Evidence map›Paper›PMID 39595144›Full record

ArticleBiomedicines2024

Safety and Tolerability of a Shorter Agalsidase Beta Infusion Time in Patients with Classic or Later-Onset Fabry Disease.

Dominique P Germain, Alice Porto Vasconcelos, Lien Tran Thi Phuong, Najya Bedreddine, Mihaela Turcan, Wenting Trang, Lynda Barache

Abstract read
In one paragraph

Article in Biomedicines, 2024. 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. Progress and Challenges in the Treatment of Fabry Disease.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    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

7 authors.

Dominique P GermainDivision of Medical Genetics, University of Versailles-St Quentin en Yvelines (UVSQ), Paris-Saclay University, 2 Avenue de la Source de la Bièvre, F-78180 Montigny, France.ORCID 0000-0002-8355-007X
Alice Porto VasconcelosDivision of Medical Genetics, APHP Paris Saclay University, F-92380 Garches, France.ORCID 0000-0002-9423-956X
Lien Tran Thi PhuongReferral Center for Fabry Disease and Lysosomal Diseases, MetabERN, F-92380 Garches, France.
Najya BedreddineAssociation des Patients de la Maladie de Fabry, F-21160 Perrigny les Dijon, France.
Mihaela TurcanDivision of Medical Genetics, APHP Paris Saclay University, F-92380 Garches, France.
Wenting TrangDivision of Medical Genetics, APHP Paris Saclay University, F-92380 Garches, France.
Lynda BaracheReferral Center for Fabry Disease and Lysosomal Diseases, MetabERN, F-92380 Garches, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe multisystem manifestations of Fabry disease can create major challenges in patient care. Although enzyme replacement therapy with recombinant agalsidase beta has demonstrated clinical benefits, the standard fortnightly, multi-hour infusion regimen imposes a substantial burden on patients.

methodsWe assessed the safety and feasibility of shortening the agalsidase beta infusion time to 90 min in adult patients with classic or later-onset Fabry disease in the absence of premedication. A total of 39 consecutive adult patients (agalsidase-naïve: n = 7; with significant comorbidities: n = 15) with no recent infusion-associated reactions underwent a total of 85 agalsidase beta infusions in our tertiary reference centre for lysosomal diseases. Each infusion was administered at a constant rate (between 0.78 and 1.17 mg/min, depending on the total dose administered).

resultsNo adverse events of any type (including discomfort and infusion-associated reactions) were reported during or after infusions. The patients' vital signs and physical examination remained stable, and patients' satisfaction was high.

conclusionsOur results suggest that shortening the agalsidase beta infusion time to 90 min is safe and feasible in stably treated adult patients with Fabry disease and no recent infusion-associated reactions.

Indexed as

agalsidase betaFabry diseasequality of lifereduced infusion timesafetytolerability

Identifiers

PMID39595144
PMCPMC11591728

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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.