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Villa C, Auerbach SR, Bansal N, et al. Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION. Pediatr Cardiol. 2022;doi: 10.1007/s00246-021-02807-7.
Villa, C., Auerbach, S. R., Bansal, N., Birnbaum, B. F., Conway, J., Esteso, P., Gambetta, K., Hall, E. K., Kaufman, B. D., Kirmani, S., Lal, A. K., Martinez, H. R., Nandi, D., O'Connor, M. J., Parent, J. J., Raucci, F. J., Shih, R., Shugh, S., Soslow, J. H., Tunuguntla, H., Wittlieb-Weber, C. A., Kinnett, K., & Cripe, L. (2022). Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION. Pediatric cardiology, . https://doi.org/10.1007/s00246-021-02807-7
Villa, Chet, et al. "Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION." Pediatric cardiology vol. (2022). doi: https://doi.org/10.1007/s00246-021-02807-7
Villa C, Auerbach SR, Bansal N, Birnbaum BF, Conway J, Esteso P, Gambetta K, Hall EK, Kaufman BD, Kirmani S, Lal AK, Martinez HR, Nandi D, O'Connor MJ, Parent JJ, Raucci FJ, Shih R, Shugh S, Soslow JH, Tunuguntla H, Wittlieb-Weber CA, Kinnett K, Cripe L. Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION. Pediatr Cardiol. 2022 Jan 13; doi: 10.1007/s00246-021-02807-7. Epub 2022 Jan 13. PMID: 35024902.
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Pediatr Cardiol. 2022 Jan 13; doi: 10.1007/s00246-021-02807-7. Epub 2022 Jan 13.

Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION.

Pediatric cardiology

Chet Villa, Scott R Auerbach, Neha Bansal, Brian F Birnbaum, Jennifer Conway, Paul Esteso, Katheryn Gambetta, E Kevin Hall, Beth D Kaufman, Sonya Kirmani, Ashwin K Lal, Hugo R Martinez, Deipanjan Nandi, Matthew J O'Connor, John J Parent, Frank J Raucci, Renata Shih, Svetlana Shugh, Jonathan H Soslow, Hari Tunuguntla, Carol A Wittlieb-Weber, Kathi Kinnett, Linda Cripe

Affiliations

  1. Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, 3333 Burnet Ave, MLC 2003, Cincinnati, OH, 45229, USA. [email protected].
  2. Department of Pediatrics, Division of Cardiology, University of Colorado, Denver Anschutz Medical Campus, Children's Hospital Colorado Aurora, Aurora, CO, USA.
  3. Division of Pediatric Cardiology, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, USA.
  4. Children's Mercy Hospital and Clinics, Kansas City, MO, USA.
  5. Stollery Children's Hospital, Edmonton, AB, T6G 2B7, Canada.
  6. Boston Children's Hospital, Boston, MA, USA.
  7. Ann and Robert H Lurie Children's Hospital, Chicago, IL, USA.
  8. Yale New Haven Children's Hospital, Yale University School of Medicine, New Haven, CT, USA.
  9. Department of Pediatrics (Cardiology), Stanford University School of Medicine, Palo Alto, CA, USA.
  10. Duke Children's Pediatric and Congenital Heart Center, Duke Children's Hospital, Durham, NC, USA.
  11. Division of Pediatric Cardiology, Primary Children's Hospital, University of Utah, Salt Lake City, UT, USA.
  12. The Heart Institute at Le Bonheur Children's Hospital and The University of Tennessee Health Science Center, Memphis, TN, USA.
  13. Nationwide Children's Hospital, Columbus, OH, USA.
  14. Division of Cardiology, Department of Pediatrics, University of Pennsylvania School of Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  15. Riley Hospital for Children, Indiana University, Indianapolis, IN, USA.
  16. Children's Hospital of Richmond, Virginia Commonwealth University Health System, Richmond, VA, USA.
  17. Congenital Heart Center, University of Florida, Gainesville, FL, USA.
  18. Heart Institute, Joe DiMaggio Children's Hospital, Hollywood, FL, USA.
  19. Department of Pediatrics, Thomas P. Graham Division of Pediatric Cardiology, Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, TN, USA.
  20. Department of Pediatrics, Baylor College of Medicine, Houston, TX, USA.
  21. Parent Project Muscular Dystrophy, Hackensack, NJ, USA.

PMID: 35024902 DOI: 10.1007/s00246-021-02807-7

Abstract

Cardiac disease has emerged as a leading cause of mortality in Duchenne muscular dystrophy in the current era. This survey sought to identify the diagnostic and therapeutic approach to DMD among pediatric cardiologists in Advanced Cardiac Therapies Improving Outcomes Network. Pediatric cardiology providers within ACTION (a multi-center pediatric heart failure learning network) were surveyed regarding their approaches to cardiac care in DMD. Thirty-one providers from 23 centers responded. Cardiac MRI and Holter monitoring are routinely obtained, but the frequency of use and indications for ordering these tests varied widely. Angiotensin converting enzyme inhibitor and aldosterone antagonist are generally initiated prior to onset of systolic dysfunction, while the indications for initiating beta-blocker therapy vary more widely. Seventeen (55%) providers report their center has placed an implantable cardioverter defibrillator in at least 1 DMD patient, while 11 providers (35%) would not place an ICD for primary prevention in a DMD patient. Twenty-three providers (74%) would consider placement of a ventricular assist device (VAD) as destination therapy (n = 23, 74%) and three providers (10%) would consider a VAD only as bridge to transplant. Five providers (16%) would not consider VAD at their institution. Cardiac diagnostic and therapeutic approaches vary among ACTION centers, with notable variation present regarding the use of advanced therapies (ICD and VAD). The network is currently working to harmonize medical practices and optimize clinical care in an era of rapidly evolving outcomes and cardiac/skeletal muscle therapies.

© 2022. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

Keywords: Cardiomyopathy; Duchenne muscular dystrophy; Heart failure

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