[Treatment of amyloidosis]

Treatment of amyloidosis is a substantial challenge. Various types of amyloidosis are associated with a broad spectrum of organ manifestations and considerable morbidity and mortality.

The present paper is based on a review of international literature, retrieved through a PubMed search on amyloidosis, and the author's clinical experience.

Current treatment of amyloidosis is first and foremost aimed at reducing the level of amyloid precursor proteins; e.g. immunosuppression in AA amyloidosis, chemotherapy in AL amyloidosis and supportive treatment of diminished or lost organ function. Aggressive immunomodulating treatment increases survival in AA amyloidosis associated with rheumatic disease, and high-dose melphalan with autologous stem-cell transplantation (HMAS) has in selected studies shown increased survival in AL amyloidosis. More recent knowledge on mechanisms of amyloid formation has given rise to novel therapeutic targets that may be useful for all amyloidoses. Examples of this are immunotherapy and inhibition of the interaction of extrafibrillary components, which have shown benefit in animal models for AA, AL and Abeta amyloidosis as well as in the first human studies. More studies are needed to define their place in treatment of amyloidosis.