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Early-Onset Late Gadolinium Enhancement is a Prognostic Factor for Duchenne Cardiomyopathy

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Abstract

Dilated cardiomyopathy (DCM) is an inevitable complication of Duchenne muscular dystrophy (DMD). Late gadolinium enhancement (LGE) demonstrated by cardiac MRI occurs in DMD-related DCM, indicating myocyte death and remodeling. We conducted a retrospective chart review identifying DMD patients in our center between January 2009 and July 2013. Subjects were cohorted by presence of LGE before age 14. We excluded patients in whom we could not determine LGE status prior to age 14. We reviewed comprehensive clinical data. Of the 41 subjects with complete data, 15 demonstrated LGE before age 14 (“early LGE”) and 26 had no LGE by age 14 (“controls”). Those with early LGE exhibited a more rapid decline in LV fractional shortening (p = 0.028). Patients with early LGE were younger at age of initiation of ACE inhibition (p = 0.025), mineralocorticoid receptor antagonism (p = 0.0024), and beta-blockade (p = 0.0017), suggesting aggressive clinical management in response to abnormal MRI findings. There were no significant differences in LV dilation between the two groups (p = 0.1547). Early LGE was not associated with obesity (p = 0.32), age at loss of ambulation (p = 0.31), or heart rate (p-value > 0.8). Early onset of myocardial fibrosis as indicated by LGE on cardiac MRI is associated with earlier progression of cardiomyopathic changes despite earlier medication therapy. Identifying this risk factor, observed in 34% of our cohort during preadolescence, may guide medical therapy and early counseling about cardiomyopathy progression. We advocate for obtaining at least one MRI in patients with DMD prior to age 14 to risk stratify patients.

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Funding

The authors have no relevant financial or non-financial interests to disclose. Partial funding for statistics was obtained through the Division of Cardiology and the Pediatric Residency Program at Children’s Hospital Los Angeles.

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Authors and Affiliations

  1. Department of Pediatrics, Children’s Hospital Los Angeles, 4650 Sunset Blvd, Los Angeles, CA, 90027, USA

    Lorraine James

  2. Department of Pediatrics, Division of Cardiology, Children’s Hospital Los Angeles, Los Angeles, CA, USA

    Jondavid Menteer, John C. Wood & Jennifer A. Su

  3. The Saban Research Institute, Biostatistics Core, Children’s Hospital Los Angeles, Los Angeles, CA, USA

    Lilith C. Moss & Ramon Durazo-Arvizu

  4. Department of Pediatrics, Division of Neurology, Children’s Hospital Los Angeles, Los Angeles, CA, USA

    Leigh Ramos-Platt & Emmanuelle Tiongson

  5. Keck School of Medicine, University of Southern California, Los Angeles, CA, USA

    Jondavid Menteer, Lilith C. Moss, Ramon Durazo-Arvizu, John C. Wood, Leigh Ramos-Platt, Emmanuelle Tiongson & Jennifer A. Su

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  1. Lorraine James
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  2. Jondavid Menteer
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Contributions

Dr. James collected the data, contributed to study design, carried out the initial analyses, drafted the initial manuscript, and reviewed and revised the manuscript. Dr. Menteer reviewed initial analysis, contributed to study design, and reviewed and significantly revised the manuscript. Drs. Lilith Moss and Durazo-Arvizu performed statistical analysis, wrote the section on statistics methodology, composed the figures and table design, and reviewed and revised the manuscript. Dr. Wood reviewed initial analysis, contributed to study design, wrote the section on imaging methodology, and reviewed and revised the manuscript. Drs. Ramos-Platt and Tiongson compiled genetic data and reviewed and revised the manuscript. Dr. Su conceptualized and designed the study, supervised data collection, reviewed initial analyses, coordinated statistical resources, and reviewed and significantly revised the manuscript.

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Correspondence to Lorraine James.

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The Institutional Review Board at Children’s Hospital Los Angeles approved this study and waived the need for informed consent.

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All authors approved the final manuscript as submitted and agreed to be accountable for all aspects of the work.

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James, L., Menteer, J., Moss, L.C. et al. Early-Onset Late Gadolinium Enhancement is a Prognostic Factor for Duchenne Cardiomyopathy. Pediatr Cardiol 44, 433–440 (2023). https://doi.org/10.1007/s00246-022-02989-8

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