Evidence map›Paper›PMID 37705003›Full record

ArticleOrphanet journal of rare diseases2023

Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study.

Cheng-Hsuan Tsai, Chi-Chao Chao, Sung-Tsang Hsieh, An-Li Yu, Yuan-Kun Aden Wu, Mei-Fang Cheng, Ming-Jen Lee, Chia-Hung Chou, Chia-Tung Shun, Hsueh-Wen Hsueh and 4 more

Open access · goldAbstract read
In one paragraph

Article in Orphanet journal of rare diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
2.0field-weighted citation impact, top 13% of its field
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

8 citing papers in PubMed, 1 synthesis or guideline pooled it, 13 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. Review
  5. Impact of Tafamidis on [European journal of nuclear medicine and molecular imaging · 2025
    Article
  6. Article
  7. Article
  8. 2025 Update Consensus ofActa Cardiologica Sinica · 2025
    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

14 authors at 3 institutions in 1 country.

Cheng-Hsuan TsaiNational Taiwan University College of Medicine, Graduate Institute of Clinical Medicine, Taipei, Taiwan.
Chi-Chao ChaoDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Sung-Tsang HsiehDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
An-Li YuDivision of Cardiology, National Taiwan University Hospital, Taipei, Taiwan.
Yuan-Kun Aden WuDivision of Cardiology, National Taiwan University Hospital, Taipei, Taiwan.
Mei-Fang ChengDepartment of Nuclear Medicine, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan.
Ming-Jen LeeDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Chia-Hung ChouDepartment of Obstetrics and Gynecology, Department of Internal Medicine, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan.
Chia-Tung ShunDepartment of Forensic Medicine and Pathology, National Taiwan University Hospital, Taipei, Taiwan.
Hsueh-Wen HsuehDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Jimmy Jyh-Ming JuangDivision of Cardiology, National Taiwan University Hospital, Taipei, Taiwan.
Ping-Huei TsengDivision of Gastroenterology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Mao-Yuan Su *Department of Medical Imaging, National Taiwan University Hospital, Taipei, Taiwan. maoyuansu@gmail.com.
Yen-Hung Lin *Division of Cardiology, National Taiwan University Hospital, Taipei, Taiwan. austinr34@gmail.com.ORCID http://orcid.org/0000-0001-8153-1441
National Taiwan University Hospital · TWNational Taiwan University · TWYuanpei University · TW

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundTransthyretin cardiac cardiomyopathy (ATTR-CM) is a rare but life-threatening disease. Tafamidis is an effective treatment for patients with ATTR-CM, however its long-term effects on cardiac remodeling and cardiac amyloid deposition are unknown. This study aimed to used cardiac magnetic resonance (CMR) to investigate the effects of tafamidis on patients with hereditary A97S ATTR-CM.

methodsWe retrospectively analyzed a prospective cohort of ATTR-CM patients, including 14 with hereditary A97S ATTR-CM and 17 healthy controls with baseline CMR data. All ATTR-CM patients received tafamidis treatment and received CMR with extracellular volume (ECV) at baseline and after 1 year of follow-up.

resultsBaseline N-terminal pro-B-type natriuretic peptide, left ventricular (LV) mass, LV ejection fraction, global radial, circumferential and longitudinal strain, T1 mapping and ECV were significantly worse in the patients with ATTR-CM compared with the healthy controls. After 1 year of tafamidis treatment, ECV decreased from 51.5 ± 8.9% to 49.0 ± 9.4% (P = 0.041), however there were no significant changes in LV mass, LV ejection fraction, global radial strain, global circumferential strain, global longitudinal strain and T1 mapping.

conclusionsAfter a one-year treatment period, tafamidis exhibited subtle but statistically significant reductions in ECV, potentially indicating a decrease in amyloid deposition among patients diagnosed with hereditary A97S ATTR-CM.

Indexed as

AmyloidosisCardiomyopathiesBenzoxazolesFollow-Up StudiesHumansPrealbuminProspective StudiesRetrospective StudiesBenzoxazolesPrealbumintafamidisCardiac magnetic resonanceExtracellular volumeHereditary transthyretin amyloidosisTafamidis

Identifiers

PMID37705003
PMCPMC10498629
OpenAlexW4386715570

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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.