Evidence map›Paper›PMID 34244165›Full record

ArticleArchives of disease in childhood2022

Prediagnosis pathway benchmarking audit in patients with Duchenne muscular dystrophy.

Vasantha Lakshmi Gowda, Miguel Fernandez, Manish Prasad, Anne-Marie Childs, Imelda Hughes, Sandya Tirupathi, Christian Gaudentius Engelbert Lourens De Goede, Declan O'Rourke, Deepak Parasuraman, Tracey Willis and 2 more

Open access · hybridAbstract readMulticenter Study
In one paragraph

Article in Archives of disease in childhood, 2022. 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
0.1field-weighted citation impact, top 56% 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

1 citing paper in PubMed, 3 citations in OpenAlex.

  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

12 authors at 9 institutions in 2 countries.

Vasantha Lakshmi GowdaPaediatric Neurosciences, Evelina London Children's Hospital, London, UK vasantha.gowda@gstt.nhs.uk.ORCID 0000-0002-8367-5489
Miguel FernandezPaediatric Neurosciences, Evelina London Children's Hospital, London, UK.
Manish PrasadPaediatrics, Queen's Medical Centre, Nottingham, UK.
Anne-Marie ChildsPaediatric Neurology, Leeds Teaching Hospitals NHS Trust, Leeds, UK.
Imelda HughesPaediatric Neurology, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester, UK.
Sandya TirupathiPaediatric Neurology, Royal Belfast Hospital for Sick Children, Belfast, UK.
Christian Gaudentius Engelbert Lourens De GoedePaediatric Neurology, Royal Preston Hospital, Preston, UK.ORCID 0000-0001-7772-5972
Declan O'RourkeNeurology, Children's Health Ireland at Temple Street, Dublin, Ireland.
Deepak ParasuramanPaediatrics, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
Tracey WillisMuscle Team, Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust, Oswestry, Shropshire, UK.
Samira SaberianEvidence and Access, OPEN HEALTH, Marlow, UK.
Ian DavidsonUK & Ireland Commercial Department, PTC Therapeutics Limited, Guildford, UK.
Evelina London Children's Healthcare · GBChildren's Health Ireland at Crumlin · IELeeds Teaching Hospitals NHS Trust · GBManchester University NHS Foundation Trust · GBQueen's Medical Centre · GBRobert Jones and Agnes Hunt Orthopaedic Hospital · GBRoyal Belfast Hospital for Sick Children · GBRoyal Preston Hospital · GBUniversity Hospitals Birmingham NHS Foundation Trust · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo describe age and time at key stages in the Duchenne muscular dystrophy (DMD) prediagnosis pathway at selected centres to identify opportunities for service improvement.

designA multicentre retrospective national audit.

settingNine tertiary neuromuscular centres across the UK and Ireland. A prior single-centre UK audit of 20 patients with no DMD family history provided benchmark criteria. PATIENTS: Patients with a definitive diagnosis of DMD documented within 3 years prior to December 2018 (n=122).

main outcome measuresMean age (months) at four key stages in the DMD diagnostic pathway and mean time (months) of presentational and diagnostic delay, and time from first reported symptoms to definitive diagnosis. Type of symptoms was also recorded.

resultsOverall, mean age at definitive diagnosis, age at first engagement with healthcare professional (HCP) and age at first reported symptoms were 53.9±29.7, 49.9±28.9 and 36.4±26.8 months, respectively. The presentational delay and time to diagnosis were 21.1 (±21.1) and 4.6 (±7.9) months, respectively. The mean time from first reported symptoms to definitive diagnosis was 24.2±20.9. The percentages of patients with motor and/or non-motor symptoms recorded were 88% (n=106/121) and 47% (n=57/121), respectively.

conclusionsMajority of data mirrored the benchmark audit. However, while the time to diagnosis was shorter, a presentational delay was observed. Failure to recognise early symptoms of DMD could be a contributing factor and represents an unmet need in the diagnosis pathway. Methods determining how to improve this need to be explored.

Indexed as

Age of OnsetBenchmarkingChild, PreschoolCritical PathwaysDisease ProgressionHumansMuscular Dystrophy, DuchenneRetrospective Studiesauditepidemiologyneuropathology

Identifiers

PMID34244165
PMCPMC8785041
OpenAlexW3180545857

What OpenQuestion holds

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Registered trials

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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.