Title: Autologous Stem Cell Transplant in the Era of Bortezomib-Based Induction for AL Amyloidosis – A Single Institution 11 Year Experience

Abstract

A single-institution retrospective outcome analysis was performed on a cohort of 52 patients with systemic light-chain amyloidosis comparing bortezomib-based induction versus no induction before autologous stem cell transplantation (ASCT). Thirteen patients underwent ASCT without induction, 33 received bortezomib-based induction, and 6 received non-bortezomib-based induction. Thirty patients received melphalan 200 mg/m2, while 22 received dose-reduced melphalan. Treatment-related mortality at 100-days was 3.8%. Overall hematologic (ORR) and organ response rates (OR) in the entire cohort were 85% and 67%, respectively. ORR was 94% in those receiving bortezomib-based induction versus 69% with no induction (p=0.04). Median time to maximum hematologic response post ASCT was significantly shorter with bortezomib-based induction (3 months versus 14 months). Five-year progression-free survival (PFS) and overall survival (OS) in the entire cohort were 50% and 73%, respectively. Between groups there were no statistically significant differences in depth of hematologic response, cardiac or renal response rate, or 5-year PFS or OS. This study supports ASCT, with bortezomib-based induction as a well-tolerated therapy, with higher ORR and shorter time to maximum hematologic response than no induction. Larger-scale multi-center randomized trials would be beneficial to confirm these observations.