Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy

Kristin M Conway; Shiny Thomas; Emma Ciafaloni; Rabia S Khan; Joshua R Mann; Paul A Romitti; Katherine D Mathews

doi:10.1002/bdr2.2260

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Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy

Journal article

Open access

Peer reviewed

Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy

Kristin M Conway, Shiny Thomas, Emma Ciafaloni, Rabia S Khan, Joshua R Mann, Paul A Romitti and Katherine D Mathews

Birth defects research, Vol.116(1), e2260

01/2024

DOI: 10.1002/bdr2.2260

PMID: 37850663

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Abstract

BACKGROUND Epidemiological support for prophylactic treatment of left ventricular dysfunction (LVD) in Duchenne muscular dystrophy is limited. We used retrospective, population-based surveillance data from the Muscular Dystrophy Surveillance, Tracking and Research Network to evaluate whether prophylaxis delays LVD onset. METHODS We analyzed 455 males born during 1982-2009. Age at first abnormal echocardiogram (ejection fraction <55% or shortening fraction <28%) determined LVD onset. Prophylaxis was defined as cardiac medication use at least 1 year prior to LVD. Corticosteroid use was also coded. Kaplan-Meier curve estimation and Cox Proportional Hazard modeling with time-varying covariates describe associations. RESULTSLVD was identified among 40.7%; average onset age was 14.2 years. Prophylaxis was identified for 20.2% and corticosteroids for 57.4%. Prophylaxis showed delayed LVD onset (p < .001) and lower hazard of dysfunction (adjusted hazard ratio [aHR] = 0.39, 95%CL = 0.22, 0.65) compared to untreated. Compared to no treatment, continuous corticosteroids only (aHR = 1.01, 95%CL = 0.66, 1.53) and prophylaxis only (aHR = 0.67, 95%CL = 0.25, 1.50) were not cardioprotective, but prophylaxis plus continuous corticosteroids were associated with lower hazard of dysfunction (aHR = 0.37, 95%CL = 0.15, 0.80). CONCLUSIONS Proactive cardiac treatment and monitoring are critical aspects of managing Duchenne muscular dystrophy. Consistent with clinical care guidelines, this study supports clinical benefit from cardiac medications initiated prior to documented LVD and suggests further benefit when combined with corticosteroids.

Cardiomyopathy

cardioprotective

corticosteroid

Duchenne muscular dystrophy

prophylaxis

UIOWA OA Agreement

Details

Title: Subtitle: Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy
Creators: Kristin M Conway - University of Iowa
Shiny Thomas - New York State Department of Health
Emma Ciafaloni - University of Rochester Medical Center
Rabia S Khan - University of Iowa
Joshua R Mann - University of Mississippi Medical Center
Paul A Romitti - University of Iowa
Katherine D Mathews - University of Iowa
Resource Type: Journal article
Publication Details: Birth defects research, Vol.116(1), e2260
DOI: 10.1002/bdr2.2260
PMID: 37850663
NLM abbreviation: Birth Defects Res
ISSN: 2472-1727
eISSN: 2472-1727
Publisher: Wiley
Grant note: DOI: 10.13039/100000030, name: Centers for Disease Control and Prevention
Language: English
Electronic publication date: 10/18/2023
Date published: 01/2024
Academic Unit: Neurology; Cardiology; Stead Family Department of Pediatrics; Epidemiology; Iowa Neuroscience Institute; Biostatistics; Neurology (Pediatrics)
Record Identifier: 9984482458002771

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