Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)

ConclusionsCauses for the observed mortality imbalance associated with revusiran were thoroughly investigated and no clear causative mechanism could be identified. Although the results suggest similar progression of cardiac parameters in both treatment arms, a role for revusiran cannot be excluded.Clinical Trial RegistrationNCT02319005.
Source: Cardiovascular Drugs and Therapy - Category: Cardiology Source Type: research

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Amyloid transthyretin (ATTR) cardiac amyloidosis (CA) is an increasingly recognized cause of restrictive cardiomyopathy and associated heart failure with preserved ejection fraction (HFpEF). Despite improved diagnostic techniques to identify this condition, many patients experience delayed diagnosis. Interpretation of routine cardiac biomarkers (i.e. troponins and brain natriuretic peptide (BNP)) and echocardiography in ATTR-CA is limited, particularly in relation to pyrophosphate (PYP) scintigraphy.
Source: The Journal of Heart and Lung Transplantation - Category: Transplant Surgery Authors: Tags: (64) Source Type: research
We describe a rare case of concomitant moderately severe tricuspid and mitral valve stenosis because of ongoing amyloid deposition in a patient with progressive multiple myeloma and fat pad biopsy ‐proven light chain amyloidosis. Worsening infiltrative cardiomyopathy and valvulopathy despite evidence‐based chemotherapy and heart failure pharmacotherapy led to end‐stage disease and death. Valvular involvement in cardiac amyloidosis requires early recognition of the underlying disease con dition to guide directed medical therapy and prevent its progression. In this instance, valvuloplasty or valve replacement is not a viable option.
Source: ESC Heart Failure - Category: Cardiology Authors: Tags: Case Report Source Type: research
Authors: Kristen AV Abstract Cardiac amyloidosis is a heterogeneous group of diseases characterized by extracellular deposition of amyloid fibrils in many different organs finally resulting in organ failure. Cardiac involvement is common for immunoglobulin light chain amyloidosis (AL) or transthyretin amyloidosis (ATTR); the latter is caused by a transthyretin gene variant or wild-type protein. Precise diagnostic assessment including laboratory tests, electrocardiography, echocardiography, cardiac magnetic resonance imaging, biopsy, and/or bone scintigraphy is mandatory for definition of the amyloid type ...
Source: Herz - Category: Cardiology Tags: Herz Source Type: research
Cardiac Amyloidosis (CAm) is an infiltrative disease caused by abnormal extracellular protein deposits in the myocardium, leading to restrictive cardiomyopathy. The cardinal clinical manifestation is congestion resulting in symptomatic heart failure (HF). Relief of congestion is achieved with high dose loop diuretics and regularly requires hospitalization for intravenous (IV) diuresis. Additionally, CAm is a challenge to manage given sensitivity to preload and afterload, low systemic blood pressures, and inefficacy of advanced therapies typically used in HF.
Source: Heart and Lung - Category: Intensive Care Authors: Source Type: research
Abstract Cardiac amyloidosis (CA) is an infiltrative and restrictive cardiomyopathy that leads to heart failure, reduced quality of life, and death. The disease has two main subtypes, transthyretin cardiac amyloidosis (ATTR-CA) and immunoglobulin light chain cardiac amyloidosis (AL-CA), characterized by the nature of the infiltrating protein. ATTR-CA is further subdivided into wild-type (ATTRwt-CA) and variant (ATTRv-CA) based on the presence or absence of a mutation in the transthyretin gene. CA is significantly underdiagnosed and increasingly recognized as a cause of heart failure with preserved ejection fractio...
Source: Annual Review of Medicine - Category: General Medicine Authors: Tags: Annu Rev Med Source Type: research
AbstractCurrent echocardiography techniques have allowed more precise assessment of cardiac structure and function of the several types of cardiomyopathies. Parameters derived from echocardiographic tissue imaging (ETI) —tissue Doppler, strain, strain rate, and others—are extensively used to provide a framework in the evaluation and management of cardiomyopathies. Generally, myocardial function assessed by ETI is depressed in all types of cardiomyopathies, non-ischemic dilated cardiomyopathy (DCM) in particular . In hypertrophic cardiomyopathy (HCM), ETI is useful to identify subclinical disease in family membe...
Source: Heart Failure Reviews - Category: Cardiology Source Type: research
Publication date: Available online 17 December 2019Source: Canadian Journal of CardiologyAuthor(s): Bennett Di Giovanni, Dakota Gustafson, Mitchell B. Adamson, Diego H. DelgadoAbstractAmyloidosis is a term used to describe a group of rare, heterogenous diseases which ultimately result in the deposition and accumulation of misfolded proteins. These misfolded proteins, known as amyloid, are associated with a variety of precursor proteins which have amyloidogenic potential. Ultimately, the specific type of amyloidosis is dependent on multiple factors including genetic variability of precursor proteins and the tissue/organ in ...
Source: Canadian Journal of Cardiology - Category: Cardiology Source Type: research
CONCLUSION —Tafamidis(Vyndaqel; Vyndamax) can decrease the number of hospitalizations and deaths in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Taking 20 mg ofVyndaqel daily appears to be as effective as 80 mg and costs much less. Long-term data on the comparative efficacy of the two doses are not available.MA Gertz et al. Diagnosis, prognosis, and therapy of transthyretin amyloidosis. J Am Coll Cardiol 2015; 66:2451.FL Ruberg and JL Berk. Transthyretin (TTR) cardiac amyloidosis. Circulation 2012; 126:1286.MS Maurer et al. Tafamidis treatment for patients with transthyretin amyloid cardiomy...
Source: The Medical Letter - Category: Drugs & Pharmacology Authors: Tags: inotersen Onpattro patisiran tafamidis Tegsedi Vyndaqel Vyndmax Source Type: research
ConclusionTakotsubo cardiomyopathy may develop in critically ill cardiac diseases but are often underdiagnosed. Careful echocardiographic examination is needed to unveil these “hidden” TC.
Source: Journal of Echocardiography - Category: Cardiology Source Type: research
Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is an underappreciated cause of heart failure that results from misfolded TTR (prealbumin) protein. Diflunisal is an approved non-steroidal anti-inflammatory drug (NSAID) that stabilizes TTR, with limited data available regarding effects on cardiac structure and function.
Source: Journal of Cardiac Failure - Category: Cardiology Authors: Source Type: research
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