TY - JOUR
T1 - Real-World Efficacy of Tafamidis in Patients With Transthyretin Amyloidosis and Heart Failure
AU - Ghoneem, Ahmed
AU - Bhatti, Ammar W.
AU - Khadke, Sumanth
AU - Mitchell, Joshua
AU - Liu, Jennifer
AU - Zhang, Kathleen
AU - Trachtenberg, Barry
AU - Wechalekar, Ashutosh
AU - Cheng, Richard K.
AU - Baron, Suzanne J.
AU - Nohria, Anju
AU - Lenihan, Daniel
AU - Ganatra, Sarju
AU - Dani, Sourbha S.
N1 - Publisher Copyright:
© 2023 Elsevier Inc.
PY - 2023/6
Y1 - 2023/6
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85150307283&partnerID=8YFLogxK
U2 - 10.1016/j.cpcardiol.2023.101667
DO - 10.1016/j.cpcardiol.2023.101667
M3 - Review article
C2 - 36828040
AN - SCOPUS:85150307283
SN - 0146-2806
VL - 48
JO - Current problems in cardiology
JF - Current problems in cardiology
IS - 6
M1 - 101667
ER -