Abstract
Light chain (AL) amyloidosis is a plasma cell dyscrasia characterized by the pathologic production of fibrillar proteins comprised of monoclonal light chains which deposit in tissues and cause organ dysfunction. The diagnosis can be challenging, requiring a biopsy and often specialized testing to confirm the subtype of systemic disease. The goal of treatment is eradication of the monoclonal plasma cell population and suppression of the pathologic light chains which can result in organ improvement and extend patient survival. Standard treatment approaches include high dose melphalan (HDM) followed by autologous hematopoietic stem cell transplantation (SCT) or oral melphalan with dexamethasone (MDex). The use of novel agents (thalidomide, lenalidomide and bortezomib) alone and in combination with steroids and alkylating agents has shown efficacy and continues to be explored. A risk adapted approach to SCT followed by novel agents as consolidation reduces treatment related mortality with promising outcomes. Immunotherapeutic approaches targeting pathologic plasma cells and amyloid precursor proteins or fibrils are being developed. Referral of patients to specialized centers focusing on AL amyloidosis and conducting clinical trials is essential to improving patient outcomes.
Highlights
Primary systemic or light chain amyloidosis (AL) is characterized by a clonal population of plasma cells in the bone marrow that produce monoclonal light chain of kappa or lambda type
Non-transplant approaches While high dose melphalan (HDM)/stem cell transplantation (SCT) is an effective way of achieving rapid, hematologic responses, only 20-25% of patients presenting with AL amyloidosis are eligible for such aggressive treatment [6]
An update of this study showed the median progression free and overall survival was 3.8 and 5.1 years, respectively [39]
Summary
Primary systemic or light chain amyloidosis (AL) is characterized by a clonal population of plasma cells in the bone marrow that produce monoclonal light chain of kappa or lambda type. AL amyloidosis should be suspected in any patient with a monoclonal gammopathy and unexplained shortness of breath, fatigue, edema, weight loss, orthostasis or paresthesias (Table 1)[5]. It often requires an astute clinician because symptoms are diverse and . In patients with monoclonal gammopathy and unexplained fatigue, edema, weight loss or paresthesias, AL amyloidosis should be considered. Troponin I or T provide a quantitative assessment of cardiac damage and BNP and/or NT-proBNP indicate cardiomyocyte stress and are independently associated with survival [21] By using these biomarkers, a staging system has been developed has been developed and patients can be classified as having stage I, II or III disease with survivals of 26, 11 and 3.5 months, respectively [22].
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