Cardiac Management of the Patient With Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) results in a progressive cardiomyopathy that produces significant morbidity and mortality. To improve the quality of life in patients with DMD, cardiac care is focused on surveillance and management, with the goal of slowing the onset and progression of heart failure complications. The current article is intended to be an expanded review on the cardiac management data used to inform the 2018 DMD Care Considerations recommendations as well as be a discussion on clinical controversies and future management directions. The new cardiac guidance includes changes regarding noninvasive imaging surveillance of cardiac function and pharmacologic therapy. Many emerging therapies lack sufficient evidence-based data to be recommended in the 2018 DMD Care Considerations. These are discussed in the present article as clinical controversies and future directions. Important emerging therapies include new heart failure medications, mechanical circulatory support with ventricular assist devices, heart transplantation, and internal cardiac defibrillators. Future research studies should be focused on the risks and benefits of these advanced therapies in patients with DMD. We conclude this review with a brief discussion on the relationship between the heart and the recently developed medications that are used to directly target the absence of dystrophin in DMD.
Competing Interests: POTENTIAL CONFLICT OF INTEREST: Northwestern University receives grant support on behalf of Dr McNally from Solid Biosciences. In addition, Dr McNally is a consultant to Exonics and Invitae. She is a founder of Ikaika Therapeutics and holds a patent relevant to muscular dystrophies; Dr Wagner has received an honorarium from FibroGen, Hoffmann-La Roche, and Wave Life Sciences; the other authors have indicated they have no potential conflicts of interest to disclose.
(Copyright © 2018 by the American Academy of Pediatrics.)