2026-06-14T03:47:19Zhttps://repisalud.isciii.es/rest/oai/requestoai:repisalud.isciii.es:20.500.12105/163952024-09-27T09:27:25Zcom_20.500.12105_19604com_20.500.12105_2051col_20.500.12105_19605
Repisalud
author
Elliott, Perry
author
Gundapaneni, Balarama
author
Sultan, Marla B
author
Ines, Monica
author
Garcia-Pavia, Pablo
funder
Pfizer
funder
Alnylam
funder
BridgeBio
funder
Ionis Pharmaceuticals
funder
AstraZeneca
funder
Novo Nordisk Foundation
funder
Alexion Pharmaceuticals
funder
Intellia
2023-09-04T13:11:32Z
2023-09-04T13:11:32Z
2023-07-11
Eur J Heart Fail. 2023 Jul 11
10.1002/ejhf.2974
1879-0844
European journal of heart failure
37434378
http://hdl.handle.net/20.500.12105/16395
AIM
The value of disease-modifying therapies (such as tafamidis) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and severe heart failure symptoms has been debated. This study assessed long-term all-cause survival in patients with New York Heart Association (NYHA) class III symptoms in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) long-term extension (LTE) study.
METHODS AND RESULTS
At the baseline of ATTR-ACT, 55/176 (31.3%) patients receiving tafamidis 80 mg and 63/177 (35.6%) receiving placebo had NYHA class III symptoms. After 30 months of treatment, patients could join an ongoing LTE study to receive open-label tafamidis. In an interim analysis of the LTE study (August 2021), all-cause mortality was lower among patients with NYHA class III symptoms who received continuous tafamidis in ATTR-ACT and the LTE study (hazard ratio 0.64; 95% confidence interval 0.41-0.99; median follow-up: 60 months), as compared with those who received placebo in ATTR-ACT and tafamidis in the LTE study (median follow-up: 56 months). Similar findings were observed in patients with NYHA class I/II symptoms at baseline (0.50; 0.35-0.73; tafamidis 80 mg n = 121; placebo n = 114; median follow-up of 61 and 60 months, respectively).
CONCLUSION
We observed reduced all-cause mortality with continuous tafamidis treatment compared with delayed tafamidis treatment (placebo then tafamidis) in patients with NYHA class III symptoms at baseline over a median follow-up of ∼5 years. These findings demonstrate the value of tafamidis treatment in patients with ATTR-CM and severe heart failure symptoms, and emphasize the importance of early treatment.
CLINICAL TRIAL REGISTRATIONS
ClinicalTrials.gov NCT01994889 and NCT02791230.
eng
Improved long-term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe heart failure symptoms.
journal article
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Improved long_term survival_Eur J Heart Fail_2023.pdf
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Improved long_term survival_Eur J Heart Fail_2023.pdf.txt