Novel challenges in the management of immunoglobulin light chain amyloidosis: from the bench to the bedside.

Marco Basset, Mario Nuvolone, Giovanni Palladini, Giampaolo Merlini

Expert review of hematology 2020 Sep

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Immunoglobulin light chain (AL) amyloidosis is one of the most frequent systemic amyloidosis in Western countries. It is caused by a B-cell clone producing a misfolded light chain (LC) that deposits in organs. The review examines recent findings on pathophysiology and clinical management of AL amyloidosis. It contains an update on the recent hot topics as novel therapeutic approaches, definition of relapse, and hematologic response assessment. To review literature on AL amyloidosis, a bibliographic search was performed using PubMed. Due to the proteotoxicity of amyloidogenic LCs, the therapeutic goal is a rapid and profound decrease in their concentration. The standard treatment is a risk-adapted chemotherapy targeting the B-cell clone. Novel, promising drugs, as daratumumab, are currently under evaluation in newly-diagnosed and relapsed/refractory patients. New sensitive techniques, as mass spectrometry approach and bone marrow minimal residual disease assessment, are available to evaluate depth of response. After first-line therapy, increase in LC concentration may precede worsening of organ dysfunction and should be considered carefully. Further clarification of molecular mechanisms of the disease are shedding light on new possible therapeutic targets. Innovative treatment strategies and novel technologies will improve our ability to treat AL amyloidosis, preventing organ deterioration.

Citation

Marco Basset, Mario Nuvolone, Giovanni Palladini, Giampaolo Merlini. Novel challenges in the management of immunoglobulin light chain amyloidosis: from the bench to the bedside. Expert review of hematology. 2020 Sep;13(9):1003-1015