High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis
Arnaud Jaccard, Philippe Moreau, Veronique Leblond, Xavier Leleu, Lotfi Benboubker, Olivier Hermine, Christian Recher, Bouchra Asli, Bruno Lioure, Bruno Royer, Fabrice Jardin, Frank Bridoux, Bernard Grosbois, Jérome Jaubert, Jean-Charles Piette, Pierre Ronco, Fabrice Quet, Michel Cogne, Jean-Paul Fermand, Myélome Autogreffe (MAG) and Intergroupe Francophone du Myélome (IFM) Intergroup, Arnaud Jaccard, Philippe Moreau, Veronique Leblond, Xavier Leleu, Lotfi Benboubker, Olivier Hermine, Christian Recher, Bouchra Asli, Bruno Lioure, Bruno Royer, Fabrice Jardin, Frank Bridoux, Bernard Grosbois, Jérome Jaubert, Jean-Charles Piette, Pierre Ronco, Fabrice Quet, Michel Cogne, Jean-Paul Fermand, Myélome Autogreffe (MAG) and Intergroupe Francophone du Myélome (IFM) Intergroup
Abstract
Background: High-dose chemotherapy followed by autologous hematopoietic stem-cell transplantation has been reported to provide higher response rates and better overall survival than standard chemotherapy in immunoglobulin-light-chain (AL) amyloidosis, but these two strategies have not been compared in a randomized study.
Methods: We conducted a randomized trial comparing high-dose intravenous melphalan followed by autologous hematopoietic stem-cell rescue with standard-dose melphalan plus high-dose dexamethasone in patients with AL amyloidosis. Patients (age range, 18 to 70 years) with newly diagnosed AL amyloidosis were randomly assigned to receive intravenous high-dose melphalan plus autologous stem cells or oral melphalan plus oral high-dose dexamethasone.
Results: Fifty patients were enrolled in each group. The results were analyzed on an intention-to-treat basis, with overall survival as the primary end point. After a median follow-up of 3 years, the estimated median overall survival was 22.2 months in the group assigned to receive high-dose melphalan and 56.9 months in the group assigned to receive melphalan plus high-dose dexamethasone (P=0.04). Among patients with high-risk disease, overall survival was similar in the two groups. Among patients with low-risk disease, there was a nonsignificant difference between the two groups in overall survival at 3 years (58% in the group assigned to receive high-dose melphalan vs. 80% in the group assigned to receive melphalan plus high-dose dexamethasone; P=0.13).
Conclusions: The outcome of treatment of AL amyloidosis with high-dose melphalan plus autologous stem-cell rescue was not superior to the outcome with standard-dose melphalan plus dexamethasone. (ClinicalTrials.gov number, NCT00344526 [ClinicalTrials.gov].).
Copyright 2007 Massachusetts Medical Society.
Source: PubMed