Hematopoietic Cell Transplantation in Light Chain Amyloidosis

Light chain (AL) amyloidosis is a plasma cell disorder characterized by the deposition of monoclonal immunoglobulin light chains in a variety of tissues leading to organ dysfunction. Autologous hematopoietic cell transplantation (HCT) with high-dose melphalan conditioning plays an important role in treating this condition. However, careful patient selection and high-quality supportive care are crucial to minimizing treatment-related mortality. The extent of cardiac involvement is the primary determinant of survival in AL amyloidosis. Other factors to consider when determining transplant eligibility include age, performance status, renal function, pulmonary function, and the number of organs involved. The role of pretransplant induction therapy and posttransplant consolidation therapy will continue to evolve as new treatment options become available. Current practice typically involves induction therapy with a proteasome inhibitor-based regimen (e.g., daratumumab/cyclophosphamide/bortezomib/ dexamethasone). The depth of response to treatment is the best indicator of response durability, and consolidation therapy is generally offered to patients who achieve less than a very good partial response with HCT. Future efforts will focus on expanding the pool of eligible patients for HCT through early diagnosis and the use of novel treatment strategies, including both plasma cell-directed and fibril-directed therapies.