Lead Investigator: Joel Skaistis, Corewell Health
Title of Proposal Research: Effect of tafamidis treatment on total cardiovascular and cerebrovascular events in Transthyretin (TTR) cardiac amyloidosis
Vivli Data Request: 8985
Funding Source: None
Potential Conflicts of Interest: None
Summary of the Proposed Research:
Transthyretin (TTR) is a protein which is made by the liver and circulates in the blood, transporting substances such as vitamin A and a thyroid hormone, thyroxine, around the body. Mutations in this protein can cause it to misfold or change shape, and to form into clumps called amyloids. A buildup of these abnormal proteins in the body’s organs is called amyloidosis. Cardiac transthyretin amyloidosis (ATTR) is a progressive, life-threatening disease where abnormal TTR is deposited in various organs, particularly the heart. This affects the heart’s ability to function properly, and can cause heart failure and enlarged heart which leads to significant morbidity and mortality.
ATTR is common in certain populations and found in up to 20% of individuals over 60 with heart failure, aortic stenosis (thickening and narrowing of the valve between the heart’s main pumping chamber and the body’s main artery (the aorta)) or who have a pacemaker fitted (a small device that’s implanted in the chest to help control the heartbeat). It has been previously established that treatment of cardiac amyloidosis with tafamidis, a medication that prevents amyloid formation, prevents heart failure progression and fatal events. However, there are other outcomes of concern in the amyloidosis patient population, such as heart attack, stroke, bleeding and pacemaker implantation. The effect of tafamidis on the risk of these events is unknown. This research aims to investigate the effect of tafamidis on the risk of cardiovascular, cerebrovascular and bleeding events. While heart failure risk is not always a primary concern for every patient with amyloidosis, especially those in earlier stages without significant heart failure symptoms, catastrophic events such as heart attack and stroke, typically matter greatly when making healthcare decisions. This information will be vital for physicians and patients in making informed decisions regarding treatment options.
Requested Studies:
TTRACT trial. A phase 3 randomized trial of tafamidis in TTR cardiac amyloidosis
Data Contributor: Pfizer Inc.
Study ID: NCT01994889
Sponsor ID: NCT01994889