Update on the Contemporary Treatment of Light Chain Amyloidosis Including Stem Cell Transplantation

The management of immunoglobulin light chain (AL) amyloidosis is complex. Emerging data have shown promising results for several novel agents. We review the management of AL amyloidosis, including factors that determine transplant eligibility, treatment options for transplant-ineligible patients, and treatment options for relapsed/refractory AL amyloidosis. For carefully selected patients, high-dose melphalan and stem cell transplantation is recommended. Transplant eligibility criteria generally include biopsy-proven amyloidosis, evidence of a plasma cell dyscrasia, involvement of at least one major organ, and adequate performance status. For transplant-ineligible patients, bortezomib-based regimens are recommended, including: 1) bortezomib, oral melphalan, and dexamethasone (BMDex); 2) bortezomib, cyclophosphamide, and dexamethasone (CyBorD or VCd); and 3) subcutaneous daratumumab (DARA SC) and VCd. The latter option is based on a landmark trial that led to the first US Food and Drug Administration-approved therapy for AL amyloidosis. For relapsed/refractory disease, novel therapeutics including proteosome inhibitors, immunomodulatory agents, and monoclonal antibodies have shown promising results. In this review, we summarize data for various therapeutics in different clinical scenarios of AL amyloidosis. (C) 2022 Elsevier Inc. All rights reserved.

Automated summary

The management of AL amyloidosis is complex and requires careful consideration of various factors. Emerging data have suggested promising results for novel therapeutic agents. High-dose melphalan and stem cell transplantation is recommended for eligible patients, while bortezomib-based regimens are recommended for transplant-ineligible patients. For relapsed/refractory disease, novel therapeutics such as proteosome inhibitors, immunomodulatory agents, and monoclonal antibodies have shown promising outcomes.