Evidence map›Paper›PMID 41168830›Full record

ArticleOrphanet journal of rare diseases2025

Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).

Karolina M Stepien, Barbara K Burton, Michael B Bober, Philippe M Campeau, Carolyn Ellaway, Kaustuv Bhattacharya, Nathalie Guffon, David Hinds, Abigail Hunt, Alice Lail and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2025. 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. 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

15 authors.

Karolina M StepienNorthern Care Alliance NHS Foundation Trust, Salford, UK. karolina.stepien@nca.nhs.uk.
Barbara K BurtonAnn & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Michael B BoberNemours/Alfred I. DuPont Hospital for Children, Wilmington, DE, USA.
Philippe M CampeauCHU Sainte-Justine Research Center, Montreal, Quebec, Canada.
Carolyn EllawaySydney Children's Hospital Network, The Children's Hospital at Westmead, Westmead, Sydney, NSW, Australia.
Kaustuv BhattacharyaSydney Children's Hospital Network, The Children's Hospital at Westmead, Westmead, Sydney, NSW, Australia.
Nathalie GuffonReference Centre of Inherited Metabolic Disease, Hospices Civils de Lyon, Lyon, France.
David HindsBioMarin Pharmaceutical Inc., Novato, CA, USA.
Abigail HuntBioMarin Pharmaceutical Inc., Novato, CA, USA.
Alice LailBioMarin Pharmaceutical Inc., Novato, CA, USA.
Shuan-Pei LinMacKay Children's Hospital, Taipei, Taiwan.
Martin MagnerDepartment of Pediatrics and Inherited Metabolic Disorders, General University Hospital and First Faculty of Medicine, Charles University, Prague, Czech Republic.
Elaine MurphyNational Hospital for Neurology and Neurosurgery, London, UK.
Pascal Reisewitz *BioMarin Pharmaceutical Inc., Novato, CA, USA.
John J MitchellMcGill University Health Centre, Montreal, Quebec, Canada.

Funding

BioMarin Pharmaceutical BioMarin Pharmaceutical
6 · The paper itself

Abstract

backgroundMucopolysaccharidosis (MPS) IVA is a rare disease with substantial, multisystemic morbidity. We assessed real-world safety and effectiveness of the enzyme replacement therapy (ERT) elosulfase alfa in patients with MPS IVA in the multinational, observational Morquio A Registry Study (MARS) who initiated ERT in adulthood (aged ≥ 18 years).

methodsPatients were enrolled between September 2014 and February 2022; urinary keratan sulfate (uKS), 6-minute walk test (6MWT) distance, forced expiratory volume in 1 s (FEV

resultsAs of February 13, 2022, 90 patients who initiated ERT had enrolled (median exposure: 5.6 years; median age at first ERT: 27.8 years). Reductions from baseline in uKS levels were sustained over mean follow-up of 5.4 years (mean percent change: -52.9%; p < 0.0001). In patients with available data, mean change in 6MWT distance was + 15.8 m (p = 0.3627) over a mean follow-up of 5.8 years. FEV

conclusionsReal-world data collected from MARS suggest that patients with MPS IVA who initiated ERT in adulthood remained stable over 7 years of follow-up. No new safety signals were identified.

Indexed as

ChondroitinsulfatasesEnzyme Replacement TherapyMucopolysaccharidosis IVAdolescentAdultFemaleHumansMaleMiddle AgedRegistriesTreatment OutcomeYoung AdultChondroitinsulfatasesClinical outcomesElosulfase alfaMucopolysaccharidosis IVAPatient-reported outcomesReal-world evidence

Identifiers

PMID41168830
PMCPMC12574230

What OpenQuestion holds

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LicenceCC BY
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Registered trials

None linked

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.