Evidence mapPaperPMID 39248030Full record

ArticleEuropean journal of neurology2024

Relationship between growth and ambulation loss in Duchenne muscular dystrophy boys on steroids.

Georgia Stimpson, Deborah Ridout, Anna Sarkozy, Adnan Manzur, Francesco Muntoni, Giovanni Baranello, NorthStar Network

Abstract read
In one paragraph

Article in European journal of neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Trial
  2. Article
  3. Observational
  4. 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

7 authors.

Georgia StimpsonDubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID https://orcid.org/0000-0003-2795-9898
Deborah RidoutPopulation, Policy and Practice Department, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID https://orcid.org/0000-0003-1336-6450
Anna SarkozyDubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.
Adnan ManzurDubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.
Francesco MuntoniDubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID https://orcid.org/0000-0002-9102-5232
Giovanni BaranelloDubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID https://orcid.org/0000-0003-4871-6692
NorthStar Network

Funding

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

Abstract

background and purposeTreatment with glucocorticoids (GCs) is part of the standard of care in Duchenne muscular dystrophy, but excess weight gain and height stunting are common side-effects. It is still unclear how these growth-related side-effects affect motor function.

methodsThis retrospective cohort study utilized 2228 observations from 648 participants in the UK NorthStar database who had growth and ambulation data recorded between 2006 and 2020. Joint modelling was used to analyse the effect of longitudinal growth centiles on loss of ambulation with respect to GC type and regimen.

resultsLoss of ambulation was observed in 113 patients. National estimates of loss of ambulation age were updated by GC group and showed no significant association between loss of ambulation risk and absolute growth centile. However, yearly drift in weight and/or height centile had an associated risk effect on loss of ambulation. Over a 2-year period, a yearly drift in weight from the 50th to the 75th, 75th to the 90th and 90th to the 95th centile was associated with 138%, 118% and 64% increased risk of loss of ambulation, respectively. Conversely, a 2-year drift in height from the 50th to the 25th, 25th to the 10th and 10th to the 5th centile was associated with 53%, 49% and 35% decreased risk of loss of ambulation, respectively.

conclusionsOur results suggest a complex relationship between growth and loss of ambulation in Duchenne muscular dystrophy boys on chronic GCs, the first step in understanding the effects of drugs which also affect growth patterns.

Indexed as

GlucocorticoidsMuscular Dystrophy, DuchenneAdolescentBody HeightChildChild, PreschoolGrowth DisordersHumansMaleRetrospective StudiesWalkingGlucocorticoidsDuchenne muscular dystrophyglucocorticoidsgrowthwalking

Identifiers

PMID39248030
PMCPMC11555026

What Socratic holds

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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 Socratic graph.