New Therapies for Systemic Amyloidosis

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Systemic amyloidosis can involve nearly any organ, and the kidney is often involved. Recall that there are two flavors of amyloidosis: AL amyloidosis (in which the amyloid is formed by light chains as a result of a paraproteinemia) or AA amyloidosis (in which a chronic inflammatory condition, such as rheumatoid arthritis, leads to the excess production of the amyloid-forming acute phase reactant serum amyloid A (SAA) protein.

In recent years there have been a few new approaches to treatment of this often deadly disease. One new medication is eprodisate, which functions by interfering with the interaction of amyloidogenic proteins with tissue glycosoaminoglycans. In a randomized, placebo-controlled trial published in a 1997 NEJM article (Dember et al), individuals who received eprodisate exhibited a slower rate of renal decline. This trial was done in patients with AA amyloidosis but the drug may turn out to be useful for AL amyloidosis as well.

In addition, there are numerous immunologic therapies directed at AL amyloidosis. Low-dose oral melphalan and dexamethasone is one mainstay; in younger healthier patients it is also possible to perform high-dose melphalan with autologous stem cell transplant, though the benefit of this approach is controversial.

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