Advances in the amyloidosis space: Prognostic factors, CAEL-101 immunotherapy and real-world data

Amyloidosis is the name attributed to a group of diseases caused by the extracellular accumulation of amyloid, an abnormal, insoluble protein. Amyloid forms when protein misfolding transforms soluble precursor proteins into insoluble amyloid fibrils. Amyloid fibrils can disseminate systemically and deposition on organs causes disturbance of organ function. Deposition in cardiac tissue, kidneys and the central nervous system are the primary causes of patient deterioration. There are four main types of amyloidosis: AL (light chain), AA (inflammation), ATTR (hereditary and old age) and AB2M (caused by dialysis). AL amyloidosis is the most common type and is caused by clonal plasma cell dyscrasia. Treatment strategies depend on the type of amyloidosis. Recent treatment developments include novel chemotherapy agents and immunotherapies with monoclonal antibody therapies in particular showing promise.

In this podcast, Morie Gertz, MD, MACP, of the Mayo Clinic College of Medicine, Rochester, MN, Jason Valent, MD, from the Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, and Efstathios Kastritis, MD, of the University of Athens School of Medicine, Athens, Greece, discuss updates in the amyloidosis field presented at this year’s virtual American Society of Hematology (ASH) Annual Meeting and Exposition.