AL amyloidosis: from molecular mechanisms to targeted therapies

摘要: Systemic amyloidosis is caused by misfolding and extracellular deposition of circulating proteins as amyloid fibrils, resulting in the dysfunction vital organs. The most common systemic amyloidosis, light-chain (AL) misfolded light chains produced a small, dangerous B-cell clone. process formation, organ targeting, damage multifaceted and, after disease initiation, complexity downstream pathogenic cascade increases, rendering its control challenge. Because progressive nature disease, early diagnosis to prevent end-stage vital. Improving awareness systematic use biomarkers screening populations at risk may improve still unsatisfactory diagnostic process. Amyloid imaging now emerging an important companion formulating prognosis monitoring effects therapy. An accurate basis for appropriate therapy that risk-adapted response-tailored. Effective treatments targeting clone rapidly profoundly reducing have marked improvements overall survival, making AL successful model all amyloidoses. New therapies deposits are under development, together with novel agents modulating chain aggregation proteotoxicity. future treatment combination will require innovative collaborative rapid translation from bench bedside ultimate aim achieving cure this complex disease.