Tafamidis boosts survival for patients with transthyretin amyloid cardiomyopathy

Continuous treatment with tafamidis is associated with significant improvements in five-year survival among patients with transthyretin amyloid cardiomyopathy (ATTR-CM), according to new findings published in Circulation: Heart Failure.

Researchers examined updated data from patients who participated in the ATTR-ACT trial, which was originally designed to compare ATTR-CM treatment with tafamidis meglumine and a placebo after 30 months. While that study confirmed that tafamidis meglumine was associated with reductions in mortality and functional decline, this newer analysis followed participants for a median of 58 total months; 176 participants originally given tafamidis meglumine were moved to tafamidis, and 177 patients originally given a placebo were moved to tafamidis meglumine.

Overall, the team reported, continuous treatment with tafamidis meglumine and/or tafamidis was linked to a 41% reduction in the risk of all-cause mortality.

In addition, the five-year survival rate was 53.2% among patients in the continuous treatment group and 32.4% among patients who started off with a placebo before being moved to tafamidis meglumine.

The analysis was funded by Pfizer, the manufacturer of both tafamidis and tafamidis meglumine. Pfizer sells tafamidis under the name Vyndamax and tafamidis meglumine under the name Vyndaqel.

“The survival benefit seen in the primary analysis is further supported by these long-term results, emphasizing the importance of early diagnosis and treatment with Vyndaqel/Vyndamax for patients living with ATTR-CM,” lead author Perry Elliott, MD, director of the Institute for Cardiovascular Science at University College London, said in a prepared statement. “While there has been progress in the diagnosis of ATTR-CM due to increased awareness and improvements in diagnostic approaches, the condition is still overlooked, preventing patients from benefiting from disease modifying treatment.”

“The results from this analysis build upon the positive primary results from the pivotal trial, ATTR-ACT, and reinforce that Vyndaqel and Vyndamax have the potential to significantly extend survival for patients with ATTR-CM,” added Brenda Cooperstone, MD, chief development officer for rare disease at Pfizer.

The full Circulation: Heart Failure study is available here.

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