Clinical characteristics, outcome, and therapeutic effect of tafamidis in wild-type transthyretin amyloid cardiomyopathy.
Biomarkers
Imaging
Prognosis
Tafamidis
Transthyretin amyloid cardiomyopathy
Journal
ESC heart failure
ISSN: 2055-5822
Titre abrégé: ESC Heart Fail
Pays: England
ID NLM: 101669191
Informations de publication
Date de publication:
08 2023
08 2023
Historique:
revised:
03
01
2023
received:
21
10
2022
accepted:
30
03
2023
medline:
31
7
2023
pubmed:
19
4
2023
entrez:
19
04
2023
Statut:
ppublish
Résumé
Tafamidis improves prognosis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). However, real-world data on the therapeutic effect of tafamidis are lacking. This study aimed to evaluate the clinical course, outcomes, and effectivity monitoring of the therapeutic effect of tafamidis in patients with ATTR-CM. This is a single-centre, retrospective observational study. We evaluated the clinical characteristics and outcomes in 125 consecutive patients with wild-type ATTR-CM (ATTRwt-CM) treated with tafamidis (treatment group) and 55 untreated patients (treatment-naïve group). We monitored the therapeutic effect of tafamidis for 12 months by evaluating serial cardiac biomarker and imaging findings. The treatment group had significantly more favourable outcome in all-cause mortality and hospitalization due to heart failure than the treatment-naïve group in both the entire cohort (P < 0.01) and the propensity score-matched cohort (P < 0.05). Kaplan-Meier survival curves showed that tafamidis treatment significantly reduced all-cause mortality (P = 0.03, log-rank test), with the curves diverging after approximately 18 months of treatment in the propensity score-matched cohort. On inverse probability of treatment weighting analysis, tafamidis treatment showed a reduced all-cause mortality [hazard ratio (HR), 0.31; 95% confidence interval (CI), 0.11-0.93; P = 0.04]. High-sensitivity cardiac troponin T (hs-cTnT) > 0.05 ng/mL, B-type natriuretic peptide (BNP) > 250 pg/mL, and estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m The prognosis of patients with ATTRwt-CM treated with tafamidis was more favourable than that of untreated patients. Patient stratification combined with biomarkers (hs-cTnT, BNP, and eGFR) predicted clinical outcomes. hs-cTnT may be a useful biomarker for evaluating the therapeutic effect of tafamidis.
Identifiants
pubmed: 37073415
doi: 10.1002/ehf2.14380
pmc: PMC10375127
doi:
Substances chimiques
tafamidis
8FG9H9D31J
Prealbumin
0
Biomarkers
0
Types de publication
Observational Study
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
2319-2329Informations de copyright
© 2023 The Authors. ESC Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology.
Références
JACC Heart Fail. 2014 Aug;2(4):358-67
pubmed: 25023822
Eur Heart J. 2015 Oct 7;36(38):2585-94
pubmed: 26224076
JACC Cardiovasc Imaging. 2018 Dec;11(12):1924-1926
pubmed: 30121265
J Am Heart Assoc. 2022 Jun 21;11(12):e024717
pubmed: 35699194
J Am Coll Cardiol. 2010 Sep 28;56(14):1071-8
pubmed: 20863950
J Am Coll Cardiol. 2016 Sep 6;68(10):1014-20
pubmed: 27585505
Eur Heart J. 2017 Jun 21;38(24):1895-1904
pubmed: 28329248
ESC Heart Fail. 2022 Jun;9(3):1731-1739
pubmed: 35191205
Circ J. 2022 Jun 24;86(7):1121-1128
pubmed: 35599003
Circulation. 2019 Jan 22;139(4):431-443
pubmed: 30586695
Heart Lung. 2002 Jul-Aug;31(4):262-70
pubmed: 12122390
Circ Heart Fail. 2022 Jan;15(1):e008193
pubmed: 34923848
JACC Cardiovasc Imaging. 2021 Jan;14(1):189-199
pubmed: 33129740
JACC Heart Fail. 2021 Feb;9(2):115-123
pubmed: 33309574
J Cardiol. 2018 Feb;71(2):135-143
pubmed: 29113705
N Engl J Med. 2018 Sep 13;379(11):1007-1016
pubmed: 30145929
Eur J Heart Fail. 2021 Feb;23(2):277-285
pubmed: 33070419
Sci Rep. 2019 Jan 10;9(1):33
pubmed: 30631096
J Am Coll Cardiol. 2013 Aug 13;62(7):632-40
pubmed: 23644085
Circulation. 2016 Jun 14;133(24):2404-12
pubmed: 27143678
Circulation. 2017 Apr 4;135(14):1357-1377
pubmed: 28373528
ESC Heart Fail. 2023 Aug;10(4):2319-2329
pubmed: 37073415
Angiology. 2001 Apr;52(4):273-8
pubmed: 11330510
Eur Heart J. 2021 Apr 21;42(16):1554-1568
pubmed: 33825853
ESC Heart Fail. 2020 Oct;7(5):2829-2837
pubmed: 32672425
Eur Heart J Cardiovasc Imaging. 2022 Jun 1;23(6):767-780
pubmed: 34788394
Circ J. 2019 Dec 25;84(1):15-17
pubmed: 31735731
Circulation. 2019 Jul 2;140(1):16-26
pubmed: 31109193
Eur Heart J. 2018 Aug 7;39(30):2799-2806
pubmed: 29048471
ESC Heart Fail. 2019 Feb;6(1):232-236
pubmed: 30478886