Evidence map›Paper›PMID 41417197›Full record

ArticleCardiology and therapy2026

Real-World Outcomes Among Patients in the United States Receiving Tafamidis for Transthyretin Amyloid Cardiomyopathy.

Daniel P Judge, Margarita Udall, Andrew M Rosen, Neil Lamarre, Elizabeth Nagelhout, Hanh Dung Dao, Mathew S Maurer

Abstract read
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. 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.

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

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

7 authors.

Daniel P JudgeDepartment of Medicine, Medical University of South Carolina, Charleston, SC, USA. judged@musc.edu.ORCID http://orcid.org/0000-0002-3407-0248
Margarita UdallBridgeBio Pharma, Inc., San Francisco, CA, USA.
Andrew M RosenBridgeBio Pharma, Inc., San Francisco, CA, USA.
Neil LamarreGenesis Research Group, Hoboken, NJ, USA.
Elizabeth NagelhoutGenesis Research Group, Hoboken, NJ, USA.
Hanh Dung DaoGenesis Research Group, Hoboken, NJ, USA.
Mathew S MaurerDepartment of Medicine, Columbia University Irving Medical Center, New York, NY, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionTransthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Tafamidis demonstrated efficacy in ATTR-CM clinical trials; however, real-world disease outcomes are not thoroughly characterized. We examined real-world outcomes among patients with wild-type (ATTRwt-CM) and variant (ATTRv-CM) ATTR-CM treated with tafamidis, the only approved treatment at the time of the study.

methodsThis retrospective observational study analyzed Komodo Healthcare Map® data (1/1/2016‒6/30/2024) for tafamidis-treated patients with ATTR-CM. Outcomes included all-cause hospitalization, cardiovascular-related hospitalization (CVH), heart failure (HF)-related hospitalization, outpatient worsening HF (OWHF) with oral diuretic intensification, and mortality. Subgroup analyses examined outcomes by ATTR-CM type and N-terminal pro-B-type natriuretic peptide (NT-proBNP)/B-type natriuretic peptide (BNP) baseline levels.

resultsAmong 3239 tafamidis-treated patients (mean age 77.2 years; 75.9% male; 83.0% ATTRwt-CM; 11.7% ATTRv-CM), the cumulative incidence of first all-cause hospitalization was 22% at 6 months and 36% at 12 months, and that of first CVH was 22% and 35%, respectively. Median time to first CVH was 699 days. The cumulative incidence of OWHF with oral diuretic intensification was 22% at 6 months and 33% at 12 months. Mortality was 12.0% over the 5-year follow-up, and 6.2% at 12 months. The cumulative incidence of the composite endpoint (CVH, OWHF, or death) was 37% within 6 months and 53% within 12 months. In the subgroup with NT-proBNP/BNP baseline measurements (n = 412), patients with high NT-proBNP (> 3000 pg/mL, or BNP > 600 pg/mL) had worse outcomes, including a higher cumulative incidence of first CVH (51% vs. 27%) and higher mortality (9.7% vs. 4.1%) at 12 months.

conclusionsIn this large real-world cohort of tafamidis-treated patients, the cumulative incidences of hospitalization and worsening HF were substantial regardless of ATTR-CM subtype. Elevated NT-proBNP/BNP at baseline was associated with worse outcomes. These findings characterize the burden of disease outcomes in tafamidis-treated patients and underscore ongoing unmet needs in ATTR-CM management.

Indexed as

ATTR-CMCardiovascular hospitalizationHeart failureNT-proBNPReal-world evidenceTafamidisTransthyretin amyloid cardiomyopathy

Identifiers

PMID41417197
PMCPMC12988930

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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.