Evidence map›Paper›PMID 25733532›Full record

ArticleJournal of neurology, neurosurgery, and psychiatry2016

The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials.

Valeria Ricotti, Deborah A Ridout, Marika Pane, Marion Main, Anna Mayhew, Eugenio Mercuri, Adnan Y Manzur, Francesco Muntoni, UK NorthStar Clinical Network

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of neurology, neurosurgery, and psychiatry, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 91 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
91citing papers in PubMed, 3 pooled it
5.1field-weighted citation impact, top 4% 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

91 citing papers in PubMed, 3 syntheses or guidelines pooled it, 148 citations in OpenAlex.

  1. Neurology · 2023
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  3. Corticosteroids for the treatment of Duchenne muscular dystrophy.The Cochrane database of systematic reviews · 2016
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31 more citing papers are in PubMed but not listed here.

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

9 authors at 4 institutions in 2 countries.

Valeria RicottiDubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK Great Ormond Street Hospital, London, UK.
Deborah A RidoutDepartment of Population, Policy and Practice Programme, UCL Institute of Child Health, London, UK Great Ormond Street Hospital, London, UK.
Marika PaneDepartment of Paediatric Neurology, Catholic University, Rome, Italy.
Marion MainDubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK Great Ormond Street Hospital, London, UK.
Anna MayhewInstitute of Human Genetics, Newcastle, UK.
Eugenio MercuriDubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK Department of Paediatric Neurology, Catholic University, Rome, Italy.
Adnan Y ManzurDubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK Great Ormond Street Hospital, London, UK.
Francesco MuntoniDubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK Great Ormond Street Hospital, London, UK.
UK NorthStar Clinical Network
University College London · GBGreat Ormond Street Hospital · GBMRC Institute of Genetics and Molecular Medicine · GBUniversità Cattolica del Sacro Cuore · IT

Funding

Medical Research Council
6 · The paper itself

Abstract

objectiveWith the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand the natural history of this disorder to properly design clinical trials. The aims of this study were to assess the effects produced on motor function by different DMD genotypes and early initiation of glucocorticoids.

methodsThrough the NorthStar Network, standardised clinical data including the NorthStar Ambulatory Assessment score (NSAA) on 513 ambulant UK boys with DMD were analysed from 2004 to 2012. For the analysis of the genetic subpopulation, we also included data from 172 Italian boys with DMD. NSAA raw scores were converted into linear scores.

resultsOn the linearised NSAA, we observed an average decline of 8 units/year (4 units on raw NSAA analysis) after age 7. The median age at loss of ambulation (LOA) was 13 years (95% CI 12.1 to 13.5); 2 years prior to LOA, the estimated mean linearised NSAA score was 42/100 (13/34 raw scale). Starting glucocorticoids between 3 and 5 years conferred an additional gain in motor function of 3 units/year (1.3 raw units) up to age 7. When analysing the effect of genotype in the UK and Italian cumulative cohorts, individuals with deletions amenable to exons 44 and 46 skipping declined at a slower rate over 2 years (9 units (4 raw units), p<0.001), while 53 and 51 skippable deletions showed a faster decline of 14 (4.5; p<0.001) and 5 linearised units (2.4 NSAA units; p=0.02), respectively.

conclusionsOur study provides a novel insight on the current natural history of DMD, which will be instrumental for the design of future clinical trials.

Indexed as

WalkingAdolescentAge of OnsetAnti-Inflammatory AgentsChildClinical Trials as TopicCohort StudiesDisease ProgressionEarly DiagnosisExonsFollow-Up StudiesGene DeletionGenotypeGlucocorticoidsHumansItalyAnti-Inflammatory AgentsGlucocorticoidsMUSCULAR DYSTROPHYNEUROMUSCULAR

Identifiers

PMID25733532
PMCPMC4752678
OpenAlexW2105034643

What OpenQuestion holds

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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.