Induction Therapy with Bortezomib and Dexamethasone and Conditioning with High-Dose Melphalan and Bortezomib Followed by Autologous Stem Cell Transplantation for AL Amyloidosis: Long Term Follow-Up Analysis
Vishal Gupta, MD
ABSTRACT:
In light-chain (AL) amyloidosis, the depth of hematologic response to treatment is associated with improved survival and organ responses. We conducted a clinical trial utilizing bortezomib in induction and in conditioning with melphalan prior to stem cell transplantation (SCT) for AL amyloidosis. The results of this clinical trial with a median follow-up of 36 months have been reported previously. Here we report the long-term results of this clinical trial with a median follow-up of 77 months. The objectives of this follow-up report are to describe survival, durability of hematologic and organ responses, and relapse rates. Thirty-five patients were enrolled from 2010 to 2013. Hematologic complete responses (CR) and very good partial responses (VGPR) were noted in 27/27 (100%) of assessable patients at 6 months following SCT. Four patients (15%) had hematologic relapse at a median of 42 months and 1 patient (3.7%) had organ progression despite maintaining a VGPR at 37 months. The median overall and progression-free survivals have not yet been reached. Renal and cardiac responses occurred in 65% and 88% at 5 years post SCT, respectively. Median time to renal and cardiac response was 12 months and 6 months, respectively. In conclusion, incorporating bortezomib into induction and conditioning yielded durable hematologic responses of AL amyloidosis with corresponding organ responses and prolonged survival.