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Natural history and impact of treatment with tafamidis on major cardiovascular outcome‐free survival time in a cohort of patients with transthyretin amyloidosis

European Journal of Heart Failure Nov 27, 2020

Bézard M, Kharoubi M, Galat A, et al. - Given that hereditary (ATTRv) and wild‐type (ATTRwt) transthyretin amyloidosis represent severe and fatal systemic diseases, and a reduction in the relative risk of mortality with tafamidis in ATTR amyloidosis with cardiomyopathy has been reported recently in the Phase III ATTR‐ACT trial, therefore, researchers investigated the clinical features of ATTR amyloidosis in a real‐life population vs the population included in the ATTR‐ACT trial. They also evaluated how major cardiovascular outcome (MCO)‐free survival time without cardiac decompensation, heart transplant, or death, could be impacted by tafamidis treatment. A total of 648 patients with ATTR amyloidosis (423 ATTRwt and 225 ATTRv) who were consecutively referred to the French Referral Center for cardiac amyloidosis were involved. The inclusion criteria of the ATTR‐ACT trial were matched by 467 (72%) patients. A longer median MCO‐free survival time was noted in relation to tafamidis treatment, for the 631 patients with cardiomyopathy. The present findings revealed a lower occurrence of cardiovascular outcomes in a real‐life population in correlation with tafamidis treatment, in a large cohort comprising ATTRwt and ATTRv patients, representative of the inclusion criteria of the ATTR‐ACT trial.

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