Treatment with oral melphalan plus dexamethasone produces long-term remissions in AL amyloidosis

Autologous stem cell transplantation (ASCT) is the most effective therapy for AL amyloidosis, but is associated with significant mortality. Reducing the dose of melphalan used in conditioning, also reduces the response rate. We reported that oral melphalan and dexamethasone (MDex) grants a high response rate (67%, complete remission 33%) and is well tolerated in patients ineligible for ASCT. To investigate the durability of response and its effect on survival, we extended the follow-up of the original cohort of 46 patients treated with MDex. After a 5 year median follow-up of living patients, 21 subjects died and overall median survival is 5.1 years. Hematologic response prolonged survival. Complete remissions (CR) were maintained for >3 years in 70% of cases and could be restored by repeating MDex in all relapsing patients. Cause-specific survival of patients in CR was 100%. Oral MDex is a viable alternative to ASCT performed with reduced-intensity conditioning.