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. 2025 Nov 29.
doi: 10.1002/ejhf.70071. Online ahead of print.

Clinical phenotype and prognosis of real-world patients with wild-type transthyretin amyloid cardiomyopathy treated with tafamidis

Aldostefano Porcari #  1   2   3 , Paolo Milani #  4   5 , Simone Longhi  2   6 , Francesco Cappelli  7   8 , Fabio Vagnarelli  9 , Alberto Aimo  10   11 , Alberto Cipriani  12 , Elisa Gardini  13 , Stefania Marazia  14 , Emanuele Monda  15 , Giacomo Tini  16 , Beatrice Musumeci  16 , Matteo Serenelli  17 , Anna Cantone  17 , Carla Lofiego  9 , Marco Marini  9 , Giuseppe Vergaro  10   11 , Grazia Foti  18 , Francesco Musca  18 , Daniela Tomasoni  19 , Giacomo Bonacchi  7   8 , Federica Colio  7   8 , Giulio Sinigiani  12 , Laura De Michieli  12 , Francesca Sturdà  14 , Marco Pozzan  1   2 , Piero Gentile  18 , Samuela Carigi  20 , Michela Bartolotti  21 , Giuseppe Sena  6   22 , Irene Ruotolo  6   22 , Giuseppe Damiano Sanna  4   5   23 , Margherita Zanoletti  24   25 , Marco Canepa  24   25 , Massimo Di Marco  26 , Emilia D'Elia  27 , Gianluca Di Bella  28 , Mauro Driussi  29 , Massimo Imazio  29 , Federico Perfetto  7   8 , Elena Biagini  2   6 , Giuseppe Limongelli  2   15 , Marco Metra  19 , Michele Emdin  10   11 , Giampaolo Merlini  4   5 , Stefano Perlini  5   30 , Marco Merlo  1   2 , Giovanni Palladini #  4   5 , Gianfranco Sinagra #  1   2
Affiliations

Clinical phenotype and prognosis of real-world patients with wild-type transthyretin amyloid cardiomyopathy treated with tafamidis

Aldostefano Porcari et al. Eur J Heart Fail. .

Abstract

Aims: Tafamidis reshaped the treatment paradigm in transthyretin amyloid cardiomyopathy (ATTR-CM) based on a phase-3 randomized controlled trial, but real-world data on its use remain limited. This study aimed to assess in a large, contemporary, real-world cohort of patients with wild-type ATTR-CM (ATTRwt-CM) (i) the clinical phenotype of patients receiving tafamidis, and (ii) the association of tafamidis with survival using propensity-matched observational data.

Methods and results: Data of patients diagnosed with ATTRwt-CM (January 2017 to June 2023) from 19 Italian centres were analysed. A propensity score (PS) reflecting the likelihood of being treated with tafamidis for each patient was determined using four variables that were significantly different among the two groups: age, New York Heart Association (NYHA) class, National Amyloidosis Centre (NAC) stage and mineralocorticoid receptor antagonists (MRAs). The primary outcome was all-cause mortality. The study comprised 1556 ATTRwt-CM patients: 965 (62%) patients initiated on tafamidis by June 2023 and 591 (38%) patients never treated with disease-modifying therapy. Tafamidis-treated patients were older, exhibited a lower NYHA class and NAC stage, and were more often treated with MRAs compared to untreated patients. The PS-matched cohort comprised 426 patients treated with tafamidis and 426 PS-matched untreated patients (mean age 78.9 ± 5.0 years, 88.3% men, 12.9% in NYHA class III). Adequacy of matching was verified (standardized differences: <0.20 between groups). Over 25 months (interquartile range: 15-40), treatment with tafamidis was associated with lower rates of all-cause mortality (hazard ratio 0.55, 95% confidence interval 0.39-0.77, p = 0.001) across the spectrum of NAC disease stages (p-interaction = 0.94).

Conclusions: In this large, contemporary, real-world cohort of patients with ATTRwt-CM, predominantly in NYHA class I or II, treatment with tafamidis was consistently associated with a significantly lower risk of all-cause mortality.

Keywords: Clinical phenotype; Disease‐modifying treatment; Survival; Transthyretin amyloid cardiomyopathy.

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References

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