A selective transthyretin-adsorption column for the treatment of patients with familial amyloid polyneuropathy.
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.
Tokuda
T
T
Department of Medicine (Neurology), Shinshu University School of Medicine, Matsumoto, Japan.
Kondo
T
T
Hanaoka
N
N
Takei
Y
Y
Kametani
F
F
Yamada
Y
Y
Tamura
Y
Y
Yoshida
M
M
Yamawaki
N
N
Ikeda
S
S
D000327:Adsorption; D000328:Adult; D000595:Amino Acid Sequence; D017772:Amyloid Neuropathies / Q000235:genetics / Q000628:therapy*; D000818:Animals; D002852:Chromatography, Ion Exchange; D004285:Dogs; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D008969:Molecular Sequence Data; D011228:Prealbumin / Q000302:isolation & purification*
D011228:Prealbumin