Evidence map›Paper›PMID 35321113›Full record

ReviewFrontiers in cardiovascular medicine2022

Non-cardiac Manifestations in Adult Patients With Mucopolysaccharidosis.

Karolina M Stepien, Andrew Bentley, Cliff Chen, M Wahab Dhemech, Edward Gee, Peter Orton, Catherine Pringle, Jonathan Rajan, Ankur Saxena, Govind Tol and 1 more

Abstract readReview
In one paragraph

Review in Frontiers in cardiovascular medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing 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

8 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
  7. Article
  8. MPSI Manifestations and Treatment Outcome: Skeletal Focus.International journal of molecular sciences · 2022
    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

11 authors.

Karolina M StepienAdult Inherited Metabolic Diseases, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Andrew BentleyNorthwest Ventilation Unit and Sleep Department, Wythenshawe Hospital, Manchester University National Health Service Foundation Trust, Manchester, United Kingdom.
Cliff ChenClinical Neuropsychology, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
M Wahab DhemechNorthwest Ventilation Unit and Sleep Department, Wythenshawe Hospital, Manchester University National Health Service Foundation Trust, Manchester, United Kingdom.
Edward GeeTrauma and Orthopaedic Surgery, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Peter OrtonTrauma and Orthopaedic Surgery, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Catherine PringleNeurosurgery, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Jonathan RajanManchester and Salford Pain Centre, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Ankur SaxenaNeurosurgery, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Govind TolAnaesthetics Department, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.
Chaitanya GadepalliEar, Nose and Throat, Salford Royal National Health Service Foundation Trust, Salford, United Kingdom.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucopolysaccharidoses (MPS) are a heterogeneous group of disorders that results in the absence or deficiency of lysosomal enzymes, leading to an inappropriate storage of glycosaminoglycans (GAGs) in various tissues of the body such as bones, cartilage, heart valves, arteries, upper airways, cornea, teeth, liver and nervous system. Clinical manifestations can become progressively exacerbated with age and affect their quality of life. Developments in advanced supportive treatment options such as enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT) may have improved patients' life span. Adult MPS patients require specialist clinical surveillance long-term. In many cases, in addition to the MPS-related health problems, they may develop age-related complications. Considering the complexity of their clinical manifestations and lack of guidelines on the management of adult MPS disorders, multispecialty and multidisciplinary teams' care is essential to diagnose and treat health problems that are likely to be encountered. This review presents non-cardiac clinical manifestations, their pathophysiology, management and long-term outcomes in adult MPS patients.

Indexed as

adult mucopolysaccharidosislife spanlong-term complicationslong-term outcomesmortality

Identifiers

PMID35321113
PMCPMC8935042

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.