et al. Skin denervation in type 2 diabetes: correlations with diabetic duration and functional impairments. Brain 2004;127:1593-605
  • Boucek P, Havrdova T, Voska L, et al. Epidermal innervation in type 1 diabetic patients: a 2.5-year prospective study after simultaneous pancreas/kidney transplantation. Diabetes Care 2008;31:1611-12
  • Uceyler N, Devigili G, Toyka K, Sommer C. Skin biopsy as an additional diagnostic tool in non-systemic vasculitic neuropathy. Acta Neuropathol 2010;120:109-16
  • Lee JE, Shun CT, Hsieh SC, Hsieh ST. Skin denervation in vasculitic neuropathy. Arch Neurol 2005;62:1570-3
  • Chao CC, Hsieh ST, Shun CT, Hsieh SC. Skin denervation and cutaneous vasculitis in eosinophilia-associated neuropathy. Arch Neurol 2007;64:959-65
  • Tseng MT, Hsieh SC, Shun CT, et al. Skin denervation and cutaneous vasculitis in systemic lupus erythematosus. Brain 2006;129:977-85
  • Goransson LG, Tjensvoll AB, Herigstad A, et al. Small-diameter nerve fiber neuropathy in systemic lupus erythematosus. Arch Neurol 2006;63:401-4
  • Chao CC, Sun HY, Chang YC, Hsieh ST. Painful neuropathy with skin denervation after prolonged use of linezolid. J Neurol Neurosurg Psychiatry 2008;79:97-9
  • Tan CH, Chen YF, Chen CC, et al. Painful neuropathy due to skin denervation after metronidazole-induced neurotoxicity. J Neurol Neurosurg Psychiatry 2011;82:462-5
  • Koike H, Tanaka F, Hashimoto R, 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
  • Kim DH, Zeldenrust SR, Low PA, Dyck PJ. Quantitative sensation and autonomic test abnormalities in transthyretin amyloidosis polyneuropathy. Muscle Nerve 2009;40:363-70
  • Wang AK, Fealey RD, Gehrking TL, Low PA. Patterns of neuropathy and autonomic failure in patients with amyloidosis. Mayo Clin Proc 2008;83:1226-30
  • Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch Neurol 2005;62:1057-62
  • Waxman SG. Painful Na-channelopathies: an expanding universe. Trends Mol Med 2013;19:406-9
  • Faber CG, Hoeijmakers JGJ, Ahn HS, et al. Gain of function NaV1.7 mutations in idiopathic small fiber neuropathy. Ann Neurol 2012;71:26-39
  • Gorson KC, Herrmann DN, Thiagarajan R, et al. Non-length dependent small fibre neuropathy/ganglionopathy. J Neurol Neurosurg Psychiatry 2008;79:163-9
  • Samuelsson K, Kostulas K, Vrethem M, et al. Idiopathic small fiber neuropathy: phenotype, etiologies, and the search for Fabry disease. J Clin Neurol 2014;10:108-18
  • Dyck PJ, Herrmann DN, Staff NP, Dyck PJ. Assessing decreased sensation and increased sensory phenomena in diabetic polyneuropathies. Diabetes 2013;62:3677-86
  • Spallone V, Morganti R, D’Amato C, et al. Clinical correlates of painful diabetic neuropathy and relationship of neuropathic pain with sensorimotor and autonomic nerve function. Eur J Pain 2011;15:153-60
  • Bloomgarden ZT. Diabetic neuropathy. Diabetes Care 2008;31:616-21
  • Brock C, Softeland E, Gunterberg V, et al. Diabetic autonomic neuropathy affects symptom generation and brain-gut axis. Diabetes Care 2013;36:3698-705
  • Schonauer M, Thomas A, Morbach S, et al. Cardiac autonomic diabetic neuropathy. Diab Vasc Dis Res 2008;5:336-44
  • Vinik AI, Ziegler D. Diabetic cardiovascular autonomic neuropathy. Circulation 2007;115:387-97
  • Astrup AS, Tarnow L, Rossing P, et al. Cardiac autonomic neuropathy predicts cardiovascular morbidity and mortality in type 1 diabetic patients with diabetic nephropathy. Diabetes Care 2006;29:334-9
  • Vinik AI, Maser RE, Mitchell BD, Freeman R. Diabetic autonomic neuropathy. Diabetes Care 2003;26:1553-79
  • Clauw DJ. Fibromyalgia: a clinical review. JAMA 2014;311:1547-55
  • Gierthmuhlen J, Binder A, Baron R. Mechanism-based treatment in complex regional pain syndromes. Nat Rev Neurol 2014;10:518-28
  • Lutz J, Jager L, de Quervain D, et al. White and gray matter abnormalities in the brain of patients with fibromyalgia: a diffusion-tensor and volumetric imaging study. Arthritis Rheum 2008;58:3960-9
  • Oaklander AL, Herzog ZD, Downs HM, Klein MM. Objective evidence that small-fiber polyneuropathy underlies some illnesses currently labeled as fibromyalgia. Pain 2013;154:2310-16
  • Uceyler N, Zeller D, Kahn AK, et al. Small fibre pathology in patients with fibromyalgia syndrome. Brain 2013;136:1857-67
  • Caro XJ, Winter EF. Evidence of abnormal epidermal nerve fiber density in fibromyalgia: clinical and immunologic implications. Arthritis Rheumatol 2014;66:1945-54
  • Marinus J, Moseley GL, Birklein F, et al. Clinical features and pathophysiology of complex regional pain syndrome. Lancet Neurol 2011;10:637-48
  • Bruehl S. An update on the pathophysiology of complex regional pain syndrome. Anesthesiology 2010;113:713-25
  • Parkitny L, McAuley JH, Di Pietro F, et al. Inflammation in complex regional pain syndrome: a systematic review and meta-analysis. Neurology 2013;80:106-17
  • Lenz M, Hoffken O, Stude P, et al. Bilateral somatosensory cortex disinhibition in complex regional pain syndrome type I. Neurology 2011;77:1096-101
  • Albrecht PJ, Hines S, Eisenberg E, et al. Pathologic alterations of cutaneous innervation and vasculature in affected limbs from patients with complex regional pain syndrome. Pain 2006;120:244-66
  • Oaklander AL, Rissmiller JG, Gelman LB, et al. Evidence of focal small-fiber axonal degeneration in complex regional pain syndrome-I (reflex sympathetic dystrophy). Pain 2006;120:235-43
  • Oaklander AL, Fields HL. Is reflex sympathetic dystrophy/complex regional pain syndrome type I a small-fiber neuropathy? Ann Neurol 2009;65:629-38
  • Birklein F, O’Neill D, Schlereth T. Complex regional pain syndrome: an optimistic perspective. Neurology 2015;84:89-96
  • Persson AK, Kim I, Zhao P, et al. Sodium channels contribute to degeneration of dorsal root ganglion neurites induced by mitochondrial dysfunction in an in vitro model of axonal injury. J Neurosci 2013;33:19250-61
  • Koike H, Iijima M, Mori K, et al. Neuropathic pain correlates with myelinated fibre loss and cytokine profile in POEMS syndrome. J Neurol Neurosurg Psychiatry 2008;79:1171-9
  • Teixeira F, Aranda F, Cabanas A, Estanol B. POEMS syndrome: ultrastructural observations on the sural nerve. Ultrastruct Pathol 1990;14:31-40
  • Uceyler N, Kafke W, Riediger N, et al. Elevated proinflammatory cytokine expression in affected skin in small fiber neuropathy. Neurology 2010;74:1806-13
  • Woolf CJ. Central sensitization: implications for the diagnosis and treatment of pain. Pain 2011;152:S2-S15
  • Latremoliere A, Woolf CJ. Central sensitization: a generator of pain hypersensitivity by central neural plasticity. J Pain 2009;10:895-926
  • Tseng MT, Chiang MC, Chao CC, et al. fMRI evidence of degeneration-induced neuropathic pain in diabetes: enhanced limbic and striatal activations. Hum Brain Mapp 2013;34:2733-46
  • Spallone V. Management of painful diabetic neuropathy: guideline guidance or jungle? Curr Diab Rep 2012;12:403-13
  • Bril V, England J, Franklin GM, et al. Evidence-based guideline: treatment of painful diabetic neuropathy: Report of the American Academy of Neurology, the American Association of Neuromuscular and Electrodiagnostic Medicine, and the American Academy of Physical Medicine and Rehabilitation. Neurology 2011;76:1758-65
  • American Society of Anesthesiologists. Practice guidelines for chronic pain management: an updated report by the American Society of Anesthesiologists Task Force on Chronic Pain Management and the American Society of Regional Anesthesia and Pain Medicine. Anesthesiology 2010;112:810-33
  • Attal N, Cruccu G, Baron R, et al. EFNS guidelines on the pharmacological treatment of neuropathic pain: 2010 revision. Eur J Neurol 2010;17:1113-23
  • O’Connor AB, Dworkin RH. Treatment of neuropathic pain: an overview of recent guidelines. Am J Med 2009;122:S22-32
  • Moore A, Wiffen P, Kalso E. Antiepileptic drugs for neuropathic pain and fibromyalgia. JAMA 2014;312:182-3
  • Moore RA, Wiffen PJ, Derry S, et al. Gabapentin for chronic neuropathic pain and fibromyalgia in adults. Cochrane Database Syst Rev 2014;4:CD007938
  • Wiffen PJ, Derry S, Moore RA, Kalso EA. Carbamazepine for chronic neuropathic pain and fibromyalgia in adults. Cochrane Database Syst Rev 2014;4:CD005451
  • Desai B, Freeman E, Huang E, et al. Clinical value of tapentadol extended-release in painful diabetic peripheral neuropathy. Expert Rev Clin Pharmacol 2014;7:203-9
  • Games G, Hutchison A. Tapentadol-ER for the treatment of diabetic peripheral neuropathy. Consult Pharm 2013;28:672-5
  • Derry S, Sven-Rice A, Cole P, et al. Topical capsaicin (high concentration) for chronic neuropathic pain in adults. Cochrane Database Syst Rev 2013;2:CD007393
  • Simpson DM, Brown S, Tobias J; For the NGX-4010 C107 Study Group. Controlled trial of high-concentration capsaicin patch for treatment of painful HIV neuropathy. Neurology 2008;70:2305-13
  • Brederson JD, Kym PR, Szallasi A. Targeting TRP channels for pain relief. Eur J Pharmacol 2013;716:61-76
  • Campbell CM, Kipnes MS, Stouch BC, et al. Randomized control trial of topical clonidine for treatment of painful diabetic neuropathy. Pain 2012;153:1815-23
  • Anand P, Bley K. Topical capsaicin for pain management: therapeutic potential and mechanisms of action of the new high-concentration capsaicin 8% patch. Br J Anaesth 2011;107:490-502
  • Kawagashira Y, Watanabe H, Oki Y, et al. Intravenous immunoglobulin therapy markedly ameliorates muscle weakness and severe pain in proximal diabetic neuropathy. J Neurol Neurosurg Psychiatry 2007;78:899-901
  • Morozumi S, Kawagashira Y, Iijima M, et al. Intravenous immunoglobulin treatment for painful sensory neuropathy associated with Sjogren’s syndrome. J Neurol Sci 2009;279:57-61
  • Goebel A, Baranowski A, Maurer K, et al. Intravenous immunoglobulin treatment of the complex regional pain syndrome: a randomized trial. Ann Intern Med 2010;152:152-8
  • Miyazaki Y, Koike H, Akane A, et al. Spinal cord stimulation markedly ameliorated refractory neuropathic pain in transthyretin Val30Met familial amyloid polyneuropathy. Amyloid 2011;18:87-90
  • Dib-Hajj SD, Waxman SG. Translational pain research: lessons from genetics and genomics. Sci Transl Med 2014;6:249sr4
  • Zhang XY, Wen J, Yang W, et al. Gain-of-function mutations in SCN11A cause familial episodic pain. Am J Hum Genet 2013;93:957-66
  • Kremeyer B, Lopera F, Cox JJ, et al. A gain-of-function mutation in TRPA1 causes familial episodic pain syndrome. Neuron 2010;66:671-80
  • Klein CJ, Lennon VA, Aston PA, et al. Chronic pain as a manifestation of potassium channel-complex autoimmunity. Neurology 2012;79:1136-44
  • Costigan M, Belfer I, Griffin RS, et al. Multiple chronic pain states are associated with a common amino acid changing allele in KCNS1. Brain 2010;133:2519-27
  • Bruehl S, Denton JS, Lonergan D, et al. Associations between KCNJ6 (GIRK2) gene polymorphisms and pain-related phenotypes. Pain 2013;154(12):2853-9
  • Benarroch EE. The clinical approach to autonomic failure in neurological disorders. Nat Rev Neurol 2014;10:396-407
  • Freeman R. Autonomic peripheral neuropathy. Neurol Clin 2007;25:277-301
  • Koike H, Watanabe H, Sobue G. The spectrum of immune-mediated autonomic neuropathies: insights from the clinicopathological features. J Neurol Neurosurg Psychiatry 2013;84:98-106
  • Muppidi S, Vernino S. Autoimmune autonomic failure. Handb Clin Neurol 2013;117:321-7
  • Yasuda T, Sobue G, Doyu M, et al. Familial amyloidotic polyneuropathy with late-onset and well-preserved autonomic function: a Japanese kindred with novel mutant transthyretin (Ala97 to Gly). J Neurol Sci 1994;121:97-102
  • Low PA, Singer W. Management of neurogenic orthostatic hypotension: an update. Lancet Neurol 2008;7:451-8
  • Singer W, Joyner MJ, Sandroni P, et al. Midodrine efficacy in orthostatic hypotension. J Gen Intern Med 2014;29:1440-1
  • Singer W, Sandroni P, Opfer-Gehrking TL. Pyridostigmine treatment trial in neurogenic orthostatic hypotension. Arch Neurol 2006;63:513-18
  • Goldstein DS. L-Dihydroxyphenylserine (L-DOPS): a norepinephrine prodrug. Cardiovasc Drug Rev 2006;24:189-203
  • Kaufmann H, Freeman R, Biaggioni I, et al. Droxidopa for neurogenic orthostatic hypotension: a randomized, placebo-controlled, phase 3 trial. Neurology 2014;83:328-35
  • Shibao C, Okamoto L, Biaggioni I. Pharmacotherapy of autonomic failure. Pharmacol Ther 2012;134:279-86
  • Logan IC, Witham MD. Efficacy of treatments for orthostatic hypotension: a systematic review. Age Ageing 2012;41:587-94
  • Unger CA, Tunitsky-Bitton E, Muffly T, Barber MD. Neuroanatomy, neurophysiology, and dysfunction of the female lower urinary tract: a review. Female Pelvic Med Reconstr Surg 2014;20:65-75
  • Fullhase C, Soler R, Gratzke C. New strategies in treating male lower urinary tract symptoms. Curr Opin Urol 2014;24:29-35
  • Mangera A, Apostolidis A, Andersson KE, et al. An updated systematic review and statistical comparison of standardised mean outcomes for the use of botulinum toxin in the management of lower urinary tract disorders. Eur Urol 2014;65:981-90
  • Camilleri M. Pharmacological agents currently in clinical trials for disorders in neurogastroenterology. J Clin Invest 2013;123:4111-20
  • Hasler WL. Emerging drugs for the treatment of gastroparesis. Expert Opin Emerg Drugs 2014;19:261-79
  • Janssen P, Scott Harris M, Jones M, et al. The relation between symptom improvement and gastric emptying in the treatment of diabetic and idiopathic gastroparesis. Am J Gastroenterol 2013;108:1382-91
  • Camilleri M. Novel therapeutic agents in neurogastroenterology: advances in the past year. Neurogastroenterol Motil 2014;26:1070-8
  • Toyooka K. Fabry disease. Handb Clin Neurol 2013;115:629-42
  • Uceyler N, Kahn AK, Kramer D, et al. Impaired small fiber conduction in patients with Fabry disease: a neurophysiological case-control study. BMC Neurol 2013;13:47
  • El Dib RP, Nascimento P, Pastores GM. Enzyme replacement therapy for Anderson-Fabry disease. Cochrane Database Syst Rev 2013;2:CD006663
  • Rombach S, Smid B, Linthorst G, et al. Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: a systematic review and meta-analysis. J Inherit Metab Dis 2014;37:341-52
  • Plante-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol 2011;10:1086-97
  • Yang NC, Lee MJ, Chao CC, et al. Clinical presentations and skin denervation in amyloid neuropathy due to transthyretin Ala97Ser. Neurology 2010;75:532-8
  • Koike H, Misu K, Ikeda S, et al. Type I (transthyretin Met30) familial amyloid polyneuropathy in Japan: early- vs late-onset form. Arch Neurol 2002;59:1771-6
  • Ando Y, Coelho T, Berk J, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 2013;8:31
  • Sekijima Y. Recent progress in the understanding and treatment of transthyretin amyloidosis. J Clin Pharm Ther 2014;39:225-33
  • Kolstoe SE, Wood SP. Drug targets for amyloidosis. Biochem Soc Trans 2010;38:466-70
  • Lindgren M, Hammarstrom P. Amyloid oligomers: spectroscopic characterization of amyloidogenic protein states. FEBS J 2010;277:1380-8
  • Hund E, Linke RP, Willig F, Grau A. Transthyretin-associated neuropathic amyloidosis: pathogenesis and treatment. Neurology 2001;56:431-5
  • Yamashita T, Ando Y, Okamoto S, et al. Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology 2012;78:637-43
  • Okamoto S, Wixner J, Obayashi K, et al. Liver transplantation for familial amyloidotic polyneuropathy: impact on Swedish patients’ survival. Liver Transpl 2009;15:1229-35
  • Yamamoto S, Wilczek HE, Nowak G, et al. Liver transplantation for familial amyloidotic polyneuropathy (FAP): a single-center experience over 16 years. Am J Transplant 2007;7:2597-604
  • Stangou AJ, Hawkins PN. Liver transplantation in transthyretin-related familial amyloid polyneuropathy. Curr Opin Neurol 2004;17:615-20
  • Adams D, Samuel D, Goulon-Goeau C, et al. The course and prognostic factors of familial amyloid polyneuropathy after liver transplantation. Brain 2000;123:1495-504
  • Obici L, Merlini G. An overview of drugs currently under investigation for the treatment of transthyretin-related hereditary amyloidosis. Expert Opin Investig Drugs 2014;23:1239-51
  • Lachmann HJ. A new era in the treatment of amyloidosis? N Engl J Med 2013;369:866-8
  • Coelho T, Maia LF, da Silva AM, et al. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol 2013;260:2802-14
  • Lozeron P, Theaudin M, Mincheva Z, et al. Effect on disability and safety of tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy. Eur J Neurol 2013;20:1539-45
  • Coelho T, Maia LF, Martins da SA, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology 2012;79:785-92
  • Berk JL, Suhr OB, Obici L. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA 2013;310:2658-67
  • Connelly S, Choi S, Johnson SM, et al. Structure-based design of kinetic stabilizers that ameliorate the transthyretin amyloidoses. Curr Opin Struct Biol 2010;20:54-62
  • Reixach N, Deechongkit S, Jiang X, et al. Tissue damage in the amyloidoses: transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture. Proc Natl Acad Sci USA 2004;101:2817-22
  • Boudreau RL, Davidson BL. Generation of hairpin-based RNAi vectors for biological and therapeutic application. Methods Enzymol 2012;507:275-96
  • Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 2013;369:819-29

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.