Evidence map›Paper›PMID 41162545›Full record

ArticleScientific reports2025

Applicability of phase 3 trial selection criteria to real-world transthyretin amyloid cardiomyopathy patients.

Michael Poledniczek, Lena Marie Schmid, Christina Kronberger, Nikita Ermolaev, René Rettl, Christina Binder, Luciana Camuz Ligios, Mahshid Eslami, Christian Nitsche, Christian Hengstenberg and 5 more

Abstract read
In one paragraph

Article in Scientific reports, 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. Review
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

15 authors.

Michael PoledniczekDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Lena Marie SchmidDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Christina KronbergerDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Nikita ErmolaevDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
René RettlDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Christina BinderDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Luciana Camuz LigiosDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Mahshid EslamiDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Christian NitscheDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Christian HengstenbergDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Roza Badr EslamDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Andreas KammerlanderDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Johannes KastnerDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Jutta Bergler-Klein FescDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria.
Franz DucaDepartment of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Währinger Gürtel 18-20, Vienna, 1090, Austria. franz.duca@meduniwien.ac.at.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Several randomized, double-blind, placebo-controlled trials (RCT) explore disease-modifying therapeutics in transthyretin amyloid cardiomyopathy (ATTR-CM). However, it is currently unclear whether patients eligible to participate in the RCT are representative of real-world patients. ATTR-CM patients presenting to a tertiary referral center for cardiac amyloidosis at the Medical University of Vienna between March 2012 and May 2024 were included in a prospective registry. Inclusion and exclusion criteria of the ATTRACT, ATTRIBUTE, HELIOS-B, CARDIO-TTRANSFORM, and the DEPLETTR-CM trial were applied, and the baseline characteristics of the hypothetical trial cohorts as well as their survival were compared. 353 patients (80.3 years, IQR: 75.5-84.2, 17.6% female) were included, and 192 (57.8%) patients would have been eligible to participate in ATTRIBUTE, ATTRACT would have recruited 163 (49.1%) patients, HELIOS-B 105 (31.6%) patients, CARDIO-TTRANSFORM 80 (24.1%) subjects, and 71 (21.4%) patients would have been eligible for DEPLETTR-CM. Hypothetical ATTRIBUTE, ATTRACT, HELIOS-B, and CARDIO-TTRANSFORM patients demonstrated only minor differences regarding baseline characteristics, both among each other and compared to the real-world cohort. However, DEPLETTR-CM eligible patients exhibited more severely elevated biomarkers of heart failure (NT-proBNP: 2590pg/mL, IQR: 1614-4423, vs. 2339pg/mL, IQR: 1154-4250; p < 0.001) and more advanced National Amyloidosis Centre stage (p < 0.001). Patients who could have been included in DEPLETTR-CM also showed significantly worse survival. Applied to our real-world ATTR-CM cohort, RCT inclusion and exclusion criteria would have selected patients comparable to the real-world cohort. Only the DEPLETTR-CM trial would have selected patients with more advanced disease and worse prognosis.

Indexed as

Amyloid Neuropathies, FamilialCardiomyopathiesClinical Trials, Phase III as TopicPatient SelectionAgedAged, 80 and overBenzoic AcidBenzoxazolesBiomarkersFemaleHeart FailureHumansMaleOligonucleotidesPrognosisProspective StudiesacoramidisBenzoic AcidBenzoxazolesBiomarkerseplontersenOligonucleotidesPyrazolestafamidisDisease-specific therapyPhase III trialTransthyretin amyloid cardiomyopathy

Identifiers

PMID41162545
PMCPMC12572294

What OpenQuestion holds

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Registered trials

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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.