Evidence map›Paper›PMID 42658465›Full record

ArticleCardiology and therapy2026

Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis.

Fabian Aus dem Siepen, Angela Dispenzieri, Mazen Hanna, Martin Carlsson, Pablo Garcia-Pavia

2 registry-linked trialsAbstract read
PubMed Publisher
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. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

5 authors.

Fabian Aus dem SiepenDepartment of Cardiology, University Hospital Heidelberg, Im Neuenheimer Feld 410, 69120, Heidelberg, Germany. Fabian.Siepen@med.uni-heidelberg.de.ORCID http://orcid.org/0000-0003-4264-1722
Angela DispenzieriMayo Clinic, Rochester, MN, USA.ORCID http://orcid.org/0000-0001-8780-9512
Mazen HannaHeart, Vascular and Thoracic Institute, Cleveland Clinic, Cleveland, OH, USA.ORCID http://orcid.org/0000-0002-7251-3003
Martin CarlssonPfizer Inc, New York, NY, USA.
Pablo Garcia-PaviaHospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, and Centro Nacional Investigaciones Cardiovasculares (CNIC), Madrid, Spain.ORCID http://orcid.org/0000-0002-5470-2257

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionTafamidis is approved for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) and has shown a survival benefit in the phase 3 study ATTR-ACT and in real-world settings. This analysis compared survival in a contemporary, matched, real-world cohort of patients with ATTR-CM treated with tafamidis vs untreated.

methodsData were pooled from an early access cohort (NCT02791230) and from patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) in 2019-2023 with a mixed or predominantly cardiac phenotype. Propensity score matching (1:1) was performed to match treated and untreated patients in the pooled sample based on baseline age, New York Heart Association class, and phenotype. Treated patients received tafamidis at the approved dose for ATTR-CM.

resultsA total of 283 treated and 283 untreated patients were included. Of the 283 treated patients, 85 were from the early access cohort and 198 from THAOS. All untreated patients were from THAOS. In treated vs untreated patients, mean age at enrollment was 76.9 vs 77.4 years, 89.0% vs 83.4% were male, 11.0% vs 16.6% were female, and 80.9% vs 73.1% had wild-type ATTR-CM. Estimated adjusted survival probabilities for all-cause mortality for treated vs untreated patients were 90.2% vs 76.0% at 30 months and 85.3% vs 67.5% at 42 months. Treated patients had a 74% lower risk of all-cause mortality compared with untreated patients (hazard ratio 0.26; 95% confidence interval 0.13-0.51). No new safety signals were identified.

conclusionsThis matched-cohort analysis demonstrated improved survival in a contemporary, real-world cohort of patients with ATTR-CM receiving the approved dose of tafamidis compared with those untreated. These findings further extend the results of ATTR-ACT to contemporary populations of patients with ATTR-CM treated clinically with tafamidis and reinforce earlier real-world evidence supporting tafamidis use.

trial registrationClinicaltrials.gov identifiers NCT00628745 and NCT02791230.

Indexed as

ATTR-CMPropensity score matchingReal-world evidenceSurvival analysisTafamidis

Identifiers

PMID42658465

What OpenQuestion holds

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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.