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[Familial amyloidotic polyneuropathies]

Bull Acad Natl Med. 2012 Oct;196(7):1321-9; discussion 1329-31.
[Article in French]

Abstract

Transthyretin familial amyloid polyneuropathy (TTRFAP) is an autosomal dominant neuropathy that is fatal within about 10 years after symptom onset. TTRFAP is observed worldwide, albeit with a higher frequency of the most common variant, Val30met, in Portugal, Sweden and Japan. Various phenotypic differences are observed. TTRFAP should be considered in patients with a progressive axonal polyneuropathy of unknown origin, especially when associated with autonomic nervous system dysfunction. A positive family history is found in most cases when onset begins around 30 years of age, while late-onset FAP is often sporadic and may be confused with chronic inflammatory demyelinating polyneuropathy. Nerve biopsy is often used to confirm the presence of extracellular amyloid deposits in interstitial tissue of the endoneurial space, although amyloid can also befound in muscle, salivary gland and abdominal fat. It is important to stress that biopsy negativity does not rule out amyloidosis. Genetic testing for TTR gene mutations should be performed in case of progressive length-dependent axonal polyneuropathy predominantly involving small nerve fibers.

Publication types

  • English Abstract
  • Review

MeSH terms

  • Adult
  • Age of Onset
  • Americas / epidemiology
  • Amyloid / ultrastructure
  • Amyloid Neuropathies, Familial / diagnosis
  • Amyloid Neuropathies, Familial / epidemiology
  • Amyloid Neuropathies, Familial / genetics*
  • Apolipoprotein A-I / genetics
  • Biopsy
  • Electromyography
  • Europe / epidemiology
  • Gelsolin / genetics
  • Genes, Dominant
  • Genetic Heterogeneity
  • Humans
  • Japan / epidemiology
  • Microscopy, Electron
  • Mutation
  • Peripheral Nerves / pathology
  • Prealbumin / genetics
  • Prealbumin / physiology

Substances

  • Amyloid
  • Apolipoprotein A-I
  • Gelsolin
  • Prealbumin

Supplementary concepts

  • Amyloidosis, Hereditary, Transthyretin-Related