A SELECTIVE TRANSTHYRETIN-ADSORPTION COLUMN FOR THE TREATMENT OF PATIENTS WITH FAMILIAL AMYLOID POLYNEUROPATHY

A transthyretin (TTR)-adsorption column has been developed for the rem oval of variant TTR from the plasma of patients with familial amyloid polyneuropathy (FAP). The adsorbent is an ion-exchange resin made of p orous beads of polyvinyl alcohol gel covalently bound with dimethylami noethanol. This column was used for three patients with type 1 FAP. It reduced the concentrations of both normal and variant TTR in the plas ma to about half of their pre-adsorption levels. Except for thyroxine, retinol-binding protein and IgM; other proteins in serum were not sig nificantly decreased and there were no adverse effects in long term cl inical 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 a ble to apply this technique more frequently and effectively, TTR-adsor ption therapy using our column might be useful for the treatment of FA P patients.