Journal Article

High-Dose Melphalan versus Melphalan plus Dexamethasone for AL Amyloidosis

Asli, Bouchra, Piette, Jean-Charles, Moreau, Philippe, Ronco, Pierre, Leleu, Xavier, Fermand, Jean-Paul, Lioure, Bruno, Jaccard, Arnaud, Hermine, Olivier, Jardin, Fabrice, Cogne, Michel, Recher, Christian, Bridoux, Frank, Benboubker, Lotfi, Royer, Bruno, Leblond, Veronique, Grosbois, Bernard, Quet, Fabrice, Jaubert, Jérome

2007

Overview

Myeloablative doses of melphalan with rescue by autologous hematopoietic stem cells are currently used in the treatment of immunoglobulin-light-chain (AL) amyloidosis, but the efficacy of the therapy is unproven. This randomized trial compared a regimen of high-dose melphalan with standard doses of melphalan plus high doses of dexamethasone. The aggressive treatment had no survival advantage as compared with conventional treatment with standard-dose melphalan. This trial compared a regimen of high-dose melphalan with standard doses of melphalan plus high doses of dexamethasone. The aggressive treatment had no survival advantage as compared with conventional treatment. The origin of amyloid in systemic immunoglobulin-light-chain (AL) amyloidosis is a clone of plasma cells in the bone marrow that synthesizes monoclonal immunoglobulin light chains. In tissues, these light chains aggregate into amyloid fibrils. The accumulation of amyloid deposits in vital organs leads to progressive disability and death. Life expectancy depends on the degree of organ involvement and ranges from a few years to less than 6 months for patients with severe cardiomyopathy. 1 In the mid-1990s, two randomized trials showed that standard-dose chemotherapy with melphalan and prednisone could prolong survival in patients with AL amyloidosis, 1 , 2 but clinical responses were . . .

• Publisher: Massachusetts Medical Society
• Subject: Aged / Amyloidosis - drug therapy / Amyloidosis - mortality / Amyloidosis - therapy / Biological and medical sciences / Bone marrow / Combined Modality Therapy / Dexamethasone - administration & dosage / Disease Progression / Drug Therapy, Combination / Female / General aspects / Hematopoietic Stem Cell Transplantation / Humans / Immunoglobulin Light Chains / Immunology / Kaplan-Meier Estimate / Life Sciences / Male / Medical sciences / Melphalan - administration & dosage / Middle Aged / Mortality / Myeloablative Agonists - administration & dosage / Stem cells