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. 2025 Apr;71(4):574-582.
doi: 10.1002/mus.28353. Epub 2025 Jan 24.

Prophylactic Use of Cardiac Medications and Survival in Duchenne Muscular Dystrophy

Kristin M Conway  1 , Shiny Thomas  2 , Tahereh Neyaz  1 , Emma Ciafaloni  3 , Joshua R Mann  4 , Michelle Staron-Ehlinger  5 , Gary S Beasley  5 , Paul A Romitti  1 , Katherine D Mathews  5 ; Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet)
Affiliations

Prophylactic Use of Cardiac Medications and Survival in Duchenne Muscular Dystrophy

Kristin M Conway et al. Muscle Nerve. 2025 Apr.

Abstract

Introduction/aims: Prophylactic 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.

Methods: Retrospective, 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.

Results: Prophylactic 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.

Keywords: Duchenne muscular dystrophy; cardiomyopathy; cardioprotective; corticosteroid; prophylaxis.

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Conflict of interest statement

Katherine D. Mathews serves as an advisory board member for MDA and the FSH Society; is a board member for the Friedreich Ataxia Research Alliance (FARA); receives or has recently received clinical trial funding from PTC Therapeutics, Sarepta Therapeutics, Pfizer, Reata, Italfarmaco, Fibrogen, Italfarmaco, CSL Behring, AMO, and Reata. Emma Ciafaloni has received personal compensation for serving on advisory boards and/or as a consultant for Alexion, Argenx, Biogen, Amicus, Momenta, Medscape, Pfizer, PTC Therapeutics, Sanofi/Genzyme, Sarepta, Janssen, NS Pharma, Wave, and Strongbridge Biopharma; has received research and/or grant support from the Centers for Disease Control and Prevention, CureSMA, Muscular Dystrophy Association, National Institutes of Health, Orphazyme, the Patient‐Centered Outcomes Research Institute, Parent Project Muscular Dystrophy, PTC Therapeutics, Santhera, Sarepta Therapeutics, Orphazyme, and the US Food and Drug Administration; and has received royalties from Oxford University Press and compensation from Medlink for editorial duties. The other authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Flowchart of exclusions. aComorbid conditions affecting disease progression or survival included: Seizure disorder, spina bifida, cerebral palsy, encephalitis, Xp21.3 deletion, cerebral hemorrhage (near birth). LVD = left ventricular dysfunction.
FIGURE 2
FIGURE 2
Kaplan–Meier curve estimation for time from birth to death by cardiac prophylaxis.
See this image and copyright information in PMC

References

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