Evidence map›Paper›PMID 39906339›Full record

SynthesisFrontiers in cardiovascular medicine2024

A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta.

Gavin Y Oudit, Pronabesh DasMahapatra, Nicole Lyn, Florence R Wilson, Adekemi Adeyemi, Chae Sung Lee, Ana Crespo, Mehdi Namdar

Abstract readSystematic Review
In one paragraph

Synthesis in Frontiers in cardiovascular medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
–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 synthesis or guideline pooled it.

  1. Pooled it
  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

8 authors.

Gavin Y OuditDivision of Cardiology, Department of Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada.
Pronabesh DasMahapatraSpecialty Care, Sanofi, Cambridge, MA, United States.
Nicole LynSpecialty Care, Sanofi, Cambridge, MA, United States.
Florence R WilsonPRECISIONheor, Vancouver, BC, Canada.
Adekemi AdeyemiPRECISIONheor, Vancouver, BC, Canada.
Chae Sung LeeSpecialty Care, Sanofi, Cambridge, MA, United States.
Ana CrespoSpecialty Care, Sanofi, Milan, Italy.
Mehdi NamdarCardiology Division, Geneva University Hospitals, Geneva, Switzerland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Agalsidase beta is used to treat Fabry disease (FD); however, data on cardiac and cerebrovascular outcomes with agalsidase beta treatment come from studies with limited numbers of patients. Methods: A systematic literature review of studies reporting on the efficacy and effectiveness of agalsidase beta in FD was conducted. Studies were identified in searches of MEDLINE, Embase, and the Cochrane Central Register of Controlled Trials from January 2000-June 2022. Outcomes of interest included cardiac structure and mass, cardiac events, and cerebrovascular events. Results: Fifty-two citations (41 studies) were included. Reductions in interventricular septal thickness (IVST) and/or left ventricular posterior wall thickness (LVPWT) were demonstrated in six studies (follow-up 1-6 years, Conclusion: Literature over the last 20 years indicates that agalsidase beta treatment may lead to stabilization or regression of cardiac structural thickness and mass, and reduction in cardiac and cerebrovascular events relative to placebo.

Indexed as

agalsidase betacardiaccardiovascularenzyme replacement therapyFabry diseasegenetic disorders

Identifiers

PMID39906339
PMCPMC11790562

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.