Role of Autologous Stem Cell Transplantation in Systemic Light Chain Amyloidosis.

ABSTRACT

Systemic light chain (AL) amyloidosis is a multisystem disorder characterized by extracellular deposition of misfolded insoluble amyloid fibrils resulting in progressive organ dysfunction. AL. amyloidosis most commonly affects the heart, kidneys, gastrointestinal tract and peripheral nerves. Early mortality is chiefly determined by the degree of cardiac involvement. The aim of therapy is to rapidly reduce amyloidogenic light chain production by targeting the underlying clonal plasma or lymphoma cell population. High dose therapy with melphalan followed by autologous peripheral blood stem cell transplant (ASCT) continues to remain a highly effective treatment and is considered a standard of care for transplant eligible patients, which offers long term disease control in patients with AL amyloidosis. In recent years, several new therapeutic options have emerged (including anti-CD38 monoclonal antibodies) which are very effective alone or in combination in eradicating clonal plasma cells. In this review, we discuss the role of ASCT in the current setting of a rapidly evolving treatment landscape for patients with AL amyloidosis and provide our practice recommendations.