Evidence map›Paper›PMID 39853770›Full record

ArticleMuscle & nerve2025

Prophylactic Use of Cardiac Medications and Survival in Duchenne Muscular Dystrophy.

Kristin M Conway, Shiny Thomas, Tahereh Neyaz, Emma Ciafaloni, Joshua R Mann, Michelle Staron-Ehlinger, Gary S Beasley, Paul A Romitti, Katherine D Mathews, Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet)

Abstract read
In one paragraph

Article in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

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

  1. Pooled it
  2. Article
  3. 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

10 authors.

Kristin M ConwayDepartment of Epidemiology, College of Public Health, The University of Iowa, Iowa City, Iowa, USA.ORCID https://orcid.org/0000-0001-8633-966X
Shiny ThomasNew York State Department of Health, Albany, New York, USA.ORCID https://orcid.org/0000-0003-3099-4520
Tahereh NeyazDepartment of Epidemiology, College of Public Health, The University of Iowa, Iowa City, Iowa, USA.
Emma CiafaloniDepartment of Neurology, School of Medicine and Dentistry, University of Rochester Medical Center, Rochester, New York, USA.
Joshua R MannDepartment of Preventive Medicine, School of Medicine and John D. Bower School of Population Health, University of Mississippi Medical Center, Jackson, Mississippi, USA.
Michelle Staron-EhlingerStead Family Department of Pediatrics, Roy J. and Lucille A. Carver College of Medicine, The University of Iowa, Iowa City, Iowa, USA.
Gary S BeasleyStead Family Department of Pediatrics, Roy J. and Lucille A. Carver College of Medicine, The University of Iowa, Iowa City, Iowa, USA.
Paul A RomittiDepartment of Epidemiology, College of Public Health, The University of Iowa, Iowa City, Iowa, USA.ORCID https://orcid.org/0000-0001-5393-9984
Katherine D MathewsStead Family Department of Pediatrics, Roy J. and Lucille A. Carver College of Medicine, The University of Iowa, Iowa City, Iowa, USA.
Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet)

Funding

CDC HHS U01 DD001248NCBDD CDC HHS U01 DD000189NCBDD CDC HHS U01 DD000190NCBDD CDC HHS U01 DD000191NCBDD CDC HHS U01 DD001117NCBDD CDC HHS U01 DD001119NCBDD CDC HHS U01 DD001123NCBDD CDC HHS U01 DD001247NCBDD CDC HHS U01 DD001248
6 · The paper itself

Abstract

INTRODUCTION/

aimsProphylactic treatment of left ventricular dysfunction (LVD) in Duchenne muscular dystrophy (DMD) delays onset of LVD, but there is limited data showing impact on survival. Our aim was to describe survival among treated and untreated individuals with DMD.

methodsRetrospective, population-based surveillance data from the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet) were used. We analyzed 327 males with DMD born between 1982 and 2009 who were at least 6 years old at the last visit and who initiated cardiac prophylactic medication before age 14 years. Death status was ascertained through vital record linkages and medical record review. Prophylaxis was defined as cardiac medication use at least 1 year before LVD onset (ejection fraction < 55% or shortening fraction < 28%). Age at first visit, corticosteroid use, scoliosis surgery, initiation of noninvasive ventilation, and loss of ambulation were also coded. Cox Proportional Hazard modeling with time-varying covariates describes associations.

resultsProphylactic cardiac treatment was documented for 27.7% (n = 90); corticosteroids were used by 60.9% (n = 157). Adjusting for age at first visit and MD STARnet site, prophylactic treatment was associated with a 54% lower hazard of death (HR = 0.46, 95% CI = 0.22-0.93) compared to no prophylaxis. Adjusting for selected clinical covariates did not appreciably change the estimate (HR = 0.46, 95% CI = 0.22-0.99). DISCUSSION: Initiation of cardiac medication when left ventricular function is normal was associated with prolonged survival in this study of males with DMD. Only one-quarter of individuals received this treatment, however, indicating a topic of focus for improving care.

Indexed as

Muscular Dystrophy, DuchenneVentricular Dysfunction, LeftAdolescentChildHumansMaleRetrospective StudiesYoung AdultcardiomyopathycardioprotectivecorticosteroidDuchenne muscular dystrophyprophylaxis

Identifiers

PMID39853770
PMCPMC11887528

What OpenQuestion holds

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Registered trials

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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 OpenQuestion graph.