Key Information
AL amyloidosis is a life-threatening disease related to bone marrow cancer, multiple myeloma. It is caused when a person's antibody-producing white blood cells (i.e. plasma cells) do not function properly and generate abnormal protein which misfolds to form amyloid fibrils and deposits made of components of antibodies called light chains, which then deposit in various organs of the body.
For some and highly selected patients, treatment includes high-dose chemotherapy (melphalan) and autologous (one's own) stem cell transplantation (HDM/SCT).
In the largest study of its kind, researchers from the Boston University/Boston Medical Center Amyloidosis Center have found that HDM/SCT produces prolonged survival with improved safety in patients with AL amyloidosis. Additionally, they have for the first time developed a predictive score to assess event free survival following stem cell transplantation. Event-free survival is defined as the time between stem cell transplantation and initiation of next line of therapy or death, whichever occurs first.