Treatment: Amyloidosis

For AL, current therapies target the clonal bone-marrow plasma cells using approaches used for multiple myeloma. High-dose IV melphalan followed by autologous stem-cell transplantation produces complete hematologic responses in ~40% but only 50% are eligible for such aggressive treatment, and peritransplant mortality is higher than for other hematologic diseases because of impaired organ function. Treatment of AA is directed toward controlling the underlying inflammatory condition. Colchicine (1.2-1.8 mg/d) is the standard treatment for FMF but has not been helpful for AA of other causes. TNF inhibitors and interleukin-1 antagonists can be effective in syndromes related to cytokine elevation. Eprodisate designed to disrupt fibril formation is being studied. In certain forms of AF, genetic counseling is important and liver transplantation is a successful form of therapy.

For a more detailed discussion, see Seldin DC, Berk JL: Amyloidosis, Chap. 137, p. 719, in HPIM-19.