ATTR amyloidosis is a systemic, debilitating and fatal disease caused by transthyretin (TTR) amyloid accumulation. RNA interference (RNAi) is a clinically validated technology that may be a promising approach to the treatment of ATTR amyloidosis. The vast majority of TTR, the soluble precursor of TTR amyloid, is expressed and synthesized in the liver. RNAi technology enables robust hepatic gene...

Tamer Tetiker, Department of Internal Medicine, Medical Faculty, Çukurova University, TR-Adana (Turkey) Dear Sir, Although amyloidosis secondary to collagen vascular diseases, in particular to Beh-çet’s disease [1], is frequently reported, rheumatic heart disease with amyloidosis is rare. Herein, we report a patient with mitral valve stenosis due to rheumatic heart disease and renal amyloidosis...

Background Liver transplantation (LTx), introduced in 1990, has served as the only available treatment with capacity to halt the progress of disease in transthyretin amyloidosis. For the most common variant, Val30Met, the effect of a new liver is well known leading to stabilization in the majority of patients. However, not all patients are helped by transplantation. Progress of cardiac amyloido...

Objectives The benefit of autologous stem cell transplantation (ASCT) in the treatment light chain (AL) amyloidosis requires re-evaluation modern era. This retrospective case-matched study compares ASCT to bortezomib for patients with AL amyloidosis. Methods Newly diagnosed treated or between 2001 and 2018 were identified. Patients excluded if time from diagnosis exceeded 12 months. matched on ...

Amyloidosis is a multi-systemic diffusely infiltrating disease due to extracellular deposition of protein-mucopolysaccharide complexes. The type of protein deposited determines the subgroup of amyloid. Hepatic amyloidosis is a rare infiltrating disease affecting the hepatic parenchyma. A wide range of clinical presentation and atypical imaging findings delay the diagnosis of amyloidosis, while ...

Introduction AL amyloidosis is actually known to be the most common form of systemic amyloidosis in the Western countries [1, 2]. In this particular type of amyloidosis monoclonal immunoglobulin light chains, produced by plasma cell clone, undergo aggregation and form amyloid deposits, almost always systemically, and kidneys are one of the most frequent sites of amyloid deposition [3, 4, 5]. AL...

et al. Hereditary fibrinogen A alpha-chain amyloidosis: phenotypic characterization of a systemic disease and the role of liver transplantation. treatment, and prognosis of hereditary fibrinogen Aalpha-chain amyloidosis. Mutant fibrinogen A-alpha-chain associated with hereditary renal amyloidosis and peripheral neuropathy. and serial monitoring of human visceral amyloid deposits provide evidenc...