Eprodisate in amyloid A amyloidosis: a novel therapeutic approach?

Background: Amyloid A (AA) amyloidosis can complicate chronic inflammatory diseases, chronic infections and recurrent periodic fever syndromes. Its treatment is challenging, given its heterogeneous spectrum of etiologies.

Objective: To review the available literature regarding treatment options for AA amyloidosis, particularly focusing on eprodisate, a newly developed inhibitor of fibrillogenesis.

Methods: A PubMed search was performed without any date limits, mainly using the search terms 'amyloidosis', 'colchicine', 'eprodisate', '1,3-propanedisulfonate', 'NC-503', 'Fibrillex' and 'TNF-blockers'.

Results/conclusion: Antibiotics and colchicine are effective in preventing and treating infection-related and familial Mediterranean fever-related AA amyloidosis, respectively. Recently, TNF-alpha blockers have emerged as effective agents in inflammatory AA amyloidosis. Eprodisate binds to the glycosaminoglycan binding site on amyloid fibrils, thus targeting amyloid fibril polymerization and tissue deposition. Eprodisate has possible applicability to other types of amyloidosis; the results of a recent randomized trial showed that it may slow the progression of AA amyloidosis-related renal disease but confirmatory studies are necessary.