Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy

Transthyretin amyloid cardiomyopathy is a life-threatening disease characterized by the accumulation of amyloid fibrils composed of misfolded transthyretin protein in the heart. Symptoms for the disease are predominately manifested in male patients aged 60 years or older.

Transthyretin amyloid cardiomyopathy is a life-threatening disease characterized by the accumulation of amyloid fibrils composed of misfolded transthyretin protein in the heart. Symptoms for the disease are predominately manifested in male patients aged 60 years or older. Treatments have been limited to supportive care, with no guideline-recommended treatment. Death in most patients is from cardiac causes, including sudden death and heart failure. Patients with the disease who received an experimental Pfizer drug, tafamidis, were 30% less likely to die than those who received a placebo, meaning that 78, or 30%, of 264 people who got either of two doses of the drug died, compared with 76, or 43%, of 177 people who received placebo. That result actually counted a heart transplant or the use of an artificial heart pump called a left-ventricular assist device as a "death." But counting only people who actually died would make the result stronger, reducing deaths by 33%, Pfizer said. Tafamidis also decreased the rate of hospitalization 32%. The drug was also associated with a significant reduction in the decline in functional capacity and the decline in quality of life at month 30, with differences first observed at 6 months. In contrast, the effect on overall survival emerged after approximately 18 months. This dissociation between the effect on symptoms and survival has also been observed with other therapies for systolic heart failure in which ventricular remodeling takes months to achieve.