Phase II trial of high-dose dexamethasone for previously treated immunoglobulin light-chain amyloidosis

Immunoglobulin light-chain amyloidosis (AL) is a rare disorder characterize d by production of a monoclonal light chain, This insoluble light chain, or a fragment thereof, deposits in tissues as amyloid and results in disrupti on of organ function and, ultimately, in death. Although melphalan and pred nisone are beneficial in approximately 30% of patients with the disease, ma ny patients fail to respond, and the median survival with this disease rema ins < 2 years, There is a need for new agents for those patients who fail t o respond to melphalan based chemotherapy, A study was undertaken of high-d ose dexamethasone in the treatment of 19 patients with AL because of report s of its benefits in previously untreated patients with amyloidosis and its known activity in the management of multiple myeloma, which has many chara cteristics in common with AL. In this cohort, 3 of 19 patients showed an ob jective organ response of the disease. The median survival of the entire gr oup was 11.2 months. We conclude that high-dose dexamethasone therapy is of occasional benefit in patients previously treated for amyloidosis, Combini ng dexamethasone with other therapies may increase the response rate. Am. J , Hematol. 61:115-119, 1999, (C) 1999 Wiley-Liss, Inc.