A selective transthyretin-adsorption column for the treatment of patients with familial amyloid polyneuropathy

Amyloid. 1998 Jun;5(2):111-6. doi: 10.3109/13506129808995288.

Abstract

A transthyretin (TTR)-adsorption column has been developed for the removal of variant TTR from the plasma of patients with familial amyloid polyneuropathy (FAP). The adsorbent is an ion-exchange resin made of porous beads of polyvinyl alcohol gel covalently bound with dimethylaminoethanol. This column was used for three patients with type I FAP. It reduced the concentrations of both normal and variant TTR in the plasma to about half of their pre-adsorption levels. Except for thyroxine, retinol-binding protein and IgM, other proteins in serum were not significantly decreased and there were no adverse effects in long term clinical usage of this TTR-adsorption column. In this trial, we did not obtain concrete evidence that TTR-adsorption therapy can stop or delay the progression of the disease in a FAP patient. However, if we are able to apply this technique more frequently and effectively, TTR-adsorption therapy using our column might be useful for the treatment of FAP patients.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adsorption
  • Adult
  • Amino Acid Sequence
  • Amyloid Neuropathies / genetics
  • Amyloid Neuropathies / therapy*
  • Animals
  • Chromatography, Ion Exchange
  • Dogs
  • Female
  • Humans
  • Male
  • Middle Aged
  • Molecular Sequence Data
  • Prealbumin / isolation & purification*

Substances

  • Prealbumin