Dettaglio Progetto Finanziato

Lombardy partner:

– Giovanni Palladini, Fondazione IRCCS Policlinico “S. Matteo” Pavia

Immunoglobulin light chain (AL) amyloidosis is caused by antibody fragments, light chains (LCs), produced by cells from the immune system called plasma cells (PCs). Amyloid LCs target vital organs and form typical deposits. Therapy is based on anti-PC drugs, aiming at reducing LC production, restoring organ function, improving quality of life, and extending survival. Members of our consortium made substantial contributions to the diagnosis, treatment and management of AL amyloidosis. However, previous studies on the natural history of the disease were limited by low numbers of analyzed patients, limited representativeness of patients’ cohorts, lack of molecular data, and analysis approach. In the frame of the EUREKA consortium, we will create a single patients’ registry collecting all new cases of AL amyloidosis evaluated at 4 referral Centers across Europe or at their satellite sites, linked to a cross-border biobank and sample sharing network for the study of both diseasecausing LCs and PCs with advanced molecular technologies. A fifth site will support the consortium with big data analysis and artificial intelligence applied to health. We aim at 1) defining the impact of advanced molecular technologies to promote early diagnosis and guide therapeutic choices; 2) describing the natural history of the disease in a representative cohort of AL patients in the contemporary era of effective anti-PC therapies; 3) investigating and refining novel advanced technologies to detect with high sensitivity residual disease-causing PCs in patients responding to therapy. Besides deepening our current understanding of the biology of AL amyloidosis, the data produced within this study will be instrumental in promoting early diagnosis, personalizing individual patient management and in the design of future clinical trials.