Evidence mapPaperPMID 39939137Full record

Observational studyJournal of neurology, neurosurgery, and psychiatry2025

Observational study of changes to glucocorticosteroid prescribing patterns in duchenne muscular dystrophy in the UK over the last decade.

Gregory Landon, Georgia Stimpson, Michela Guglieri, Anna Sarkozy, Adnan Y Manzur, UK NorthStar Clinical Network, Francesco Muntoni, Giovanni Baranello

Abstract readObservational Study
In one paragraph

Observational study in Journal of neurology, neurosurgery, and psychiatry, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

8 authors.

Gregory LandonGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Georgia StimpsonDubowitz Neuromuscular Centre, UCL GOS Institute of Child Health, London, UK.ORCID http://orcid.org/0000-0003-2795-9898
Michela GuglieriJohn Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Anna SarkozyDubowitz Neuromuscular Centre, UCL GOS Institute of Child Health, London, UK.
Adnan Y ManzurGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
UK NorthStar Clinical Network
Francesco MuntoniGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK f.muntoni@ucl.ac.uk.
Giovanni BaranelloGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundGlucocorticosteroids (GC) are standard-of-care treatment for most boys with duchenne muscular dystrophy (DMD). GC use has changed over time with evolving evidence, and we describe GC patterns, dosing and side-effects in the UK over 11 years.

methodNorthStar data from 2012 to 2022 were analysed to understand GC type, regime and starting age. GC dose with age, patterns of GC switching and side-effect profiles by type and regime were also analysed. Participants attributed to 'other' regimes were queried and details were included.

resultsData on GC usage were available for 1117 boys, across 6905 observations, with 74% of boys GC treated. Prednisolone was the most common regime in the period (65% of assessments) but deflazacort prescription has increased (17% in 2012 and 43% in 2022). Daily regimes were more common (66% of assessments), and the incidence of intermittent (10 days on/10 days off) regimes has declined (46% in 2012 and 26% in 2022). Older participants were more commonly on less than recommended doses, and this was more common in those on deflazacort or daily regimes. Gastrointestinal symptoms and cushingoid features were more common in those on deflazacort than prednisolone, while increased appetite, cushingoid features, gastrointestinal symptoms and insomnia were more common in those on daily than intermittent regimes.

conclusionsThe use of deflazacort and daily regimes has steadily increased across the UK North Star Network in the last decade. This study provides one of the largest up-to-date real-world set of data of evolution in prescription patterns and the occurrence of side-effects in different groups of GC-treated DMD.

Indexed as

GlucocorticoidsMuscular Dystrophy, DuchennePractice Patterns, Physicians'AdolescentChildChild, PreschoolDrug PrescriptionsHumansMalePrednisolonePregnenedionesUnited KingdomdeflazacortGlucocorticoidsPrednisolonePregnenedionesDYSTROPHINMUSCULAR DYSTROPHYNEUROMUSCULARSTEROIDS

Identifiers

PMID39939137
PMCPMC12322482

What Socratic holds

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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 Socratic graph.