Induction Therapy with Bortezomib and Dexamethasone and Conditioning with High-Dose Melphalan and Bortezomib Followed by Autologous Stem Cell Transplantation for Immunoglobulin Light Chain Amyloidosis: Long-Term Follow-Up Analysis.

Abstract

In immunoglobulin light-chain (AL) amyloidosis, the depth of hematologic response to treatment is associated with improved survival and organ responses. We conducted a clinical trial using bortezomib in induction and in conditioning with melphalan before 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. We describe survival, durability of hematologic and organ responses, and relapse rates. Thirty-five patients were enrolled between 2010 and 2013. Hematologic complete response and very good partial response (VGPR) were noted in 100% (27 of 27) of the evaluable patients at 6 months post-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 survival and progression-free survival have not yet been reached at the time of this report. Renal and cardiac responses occurred in 65% and 88%, respectively, at 5 years post-SCT. The 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.

• Authors: Vishal K Gupta, Dina Brauneis, Anthony C Shelton, Karen Quillen, Shayna Sarosiek, J Mark Sloan, Vaishali Sanchorawala
• Journal: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (Biol Blood Marrow Transplant) Vol. 25 Issue 5 Pg. e169-e173 (05 2019) ISSN: 1523-6536 [Electronic] United States
• PMID: 30639823 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2019 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

Chemical References

• Antineoplastic Agents
• Myeloablative Agonists
• Bortezomib
• Dexamethasone
• Melphalan

Topics

• Adult
• Aged
• Antineoplastic Agents (therapeutic use)
• Bortezomib (therapeutic use)
• Dexamethasone (therapeutic use)
• Female
• Follow-Up Studies
• Hematopoietic Stem Cell Transplantation (methods)
• Humans
• Immunoglobulin Light-chain Amyloidosis (therapy)
• Male
• Melphalan (therapeutic use)
• Middle Aged
• Myeloablative Agonists (therapeutic use)
• Remission Induction (methods)
• Transplantation Conditioning (methods)
• Transplantation, Autologous
• Treatment Outcome