Buddhe S, Cripe L, Friedland-Little J, Kertesz N, Eghtesady P, Finder J, Hor K, Judge DP, Kinnett K, McNally EM, Raman S, Thompson WR, Wagner KR, Olson AK
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.
Seattle Children's Hospital and School of Medicine, USA; Nationwide Children's Hospital, USA; The Ohio State University, USA; St Louis Children's Hospital and School of Medicine, USA; Children's Hospital of Pittsburgh and School of Medicine, USA; Medical University of South Carolina, USA; Parent Project Muscular Dystrophy, USA; Feinberg School of Medicine, USA; Johns Hopkins Hospital, USA; Kennedy Krieger Institute, USA; Seattle Children's Hospital and School of Medicine, USA
10.1542/peds.2018-0333I read more
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