Tafamidis

Summary: All ATTRwt patients on tafamidis 61 mg treatment will be clinically evaluated before treatment initiation and subsequently every six months for the eligibility to continue tafamidis treatment, according to Italian Medicines Agency regulations. C onsidering the significant risk of developing heart rhythm disturbances due to cardiac amyloidosis, especially in transthyretin form (ATTRwt), in routine ...

Summary: Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (va...

Summary: The objective of this study is to determine the association of clinically prescribed, on-label, TTR stabilizing therapy (e.g. tafamidis or acoramidis) with levels of circulating transthyretin amyloid aggregates (TAAs, a surrogate for amyloid disease activity) measured serially over time in patients with transthyretin cardiac amyloidosis (ATTR-CA). To accomplish this objective, the hypothesis that ...

Summary: The goal of this clinical trial is to investigate whether new imaging techniques can help us to better understand the cardiac amyloidosis. The disease can be slowed down with various medications (e.g., tafamidis, acoramidis, or vutrisiran). However, treatment is not effective in all patients-in about one-third of cases, the disease continues to progress. So far, we know little about the exact caus...

Summary: Transthyretin cardiac amyloidosis (ATTR-CA) is a relentlessly progressive disease that can progress to end stage heart failure, at which point recently approved transthyretin production silencing or structure stabilizing therapies provide no clinical benefit. For well-selected individuals, heart transplantation is an excellent therapeutic option to improve survival. Historically, concomitant liver...

Summary: The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy

Summary: Transthyretin amyloid cardiomyopathy (ATTR-CM), is a heart muscle disease that's stops the heart muscle working properly. With an ageing population, it is increasingly common but untreated, it has a poor prognosis. Several novel expensive treatments have become available, although we do not understand exactly how they work and why some patients respond, and others do not. The challenge is to devel...

Summary: This is a mono-center observational ambispective study in which patients with cardiac amyloidosis evaluated at the Fondazione Policlinico Universitario Agostino Gemelli IRCCS (Rome, Italy) will be enrolled. The primary aim is to investigate echocardiographic findings, particularly using advanced echocardiographic techniques, such as two- and three-dimensional speckle-tracking analysis, that may be...

Introduction: Transthyretin cardiac amyloidosis (ATTR) is an important cause of heart failure. Cardiac planar radionuclide imaging using 99mTc-labeled bone seeking radiopharmaceuticals is used as a noninvasive diagnostic criterion in patients without detectable monoclonal protein. The visual assessment remains the main noninvasive criterion for the diagnosis. Medical therapy using tafamidis meglumine that binds...

Summary: Recently, treatment with tafamidis in patients with cardiac ATTR lead to a significant reduction in mortality. The Perugini score is commonly used on planar bone scans to differentiate cardiac ATTR from other amyloidosis or normal patients but fails to evaluate amyloid burden and patient prognosis. Although semi-quantitative methods have been suggested to evaluate the amyloid burden, there a need ...