A new systematic literature review and meta-analysis has determined that tafamidis treatment in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) is associated with a low risk of all-cause mortality or cardiovascular events and led to no significant deterioration in left ventricle ejection fraction in patients with wild-type ATTR (wtATTR).

The review and meta-analysis, published in eClinical Medicine, also found a low risk of heart transplantation, heart assist device implantation, heart failure exacerbations, or hospitalizations in the treated patients.

“Previous studies have reported that tafamidis treatment was associated with better outcomes in patients with ATTR-CM compared with those without tafamidis treatment,” the authors wrote. “Therefore, we aimed to systematically assess the association of tafamidis treatment with outcomes in patients with ATTR-CM.”

The research team reviewed 15 published studies covering 2765 patients between January 1, 2000 and June 1, 2023, to assess the impact of tafamidis therapy on the prognosis of patients with ATTR-CM. The studies included cohort studies, randomized controlled trials, and early-phase clinical trials.

Read more about ATTR-CM therapies

The findings revealed a decrease in left ventricle ejection fraction overall, but no significant deterioration in left ventricle ejection fraction in those with wtATTR. In all patients, there was a reduced risk of all-cause death or heart transplantation compared with patients without tafamidis treatment. Finally, a subgroup analysis revealed no significant differences in outcomes between patients with wtATTR or hereditary ATTR who were treated with tafamidis.

The authors concluded that their review underscored the survival benefit of treatment with tafamidis in ATTR-CM, thereby strengthening the evidence clinicians can use in recommending the drug to their patients. They recommended further studies with larger sample sizes and longer follow-ups to more definitively assess the efficacy of tafamidis treatment in patients with ATTR.


Wang J, Chen H, Tang Z, et al. Tafamidis treatment in patients with transthyretin amyloid cardiomyopathy: a systematic review and meta-analysis. eClin Med. Published online August 24, 2023. doi:10.1016/j.eclinm.2023.102172