High-Dose Melphalan versus Melphalan plus Dexamethasone for AL Amyloidosis

Abstract

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].).

Melphalan and dexamethasone with or without bortezomib in newly diagnosed AL amyloidosis: a matched case–control study on 174 patients

“…The French multicenter trial showed no difference in response rate and survival between MDex and risk-adapted ASCT. 28 Despite the general good tolerability of MDex, the toxicity of high-dose dexamethasone in AL amyloidosis is not negligible, most common concerns being fluid retention and arrhythmias, 29,30 requiring dose reductions. Recently, we reported the results of a large 1 retrospective study showing that hematologic response could be achieved in 76% (CR in 31%) of patients who were fit enough to receive MDex with full-dose dexamethasone, whereas only 51% (CR 12%) of patients responded to MDex with attenuated dexamethasone.…”

Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide

“…The advent of dexamethasone-based regimens, particularly the combinations with melphalan (M-Dex) [2,3] and with cyclophosphamide and thalidomide (CTD) [4], offered patients with advanced disease effective and less toxic alternatives to autologous stem cell transplantation (ASCT). However, patients with heart failure often cannot tolerate high-dose dexamethasone, because of fluid retention and ventricular arrhythmias [5].…”

Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis

“…25,41 At the same time data have been accumulating, that in centers with low numbers of AL amyloidosis transplants TRM might be higher than in experienced centers. 26,27 Renal toxicity is also remarkable 54,55 especially in patients with kidney involvement, high proteinuria and low serum albumin levels. In a retrospective trial at the Mayo Clinic 80 AL amyloidosis patients were studied.…”

“…The greatest obstacle is treatment-related mortality (TRM), which could substantially be reduced in experienced centers in recent years. 25,26 There is only one randomized trial 27 in which 100 patients were randomly assigned to receive HDM or M-dex, which is considered to be the standard treatment for patients not eligible for auto-HCT. The outcome of the HDM group was inferior with a median overall survival (OS) of 22 months compared with 57 months (P ¼ 0.04).…”