OVEREXPRESSION OF CALRETICULIN IN CLONAL AL PLASMA CELLS IS ASSOCIATED WITH RESPONSE TO MELPHALAN AND AUTOLOGOUS STEM CELL TRANSPLANT IN SYSTEMIC LIGHT-CHAIN (AL) AMYLOIDOSIS

摘要

Current therapies for AL amyloidosis aim to reduce the precursor protein, the free immunoglobulin light chain (FLC), in order to stabilize or reverse disease and enhance survival Reduction of FLC occurs indirectly as the result of reduction of the clonal plasma cells, the FLC factory. With intravenous melphalan and autologous stem cell transplant (MEL SCT), the most effective single therapy, one-third of patients have a complete or near complete response with elimination of clonal disease (CR, nCR), one-third have a partial response with a greater than 50% reduction (PR) and one-third have no response (a less than 50% reduction; NR) (1). In an attempt to decipher the contribution made by clonal AL plasma cells to this response distribution, we studied the gene expression profiles of purified clonal AL plasma cells from newly diagnosed untreated patients who then underwent MEL SCT, and compared the profiles of patients who achieved CR with those who had NR.