Evidence map›Paper›PMID 37980679›Full record

SynthesisJournal of neuromuscular diseases2024

Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading.

E Landfeldt, A Aleman, S Abner, R Zhang, C Werner, I Tomazos, H Lochmüller, R M Quinlivan

Open access · bronzeAbstract readSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Journal of neuromuscular diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
0.9field-weighted citation impact, top 24% of its field
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

5 citing papers in PubMed, 1 synthesis or guideline pooled it, 6 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Review
  5. 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

8 authors at 5 institutions in 5 countries.

E LandfeldtIQVIA, Stockholm, Sweden.
A AlemanDepartment of Pediatrics, Division of Neurology, Children's Hospital of Eastern Ontario, Research Institute, University of Ottawa, Ottawa, ON, Canada.
S AbnerIQVIA, London, UK.
R ZhangPTC Therapeutics Sweden AB, Askim, Sweden.
C WernerPTC Therapeutics Germany GmbH, Frankfurt, Germany.
I TomazosPTC Therapeutics Inc, South Plainfield, NJ, USA.
H LochmüllerDepartment of Pediatrics, Division of Neurology, Children's Hospital of Eastern Ontario, Research Institute, University of Ottawa, Ottawa, ON, Canada.
R M QuinlivanCentre for Neuromuscular Diseases, UCL Institute of Neurology, National Hospital, London, UK.
University of Ottawa · CAIQ Samhällsbyggnad · SEIQVIA (United Kingdom) · GBNational Hospital for Neurology and Neurosurgery · GBPTC Therapeutics (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDespite advances in the medical management of the disease, respiratory involvement remains a significant source of morbidity and mortality in children and adults with Duchenne muscular dystrophy (DMD).

objectiveThe objective of this systematic literature review was to synthesize and grade published evidence of factors associated with respiratory health and function in DMD.

methodsWe searched MEDLINE, Embase, and the Cochrane Library for records of studies published from January 1, 2000 (to ensure relevance to current care practices), up until and including December 31, 2022, reporting evidence of prognostic indicators and predictors of disease progression in DMD. The quality of evidence (i.e., very low to high) was assessed using the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) framework.

resultsThe bibliographic search strategy resulted in the inclusion of 29 articles. In total, evidence of 10 factors associated with respiratory health and function in patients with DMD was identified: glucocorticoid exposure (high- to very low-quality evidence), DMD mutations (low-quality evidence), DMD genetic modifiers (low-quality evidence), other pharmacological interventions (i.e., ataluren, eteplirsen, idebenone, and tamoxifen) (moderate- to very low-quality evidence), body mass index and weight (low-quality evidence), and functional ability (low-quality evidence).

conclusionsIn conclusion, we identified a total of 10 factors associated with respiratory health in function in DMD, encompassing both pharmacological therapies, genetic mutations and modifiers, and patient clinical characteristics. Yet, more research is needed to further delineate sources of respiratory heterogeneity, in particular the genotype-phenotype association and the impact of novel DMD therapies in a real-world setting. Our synthesis and grading should be helpful to inform clinical practice and future research of this heavily burdened patient population.

Indexed as

Muscular Dystrophy, DuchenneActivities of Daily LivingAdultChildDisease ProgressionGlucocorticoidsHumansGlucocorticoidsDuchenneGRADE approachlung diseasesMuscular dystrophypractice guidelinepulmonary ventilation

Identifiers

PMID37980679
PMCPMC10789346
OpenAlexW4388775488

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.