| Abstract: |
In patients with light chain (AL-) amyloidosis, the fibril precursors are usually free light chains (FLCs) possessing an abnormal tertiary structure. AL-amyloidosis from symptomatic multiple myeloma is the pattern of organ damage. This chapter describes the epidemiology, pathogenesis, and distinctive clinical aspects of this disease, and offers an approach to the evaluation of patients with AL-amyloidosis. It also summarizes recent clinical research in autologous hematopoietic cell transplantation (HCT) for AL-amyloidosis. The only effective therapeutic approach to systemic AL-amyloidosis remains reduction of amyloid-forming FLCs while providing best supportive care. The FLC assay provides a direct quantitative measure of the fibril-precursor protein and of response when used serially during the course of therapy. The combination of oral cyclophosphamide, thalidomide and dexamethasone (CTD) showed promise in the relapsed setting. Bortezomib is the most active and most useful of the novel agents in the treatment of AL-amyloidosis. © 2016 John Wiley & Sons, Ltd. All rights reserved. |
