Novel strategies for the diagnosis and treatment of cardiac amyloidosis.
Systemic amyloidoses are rare, complex diseases caused by misfolding of autologous protein. The presence of heart involvement is the most important prognostic determinant. The diagnosis of amyloid cardiac involvement relies on echocardiography and magnetic resonance imaging, while scintigraphy with bone tracers is helpful in differentiating light chain amyloidosis from other types of amyloidosis involving the heart. Although these diseases are fatal, effective treatments exist that can alter their natural history, provided that they are started before irreversible cardiac damage has occurred. Refined diagnostic techniques, accurate patients' stratification based on biomarkers of cardiac dysfunction, the availability of novel, more powerful drugs, and ultimately, the unveiling of the cellular mechanisms of cardiac damage created a favorable environment for a dramatic improvement in the treatment of this disease that we expect in the next few years.
a Amyloidosis Research and Treatment Center, Foundation Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo and Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Giovanni
G
Palladini
a Amyloidosis Research and Treatment Center, Foundation Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo and Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Paolo
P
Milani
a Amyloidosis Research and Treatment Center, Foundation Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo and Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Giampaolo
G
Merlini
D000686:Amyloidosis / Q000175:diagnosis / Q000628:therapy*; D009202:Cardiomyopathies / Q000175:diagnosis / Q000628:therapy*; D004452:Echocardiography; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods; D016896:Treatment Outcome
RNA-targeted therapy; amyloidosis; biomarkers; cardiac toxicity; chemotherapy; diagnosis