Evidence map›Paper›PMID 41317156›Full record

Observational studyEuropean journal of heart failure2025

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

Aldostefano Porcari, Paolo Milani, Simone Longhi, Francesco Cappelli, Fabio Vagnarelli, Alberto Aimo, Alberto Cipriani, Elisa Gardini, Stefania Marazia, Emanuele Monda and 39 more

Abstract readMulticenter StudyObservational Study
In one paragraph

Observational study in European journal of heart failure, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

49 authors.

Aldostefano Porcari *Center for Diagnosis and Treatment of Cardiomyopathies, Cardiovascular Department, Azienda Sanitaria Universitaria Giuliano-Isontina (ASUGI), University of Trieste, Trieste, Italy.ORCID https://orcid.org/0000-0003-3307-0825
Paolo Milani *Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Simone LonghiEuropean Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, ERN GUARD-Heart, Trieste, Italy.
Francesco CappelliCardiomyopathy Unit, Careggi University Hospital, University of Florence, Florence, Italy.
Fabio VagnarelliCardiology Unit, 'G.M. Lancisi' Cardiovascular Center, Azienda Ospedaliero Universitaria delle Marche, Ancona, Italy.
Alberto AimoInterdisciplinary Center of Health Sciences, Scuola Superiore Sant'Anna, Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Alberto CiprianiDepartment of Cardiac, Thoracic and Vascular Sciences and Public Health, University of Padua, Padua, Italy.
Elisa GardiniCardiology Unit, Morgagni-Pierantoni Hospital, Forlì, Italy.
Stefania MaraziaCardiology Division, Vito Fazzi Hospital, Lecce, Italy.
Emanuele MondaInherited and Rare Cardiovascular Disease Unit, University of Campania 'Luigi Vanvitelli', AORN dei Colli, Monaldi Hospital, Naples, Italy.
Giacomo TiniDepartment of Clinical and Molecular Medicine, Faculty of Medicine and Psychology, Sapienza University, Rome, Italy.
Beatrice MusumeciDepartment of Clinical and Molecular Medicine, Faculty of Medicine and Psychology, Sapienza University, Rome, Italy.
Matteo SerenelliCardiologic Centre, University of Ferrara, Cona (FE), Italy.
Anna CantoneCardiologic Centre, University of Ferrara, Cona (FE), Italy.
Carla LofiegoCardiology Unit, 'G.M. Lancisi' Cardiovascular Center, Azienda Ospedaliero Universitaria delle Marche, Ancona, Italy.
Marco MariniCardiology Unit, 'G.M. Lancisi' Cardiovascular Center, Azienda Ospedaliero Universitaria delle Marche, Ancona, Italy.
Giuseppe VergaroInterdisciplinary Center of Health Sciences, Scuola Superiore Sant'Anna, Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Grazia FotiCardiology 4, ASST Niguarda Ca' Granda Hospital, Milan, Italy.
Francesco MuscaCardiology 4, ASST Niguarda Ca' Granda Hospital, Milan, Italy.
Daniela TomasoniCardiology, ASST Spedali Civili, Department of Medical and Surgical Specialities, Radiological Sciences, and Public Health, University of Brescia, Brescia, Italy.
Giacomo BonacchiCardiomyopathy Unit, Careggi University Hospital, University of Florence, Florence, Italy.
Federica ColioCardiomyopathy Unit, Careggi University Hospital, University of Florence, Florence, Italy.
Giulio SinigianiDepartment of Cardiac, Thoracic and Vascular Sciences and Public Health, University of Padua, Padua, Italy.
Laura De MichieliDepartment of Cardiac, Thoracic and Vascular Sciences and Public Health, University of Padua, Padua, Italy.
Francesca SturdàCardiology Division, Vito Fazzi Hospital, Lecce, Italy.
Marco PozzanCenter for Diagnosis and Treatment of Cardiomyopathies, Cardiovascular Department, Azienda Sanitaria Universitaria Giuliano-Isontina (ASUGI), University of Trieste, Trieste, Italy.
Piero GentileCardiology 4, ASST Niguarda Ca' Granda Hospital, Milan, Italy.
Samuela CarigiCardiology Unit, Infermi Hospital, AUSL della Romagna, Rimini, Italy.
Michela BartolottiCardiology Unit, Bufalini Hospital, AUSL della Romagna, Cesena, Italy.
Giuseppe SenaCardiology Unit, Cardiac Thoracic and Vascular Department, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.
Irene RuotoloCardiology Unit, Cardiac Thoracic and Vascular Department, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.
Giuseppe Damiano SannaDepartment of Molecular Medicine, University of Pavia, Pavia, Italy.
Margherita ZanolettiCardiovascular Unit, IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
Marco CanepaCardiovascular Unit, IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
Massimo Di MarcoCardiology Unit-UTIC, Santo Spirito Hospital, Pescara, Italy.
Emilia D'EliaCardiovascular Department, Papa Giovanni XXIII Hospital, Bergamo, Italy.
Gianluca Di BellaDepartment of Cardiology, University of Messina, Messina, Italy.
Mauro DriussiCardiology, Cardiothoracic Department, Santa Maria della Misericordia Hospital, Udine, Italy.
Massimo ImazioCardiology, Cardiothoracic Department, Santa Maria della Misericordia Hospital, Udine, Italy.
Federico PerfettoCardiomyopathy Unit, Careggi University Hospital, University of Florence, Florence, Italy.
Elena BiaginiEuropean Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, ERN GUARD-Heart, Trieste, Italy.
Giuseppe LimongelliEuropean Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, ERN GUARD-Heart, Trieste, Italy.
Marco MetraCardiology, ASST Spedali Civili, Department of Medical and Surgical Specialities, Radiological Sciences, and Public Health, University of Brescia, Brescia, Italy.
Michele EmdinInterdisciplinary Center of Health Sciences, Scuola Superiore Sant'Anna, Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Giampaolo MerliniDepartment of Molecular Medicine, University of Pavia, Pavia, Italy.
Stefano PerliniAmyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Marco MerloCenter for Diagnosis and Treatment of Cardiomyopathies, Cardiovascular Department, Azienda Sanitaria Universitaria Giuliano-Isontina (ASUGI), University of Trieste, Trieste, Italy.
Giovanni Palladini *Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Gianfranco Sinagra *Center for Diagnosis and Treatment of Cardiomyopathies, Cardiovascular Department, Azienda Sanitaria Universitaria Giuliano-Isontina (ASUGI), University of Trieste, Trieste, Italy.

Funding

Italian Ministry of Health RC-2024-2 789 983
6 · The paper itself

Abstract

aimsTafamidis 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

resultsData 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).

conclusionsIn 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.

Indexed as

Amyloid Neuropathies, FamilialBenzoxazolesCardiomyopathiesAgedAged, 80 and overFemaleHumansItalyMaleMiddle AgedPhenotypePrognosisPropensity ScoreRetrospective StudiesSurvival RateTreatment OutcomeBenzoxazolestafamidisClinical phenotypeDisease‐modifying treatmentSurvivalTransthyretin amyloid cardiomyopathy

Identifiers

PMID41317156
PMCPMC12803573

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