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Cardiac treatment for Duchenne muscular dystrophy: consensus recommendations from the ACTION muscular dystrophy committee

Published online by Cambridge University Press:  27 February 2025

Paul Esteso Open the ORCID record for Paul Esteso [Opens in a new window] ,
Scott R. Auerbach ,
Neha Bansal ,
Rachel Harris ,
Jonathan H. Soslow Open the ORCID record for Jonathan H. Soslow [Opens in a new window] ,
Brian F. Birnbaum ,
Jennifer Conway ,
Linda H. Cripe ,
Deipanjan Nandi  and
Emily Hayes Open the ORCID record for Emily Hayes [Opens in a new window]
...Show all authors
Paul Esteso*
Affiliation:
Cardiology, Boston Children’s Hospital, Boston, MA, USA
Scott R. Auerbach
Affiliation:
Cardiology, Denver Children’s Hospital, Aurora, CO, USA
Neha Bansal
Affiliation:
Pediatric Cardiology, Mount Sinai Kravis Children’s Hospital, New York, NY, USA
Rachel Harris
Affiliation:
Pediatrics, Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, TN, USA
Jonathan H. Soslow
Affiliation:
Pediatrics, Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, TN, USA
Brian F. Birnbaum
Affiliation:
Cardiology, Children’s Mercy, Kansas City, MO, USA
Jennifer Conway
Affiliation:
Cardiology, Stollery Children’s Hospital, Edmonton, AB, Canada
Linda H. Cripe
Affiliation:
Cardiology, Nationwide Children’s Hospital, Columbus, OH, USA
Deipanjan Nandi
Affiliation:
Cardiology, Nationwide Children’s Hospital, Columbus, OH, USA
Emily Hayes
Affiliation:
Cardiology, Nationwide Children’s Hospital, Columbus, OH, USA
Katheryn E. Gambetta
Affiliation:
Cardiology, Ann & Robert Lurie Children’s Hospital of Chicago, Chicago, IL, USA
E. Kevin Hall
Affiliation:
Pediatric Cardiology, Yale Children’s Hospital, New Heaven, CT, USA
Daphne T. Hsu
Affiliation:
Cardiology, The Children’s Hospital at Montefiore, Bronx, NY, USA
Beth D. Kaufman
Affiliation:
Pediatric Cardiology, Lucille Packard Children’s Hospital, Palo Alto, CA, USA
David Rosenthal
Affiliation:
Pediatric Cardiology, Lucille Packard Children’s Hospital, Palo Alto, CA, USA
Sonya Kirmani
Affiliation:
Cardiology, American Family Children’s Hospital at the University of Wisconsin-Madison, Madison, WI, USA
Michelle S. Ploutz
Affiliation:
Pediatric Cardiology, Primary Children’s Hospital, Salt Lake City, UT, USA
Ashwin K. Lal
Affiliation:
Pediatric Cardiology, Primary Children’s Hospital, Salt Lake City, UT, USA
David M. Peng
Affiliation:
Cardiology, C.S Mott Children’s Hospital, Ann Arbor, MI, USA,
Chet R. Villa
Affiliation:
Cardiology, Cincinnati Children’s Hospital, Cincinnati, OH, USA
Svetlana Shugh
Affiliation:
Pediatric Cardiology, Joe DiMaggio Children’s Hospital, Hollywood, FL, USA
Carol A. Wittlieb-Weber
Affiliation:
Pediatric Cardiology, Children’s Hospital of Philadelphia, Philadelphia, PA, USA
Renata Shih
Affiliation:
Child Health Research Institute at University of Florida Shands Children’s Hospital, Gainesville, FL, USA
*
Corresponding author: Paul Esteso; Email: [email protected]
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Abstract

Introduction:

Duchenne muscular dystrophy is a devastating neuromuscular disorder characterized by the loss of dystrophin, inevitably leading to cardiomyopathy. Despite publications on prophylaxis and treatment with cardiac medications to mitigate cardiomyopathy progression, gaps remain in the specifics of medication initiation and optimization.

Method:

This document is an expert opinion statement, addressing a critical gap in cardiac care for Duchenne muscular dystrophy. It provides thorough recommendations for the initiation and titration of cardiac medications based on disease progression and patient response. Recommendations are derived from the expertise of the Advance Cardiac Therapies Improving Outcomes Network and are informed by established guidelines from the American Heart Association, American College of Cardiology, and Duchenne Muscular Dystrophy Care Considerations. These expert-derived recommendations aim to navigate the complexities of Duchenne muscular dystrophy-related cardiac care.

Results:

Comprehensive recommendations for initiation, titration, and optimization of critical cardiac medications are provided to address Duchenne muscular dystrophy-associated cardiomyopathy.

Discussion:

The management of Duchenne muscular dystrophy requires a multidisciplinary approach. However, the diversity of healthcare providers involved in Duchenne muscular dystrophy can result in variations in cardiac care, complicating treatment standardization and patient outcomes. The aim of this report is to provide a roadmap for managing Duchenne muscular dystrophy-associated cardiomyopathy, by elucidating timing and dosage nuances crucial for optimal therapeutic efficacy, ultimately improving cardiac outcomes, and improving the quality of life for individuals with Duchenne muscular dystrophy.

Conclusion:

This document seeks to establish a standardized framework for cardiac care in Duchenne muscular dystrophy, aiming to improve cardiac prognosis.

Keywords

Duchenne muscular dystrophyheart failurecardiomyopathycardiac medications
Type
Original Article
Information
Cardiology in the Young , First View , pp. 1 - 6
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Copyright
© The Author(s), 2025. Published by Cambridge University Press

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