Evidence map›Paper›PMID 26966795›Full record

ArticleJournal of pediatric rehabilitation medicine2016

Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STARnet.

Emma Ciafaloni, Anil Kumar, Ke Liu, Shree Pandya, Christina Westfield, Deborah J Fox, Kristin M Caspers Conway, Christopher Cunniff, Katherine Mathews, Nancy West and 2 more

Abstract read
In one paragraph

Article in Journal of pediatric rehabilitation medicine, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed, 1 pooled it
–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

28 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Trial
  3. Trial
  4. Review
  5. Article
  6. Observational
  7. Article
  8. Article
  9. Article
  10. Arguments for and against the whole-genome sequencing of newborns.American journal of translational research · 2023
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  12. Article
  13. Article
  14. CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022
    Review
  15. Review
  16. Article
  17. Review
  18. Genetic identification of pathogenic variations of theAnnals of translational medicine · 2021
    Article
  19. Mitochondrial hydrogen sulfide supplementation improves health in theProceedings of the National Academy of Sciences of the United States of America · 2021
    Article
  20. 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.

Emma CiafaloniUniversity of Rochester, Rochester, NY, USA.
Anil KumarNew York State Department of Health, Albany, NY, USA.
Ke LiuThe University of Iowa, Iowa City, IA, USA.
Shree PandyaUniversity of Rochester, Rochester, NY, USA.
Christina WestfieldNew York State Department of Health, Albany, NY, USA.
Deborah J FoxNew York State Department of Health, Albany, NY, USA.
Kristin M Caspers ConwayThe University of Iowa, Iowa City, IA, USA.
Christopher CunniffWeill Cornell Medical, New York, NY, USA.
Katherine MathewsThe University of Iowa, Iowa City, IA, USA.
Nancy WestColorado School of Public Health, Aurora, CO, USA.
Paul A RomittiThe University of Iowa, Iowa City, IA, USA.
Michael P McDermottUniversity of Rochester, Rochester, NY, USA.

Funding

Sureillance and Epidemiologic Research of Duchenne and Becker Muscular DystrophyU01DD000191 · DD · COLORADO STATE DEPT/PUB HLTH & ENVIRONMT · PI MILLER, LISA ANN · 2006 to 2010
$2.5M
Comp A-NY State Surveillance and Research of MD and Neuromuscular DisordersU01DD001123 · DD · CENTER OF ENVIRONMENTAL HEALTH · PI SOIM, AIDA · 2014 to 2018
$2.4M
Comp A-MD and Neuromuscular Disorder Surveillance and Research in IowaU01DD001119 · DD · UNIVERSITY OF IOWA · PI ROMITTI, PAUL A · 2014 to 2018
$2.4M
NCBDD CDC HHS U01 DD000191NCBDD CDC HHS U01DD000191NCBDD CDC HHS U01 DD001119NCBDD CDC HHS U01 DD001123
6 · The paper itself

Abstract

purposeWe investigated the prognostic utility of onset age at first signs and symptoms (SS) to predict onset age at loss of ambulation (LOA) for childhood-onset Duchenne and Becker Muscular Dystrophies (DBMD).

methodsOur cohort comprised male cases with DBMD ascertained by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Adjusted hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using Cox proportional hazards models for associations between onset ages of first SS and LOA. Covariates controlled for were corticosteroid use, family history of DBMD, birth year, race/ethnicity, and MD STARnet site. Onset age at first SS was considered as a continuous and as a categorical variable.

resultsA one-year increase in onset age at first SS was significantly associated with a 10% reduction in annual risk of LOA (HR = 0.90, CI = 0.87-0.94). Treating onset age at first SS as a categorical variable yielded a similar association (≥ 5 years: referent; ≥ 3 to < 5 years: HR = 1.36, CI = 1.02-1.81; 18 months to < 3 years: HR = 1.72, CI = 1.31-2.26; < 18 months: HR = 1.52, CI = 1.14-2.02).

conclusionsEarlier onset age at first SS is associated with earlier onset age at LOA and may have clinical utility in differentiating childhood-onset Duchenne and Becker muscular dystrophies.

Indexed as

Age of OnsetChildChild, PreschoolCohort StudiesGait Disorders, NeurologicHumansInfantMaleMuscular Dystrophy, DuchennePopulation SurveillanceProportional Hazards ModelsambulationBecker Muscular DystrophyDuchenne Muscular Dystrophy

Identifiers

PMID26966795
PMCPMC5868738

What OpenQuestion holds

Textmetadata
LicenceTDM
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.