ABSTRACT
Primary systemic amyloidosis (AL) is a rare plasma cell dyscrasia with approximately 2000 cases diagnosed yearly in North America. In this disease, monoclonal immunoglobulin light chains are produced by malignant plasma cells resulting in amyloid fibril deposition in vital organs leading to progressive, fatal multi-system failure. With the promising outcomes observed with high dose chemotherapy and autologous hematopoietic stem cell transplantation in the more common plasma cell dyscrasia, multiple myeloma, pilot trials to test this approach in AL were pursued. These pilot trials, which almost uniformly utilized high dose melphalan, again based upon the experience in multiple myeloma, showed encouraging results: hematologic response rates were appreciated in approximately 60 percent of the patients; organ response rates were also observed but at a lower rate. Unfortunately, these early pilot trials were associated with high transplant-related mortality. Since AL is a rare disease, there are only a few academic programs with an in-depth focus on the treatment of this disease.
