Evidence map›Paper›PMID 42693367›Full record

ArticleCardiology and therapy2026

Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis.

Marianna Fontana, Alberto Aimo, Martha Grogan, Maria Generosa Crespo-Leiro, Mathew S Maurer, Fabian Aus dem Siepen, Mazen Hanna, Diego Delgado, Martin Carlsson, Thibaud Damy and 1 more

2 registry-linked trialsAbstract read
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In one paragraph

Article in Cardiology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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.

NCT00628745 completednot on this map

Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.

TypeobservationalSponsorPfizerRan2008 to 2023Enrolled6,718ConditionsTransthyretin Gene Mutations, Transthyretin AmyloidosisArmsNone. Observational Study.
NCT02791230 phase3completednot on this map

A phase 3 multicenter, open-label study to evaluate the safety of daily oral dosing of tafamidis meglumine (pf-06291826-83) 20 mg or 80 mg [or tafamidis (pf-06291826-00) 61 mg] in subjects diagnosed with transthyretin cardiomyopathy (attr-cm)

TypeinterventionalSponsorPfizerRan2016 to 2023Enrolled1,733ConditionsTransthyretin (TTR) Amyloid CardiomyopathyArmsTafamidis
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

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5 · Who and what money

Authors and funding

11 authors.

Marianna FontanaUniversity College London, London, UK. m.fontana@ucl.ac.uk.
Alberto AimoCardiology Division, Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Martha GroganMayo Clinic, Rochester, MN, USA.
Maria Generosa Crespo-LeiroComplexo Hospitalario Universitario A Coruña, INIBIC and CIBERCV, A Coruña, Spain.
Mathew S MaurerColumbia University Irving Medical Center, New York, NY, USA.
Fabian Aus dem SiepenUniversity Hospital of Heidelberg, Heidelberg, Germany.
Mazen HannaDepartment of Cardiovascular Medicine, Cleveland Clinic, Cleveland, OH, USA.
Diego DelgadoDivision of Cardiology, University Health Network, Toronto, Canada.
Martin CarlssonPfizer Inc, New York, NY, USA.
Thibaud DamyReferral Center for Cardiac Amyloidosis, CHU Henri Mondor, Créteil, France.
Margot K DavisUniversity of British Columbia, Vancouver, BC, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionTafamidis improves outcomes in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) versus placebo. ATTR-CM has historically been considered a predominantly male disease, and women are underrepresented in prior studies. Sex-specific data in contemporary patients treated with tafamidis remain limited.

methodsData from two cohorts were pooled: an early-access cohort (NCT02791230) and patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) in 2019-2023. This analysis evaluated sex-related differences in clinical characteristics and all-cause mortality, adjusting for age, cohort, baseline New York Heart Association (NYHA) class, and wild-type (ATTRwt-CM) versus variant (ATTRv-CM) disease.

resultsOverall, 1924 patients treated with tafamidis were included (1476 from the early-access cohort and 448 from THAOS), of whom 214 (11.1%) were women. A smaller proportion of women than men had ATTRwt-CM (59.8% vs. 88.9%), whereas a greater proportion had ATTRv-CM (40.2% vs. 11.1%; p < 0.0001). Women were more frequently of African descent (23.9% vs. 7.4%; overall p < 0.001). Age at enrollment, age at symptom onset, and time from symptom onset to diagnosis were similar between sexes (p > 0.05). Women appeared more symptomatic at baseline, with a higher proportion in NYHA class III/IV (37.7% vs. 29.4%; overall p = 0.036) and classified as National Amyloidosis Centre stage II/III (68.1% vs. 47.7%; overall p = 0.023), despite similar N-terminal pro-B-type natriuretic peptide levels (p > 0.05). In adjusted analyses, women had a higher estimated glomerular filtration rate than men (β [men vs. women] = - 10.61; p < 0.001). Over a mean follow-up of 1.5 years, men had a higher risk of all-cause mortality than women (adjusted hazard ratio [men vs. women], 1.60, 95% CI 1.12-2.30).

conclusionsIn this analysis of contemporary patients with ATTR-CM treated with tafamidis, women represented a small proportion of the overall population, and a greater proportion had ATTRv-CM compared with men. Although women appeared more symptomatic at presentation, men had a higher risk of all-cause mortality.

trial registrationClinicalTrials.gov identifiers: NCT02791230 and NCT00628745.

Indexed as

ATTR-CMReal-world evidenceSexTafamidis

Identifiers

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.