Real-world efficacy of tafamidis in patients with transthyretin amyloidosis and heart failure Review


Authors: Ghoneem, A.; Bhatti, A. W.; Khadke, S.; Mitchell, J.; Liu, J.; Zhang, K.; Trachtenberg, B.; Wechalekar, A.; Cheng, R. K.; Baron, S. J.; Nohria, A.; Lenihan, D.; Ganatra, S.; Dani, S. S.
Review Title: Real-world efficacy of tafamidis in patients with transthyretin amyloidosis and heart failure
Abstract: Tafamidis was associated with a reduction in cardiovascular hospitalizations and all-cause mortality in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in the ATTR-ACT trial. However, real-world data on the efficacy of tafamidis are limited. We conducted a retrospective, observational cohort study using the TriNetX research network. Patients with wild-type TTR amyloidosis and heart failure (HF) were divided into 2 groups based on treatment with tafamidis. Propensity score matching (PSM) was performed, and rates of heart failure exacerbations (HFE) and all-cause mortality at 12 months were compared. After PSM, 421 patients were in each group (tafamidis vs nontafamidis). During the 12-month follow-up period, patients treated with tafamidis experienced significantly less HFE and all-cause mortality. A higher probability of event-free survival for HFE and all-cause mortality was noted with tafamidis. This real-world analysis supports that tafamidis use is associated with reduced HFE and all-cause mortality in patients with wild-type TTR amyloidosis and HF. Longer-term follow-up is needed to better understand the utility of tafamidis, given the increasing recognition of ATTR-CM and the high cost of tafamidis. © 2023 Elsevier Inc.
Journal Title: Current Problems in Cardiology
Volume: 48
Issue: 6
ISSN: 0146-2806
Publisher: Mosby Elsevier  
Date Published: 2023-06-01
Start Page: 101667
Language: English
DOI: 10.1016/j.cpcardiol.2023.101667
PUBMED: 36828040
PROVIDER: scopus
DOI/URL:
Notes: Review -- Source: Scopus
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  1. Jennifer Liu
    117 Liu