%0 Journal Article
%T Autologous Stem Cell Transplant for AL Amyloidosis
%A Vivek Roy
%J Bone Marrow Research
%D 2012
%I Hindawi Publishing Corporation
%R 10.1155/2012/238961
%X AL amyloidosis is caused by clonal plasma cells that produce immunoglobulin light chains which misfold and get deposited as amyloid fibrils. Therapy directed against the plasma cell clone leads to clinical benefit. Melphalan and corticosteroids have been the mainstay of treatment for a number of years and the recent availability of other effective agents (IMiDs and proteasome inhibitors) has increased treatment options. Autologous stem cell transplant (ASCT) has been used in the treatment of AL amyloidosis for many years. It is associated with high rates of hematologic response and improvement in organ function. However, transplant carries considerable risks. Careful patient selection is important to minimize transplant related morbidity and mortality and ensure optimal patient outcomes. As newer more affective therapies become available the role and timing of ASCT in the overall treatment strategy of AL amyloidosis will need to be continually reassessed. 1. Introduction Amyloidosis is a disease of protein misfolding in which the involved protein acquires an abnormal beta-pleated sheet configuration rather than the native alpha helical state [1]. The amyloid protein is insoluble, and its deposition in various tissues causes tissue damage and organ dysfunction. Amyloidosis can be of various types based on the precursor protein involved in amyloid formation. More than 20 different human fibrillar amyloid proteins have been described [2], and accurate identification of the type is crucial to treatment planning. It is also important to distinguish localized from systemic amyloidosis since patients presenting with localized amyloidosis generally do not need systemic treatment. The most common type of systemic amyloidosis (primary amyloidosis or AL amyloidosis) is caused by production of immunoglobulin light chain or light chain fragment secondary to an underlying plasma cell dyscrasia. Other systemic amyloidoses (secondary amyloidoses) include AA amyloid (caused as a result of chronic inflammatory disease), ATTR caused by alteration in transthyretin (TTR) protein because of one of several mutations (familial amyloid) or misfolding of wild-type transthyretin protein (senile amyloidosis), and dialysis-related amyloidosis (deposition of beta 2 microglobulin). Primary amyloidosis is the only type of systemic amyloidosis that responds to cytotoxic chemotherapy directed against the abnormal plasma cell clone, the source of amyloidogenic light chains. Other treatment strategies to prevent amyloid formation by altering the equilibrium between soluble precursor and
%U http://www.hindawi.com/journals/bmr/2012/238961/