The use of medications specifically targeted at the disease processes of transthyretin amyloid cardiomyopathy (ATTR-CM) results in favorable outcomes in terms of relevant laboratory parameters, functional capacity, and quality of life, according to a study recently published in the Journal of Cardiac Failure.
ATTR-CM is a progressive disease caused by the accumulation of misfolded protein around the heart, ultimately leading to heart failure. Because this disease is incurable, current therapies are focused on slowing disease progression and alleviating symptoms of heart disease. While these therapies have been studied in clinical trials, comparing their data is difficult because of differences between the studies, such as follow-up periods of different lengths.
Researchers sought to better understand how disease-specific therapies impact outcomes for patients with ATTR-CM as a whole. They thus conducted a literature review, which involved assessing and harmonizing the findings of four randomized controlled trials where patients with ATTR-CM were given amyloid-specific medications. These trials were ATTR-ACT (which studied tafamidis), ATTRibute (which studied acoramidis), APOLLO-B (which studied patisiran) and HELIOS-B (which studied vutrisiran).
Taking all four studies into consideration, the clinical data of 2,086 patients was included. The majority of patients (84.9%) had the wild-type form of ATTR-CM.
Read more about ATTR-CM testing and diagnosis
While different trials had different ways of measuring treatment efficacy, researchers noted a number of key findings. For example, across the maximum follow-up period, they found that the use of therapies specific to ATTR-CM lowered the risk of all-cause death by 28% compared with the placebo. In technical terms, the use of these therapies prevented 2.5 deaths per 100 patient years. At 30 months, therapies specific to ATTR-CM reduced negative cardiovascular events by 42%.
At 12 months, compared with the placebo, the use of therapies specific to ATTR-CM resulted in better outcomes across the 6-minute walking distance test (which measures functional capacity), the Kansas City Cardiomyopathy Overall Score (which measures the extent of heart disease symptoms and impact on quality of life) and a biomarker known as N-terminal prohormone of brain natriuretic peptide (which is raised when significant heart disease is present).
“Regardless of agent and baseline risk profiles, ATTR-targeted medication resulted in early improvements in cardiac biomarkers, quality of life and functional capacity, which are maintained and enhanced over a period of ≥2.5 years,” the authors of the study concluded.
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