Evidence map›Paper›PMID 42204725›Full record

ArticleOrphanet journal of rare diseases2026

Unfavorable cardiovascular risk profile without increased event prevalence in late-onset Pompe disease: an individually matched cohort study.

Harmke A van Kooten, Danny van Zoest, Michelle Michels, Nadine A M E van der Beek, Esther Brusse, Ans T van der Ploeg, Janneke G Langendonk, Pieter A van Doorn, Margreet A E M Wagenmakers

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Article in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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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

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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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

9 authors.

Harmke A van KootenDepartment of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Danny van ZoestDepartment of Internal Medicine, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Michelle MichelsDepartment of Cardiology, Cardiovascular Institute, Thoraxcenter, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Nadine A M E van der BeekDepartment of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Esther BrusseDepartment of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Ans T van der PloegDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC - Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Janneke G LangendonkDepartment of Internal Medicine, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Pieter A van DoornDepartment of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Margreet A E M WagenmakersDepartment of Internal Medicine, Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands. m.wagenmakers@erasmusmc.nl.ORCID http://orcid.org/0000-0003-2587-0283

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundIt is unclear whether cardiovascular risk factors are more common or if cardiovascular disease occurs more frequently in adults with late-onset Pompe disease (LOPD) than in the general population. We investigated the prevalence of cardiovascular risk factors and cardiovascular events in adults with LOPD compared to an individually matched control cohort.

methodsAdults with LOPD were individually matched to control subjects, with the same sex, smoking status, age (± 5 years) and BMI (± 5 kg/m

resultsSeventy-eight patients (median age 55.9 years, BMI 25.1 kg/m

conclusionPatients with LOPD showed a less favorable cardiovascular risk profile compared to an individually matched control cohort. The prevalence of cardiovascular events did not significantly differ between the cohorts, although our study is likely underpowered to detect subtle differences due to the low number of events.

Indexed as

Cardiovascular DiseasesGlycogen Storage Disease Type IIAdultAgedBlood PressureCohort StudiesFemaleHeart Disease Risk FactorsHumansMaleMiddle AgedPrevalenceRisk FactorsCardiovascular diseaseCardiovascular risk factorsElectrocardiogramHypertensionLate-onset Pompe disease

Identifiers

PMID42204725
PMCPMC13422358

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