Tafamidis in ATTR-CM: Study Reports 39 Percent Mortality, Calls for Better Treatment Strategies
USA: A multicenter study evaluating long-term outcomes of tafamidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) has provided valuable insights into disease progression and survival rates. The findings were published online in JACC: CardioOncology on February 11, 2025.
New real-world data suggest that two out of every five patients with transthyretin amyloid cardiomyopathy treated with tafamidis (Vyndamax and Vyndaqel; Pfizer) may not survive beyond four years. However, despite the high mortality rate, researchers emphasize that these findings should be viewed positively, considering that tafamidis was the first approved treatment for ATTR-CM and has significantly advanced disease management in recent years.
The ATTR-ACT trial established that tafamidis improves survival and reduces cardiovascular hospitalizations in transthyretin amyloid cardiomyopathy, a progressive and life-threatening condition caused by the deposition of misfolded transthyretin proteins in the heart. This leads to restrictive cardiomyopathy and heart failure. As disease recognition and early diagnosis improve, the epidemiology of ATTR-CM is rapidly evolving. Tafamidis, the only approved disease-modifying therapy, stabilizes transthyretin to slow disease progression and enhance survival. However, real-world long-term data on its effectiveness remain limited, highlighting the need for further research on tafamidis outcomes in clinical practice.
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