Evidence map›Paper›PMID 41201188›Full record

ArticleEuropean journal of neurology2025

Joint Modelling of Growth and Motor Function Centiles in Corticosteroids Treated Boys With Duchenne Muscular Dystrophy.

Georgia Stimpson, Deborah Ridout, Amy Wolfe, Evelin Milev, Emer O'Reilly, Adnan Manzur, Anna Sarkozy, Francesco Muntoni, Giovanni Baranello, UK NorthStar Clinical Network

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in European journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

10 authors.

Georgia StimpsonDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0003-2795-9898
Deborah RidoutPopulation, Policy & Practice Department, UCL Great Ormond Street Institute of Child Health, London, UK.
Amy WolfeDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
Evelin MilevDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0002-7129-1824
Emer O'ReillyDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0009-0002-1891-3011
Adnan ManzurDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
Anna SarkozyDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
Francesco MuntoniDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
Giovanni BaranelloDubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
UK NorthStar Clinical Network

Funding

Muscular Dystrophy UK 22GRO-PG24-0598-1
6 · The paper itself

Abstract

backgroundCorticosteroid (CS) treated boys with DMD display higher rates of height stunting, higher weight gain, improved motor function scores and delayed loss of ambulation compared to untreated patients. However, the relationship between growth and motor function has historically been understudied due to modelling complexities.

methodsIn this analysis, we use the newly developed motor function centiles for the NSAA, RFF and 10MWR. We consider each combination of growth (height and weight SD) and motor function using multivariate regression models controlling for differential CS treatment (prednisolone/deflazacort, daily/intermittent). This allows inference on the growth and motor function outcomes separately and on the relationship between the outcomes.

resultsWe consider 559 steroid-treated boys with DMD between the ages of 5 and 16 over 1643 assessments. Better motor function trajectories were observed in those treated with daily CS, with the deflazacort daily group displaying a positive NSAA centile trajectory (annual change of 0.07 SD). There was a mild, negative pairwise correlation between the annual changes in NSAA and 10MWR Z-Scores, and height and weight Z-Scores, ranging from -0.25 to -0.36. This indicated that patients with a milder weight gain or more severe height stunting trajectory with respect to their CS treatment were more likely to exhibit a more favourable NSAA or 10MWR trajectory over time.

conclusionsThis work describes the complex relationships between motor function, CS treatment and growth and provides insights for conversations about the relative benefits and negative effects of CS.

Indexed as

Adrenal Cortex HormonesMuscular Dystrophy, DuchenneAdolescentBody HeightBody WeightChildChild, PreschoolHumansMalePrednisolonePregnenedionesAdrenal Cortex HormonesdeflazacortPrednisolonePregnenediones10 meter walk runDuchenne muscular dystrophymotor functionNorthStar ambulatory assessmentrise from floor

Identifiers

PMID41201188
PMCPMC12593539

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.