Review of Transthyretin Silencers, Stabilizers, and Fibril Removal Agents in the Treatment of Transthyretin Cardiac Amyl
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NEW THERAPIES FOR CARDIOVASCULAR DISEASE (AA BAVRY, SECTION EDITOR)
Review of Transthyretin Silencers, Stabilizers, and Fibril Removal Agents in the Treatment of Transthyretin Cardiac Amyloid Augustus Hough 1
&
Jessica Wearden 2 & Kristen de Almeida 3 & Stephanie Kaiser 1
# This is a U.S. Government work and not under copyright protection in the US; foreign copyright protection may apply 2020
Abstract Purpose of Review To provide a functional review for practicing clinicians on the current and emerging treatment considerations for transthyretin (TTR) cardiac amyloidosis (ATTR-CA). Recent Findings Current treatment considerations are characterized as those silencing TTR translation, stabilizing TTR tetramers, and disrupting amyloid fibril deposition. Summary Historically considered a rare disease state, ATTR-CA is increasingly recognized as an important mediator of heart failure morbidity and mortality. The emergence of widely available therapies for ATTR-CA has developed hope for patients where little was previously present. Thus, it is important that all cardiology clinicians have a functional understanding of the disease state and treatment options. This review will discuss agents within each of the above classes with expanded discussion on tafamidis given its favorable efficacy, safety, and availability. ATTR-CA diagnostic considerations are reviewed with regard to the identification of potential tafamidis candidates, and practical economic considerations are also reviewed. Keywords Transthyretin amyloid cardiomyopathy . ATTR . Treatment . Tafamidis
Introduction Amyloidosis is an umbrella term referring to a broad group of heterogeneous diseases characterized by the tissue deposition of
This article is part of the Topical Collection on New Therapies for Cardiovascular Disease * Augustus Hough [email protected] Jessica Wearden [email protected] Kristen de Almeida [email protected] Stephanie Kaiser [email protected] 1
Clinical Pharmacy Specialist – Cardiology, West Palm Beach Veterans Affairs Medical Center, 7305 N. Military Trial, West Palm Beach, FL 33410, USA
2
PGY2 Cardiology Pharmacy Practice Resident, West Palm Beach Veterans Affairs Medical Center, West Palm Beach, FL, USA
3
Clinical Pharmacy Specialist – Cardiology, Baptist Hospital of Miami, 8900 N. Kendall Dr, Miami, FL 333176, USA
β-sheets of amyloid fibrils [1]. These fibrils are derived from the aggregation of misfolded proteins whose accumulation leads to eventual organ or tissue dysfunction. The manifestations of the various amyloid diseases are determined by the amyloidogenic protein source involved and the location of fibril deposition. Cardiac amyloidosis (CA) is primarily manifested through two different etiologies, transthyretin amyloidosis (ATTR) and immunoglobulin light chain amyloidosis (AL). Recognition of the CA source is key for several reasons. The most critical reason is that, though clinical presentations may be similar, the prognosis of AL-CA is grim with untreated survival periods of less than 6
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