Am J Med. 2022 Jan 23:S0002-9343(22)00031-6. doi: 10.1016/j.amjmed.2022.01.011. Online ahead of print.
The management of immunoglobulin light chain (AL) amyloidosis is complex. Emerging data has 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 (HDM/SCT) 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 FDA-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.