Evidence map›Paper›PMID 41196191›Full record

Observational studyEuropean journal of clinical investigation2026

Long-term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey.

Derralynn A Hughes, Guillem Pintos-Morell, Christoph Kampmann, Christina Anagnostopoulou, Jaco Botha, Siddharth Jain, Kathleen Nicholls, Dau-Ming Niu, Ricardo Reisin, Michael L West and 3 more

Registry-linked trialAbstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in European journal of clinical investigation, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03289065 (Fabry Outcome Survey), which is not on this 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.

NCT03289065 completednot on this map

Fabry Outcome Survey (FOS)

TypeobservationalSponsorShireRan2001 to 2021Enrolled4,000ConditionsFabry Disease
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Observational
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

13 authors.

Derralynn A HughesRoyal Free London NHS Foundation Trust, University College London, London, UK.
Guillem Pintos-MorellVall d'Hebron Research Institute, Barcelona, Spain.
Christoph KampmannJohannes Gutenberg School of Medicine, University of Mainz, Mainz, Germany.
Christina AnagnostopoulouTakeda Pharmaceuticals International AG, Zurich, Switzerland.
Jaco BothaTakeda Pharmaceuticals International AG, Zurich, Switzerland.ORCID https://orcid.org/0000-0001-8974-2438
Siddharth JainTakeda Development Center Americas, Inc., Cambridge, Massachusetts, USA.
Kathleen NichollsThe Royal Melbourne Hospital, Parkville, Victoria, Australia.
Dau-Ming NiuInstitute of Clinical Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan.
Ricardo ReisinDepartment of Neurology, Hospital Británico, Buenos Aires, Argentina.
Michael L WestDepartment of Medicine, Dalhousie University, Halifax, Nova Scotia, Canada.
Jörn SchenkTakeda Pharmaceuticals International AG, Zurich, Switzerland.
Uma RamaswamiRoyal Free London NHS Foundation Trust, University College London, London, UK.
Roberto GiuglianiDepartment of Genetics, Federal University of Rio Grande do Sul (UFRGS), Medical Genetics Service, Hospital de Clinicas de Porto Alegre (HCPA), Instituto Nacional de Ciência e Tecnologia em Multiômica Aplicada à Saúde de Precisão (IMASP), Dasa Genomics and Casa dos Raros, Porto Alegre, Brazil.

Funding

Takeda Development Center Americas, Inc.
6 · The paper itself

Abstract

backgroundWe present the final report from the Fabry Outcome Survey (FOS) on long-term effectiveness and safety of agalsidase alfa in adults (≥18 years old).

methodsFOS was an international, multicentre, observational registry (NCT03289065), designed to enhance the understanding of Fabry disease and improve clinical management. Primary effectiveness endpoints were annualized change in estimated glomerular filtration rate (eGFR) and left ventricular mass index (LVMI), and time to and age at composite morbidity event (comprising renal, cardiac or stroke events) and death. Safety outcomes were also assessed.

resultsFOS included data for 1864 adults (female/male, n = 907/957) who received agalsidase alfa only for a median (minimum, maximum) of 6.0 (0, 21.6) years, and 1613 untreated adults (female/male, n = 1235/378). At baseline, mean (standard deviation [SD]) eGFR was 94.01 (27.60) mL/min/1.73 m

conclusionsThis report further supports the long-term effectiveness and safety of agalsidase alfa in adults with Fabry disease.

Indexed as

alpha-GalactosidaseFabry DiseaseAdultAgedEnzyme Replacement TherapyFemaleGlomerular Filtration RateHumansHypertrophy, Left VentricularIsoenzymesMaleMiddle AgedRecombinant ProteinsRegistriesStrokeTreatment Outcomeagalsidase alfaalpha-GalactosidaseIsoenzymesRecombinant Proteinsagalsidase alfacardiacFabry diseaseFabry Outcome Surveyregistryrenal

Identifiers

PMID41196191
PMCPMC12820918

What OpenQuestion holds

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Registered trials

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.