Cardiovascular disease in the literature: A selection of recent original research papers
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Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med2018;379(11):1007-1016
Background: Transthyretin amyloid cardiomyopathy (ATTR) is caused by deposition of misfolded transthyretin protein in the myocardium. Tafamidis has been demonstrated to bind to transthyretin with high affinity and inhibit its dissociation into monomers. Maurer et al. at the Columbia University Irving Medical Center, NY conducted a multicenter international double-blind placebo-controlled phase 3 clinical trial of 441 patients with ATTR to determine the efficacy and safety of tafamidis. The trial randomized patients (mean age 74 years) with wildtype (76%) or mutant (24%) ATTR confirmed by cardiac or noncardiac biopsy to receive once daily 80 mg tafamidis, and 20 mg tafamidis and placebo in a 2:1:2 ratio. The hierarchically assessed outcomes included all-cause mortality and cardiovascular-related hospitalizations were analyzed in the pooled tafamidis groups vs. placebo.