For many neuromuscular diseases (NMDs), cardiac disease represents a major cause of morbidity and mortality. The management of cardiac disease in NMDs is made challenging by the broad clinical heterogeneity that exists among many NMDs and by limited knowledge about disease-specific cardiovascular pathogenesis and course-modifying interventions. The overlay of compromise in peripheral muscle function and other organ systems, such as the lungs, also makes the simple application of endorsed adult or pediatric heart failure guidelines to the NMD population problematic. In this statement, we provide background on several NMDs in which there is cardiac involvement, highlighting unique features of NMD-associated myocardial disease that require clinicians to tailor their approach to prevention and treatment of heart failure. Undoubtedly, further investigations are required to best inform future guidelines on NMD-specific cardiovascular health risks, treatments, and outcomes. N euromuscular diseases (NMDs) encompass a broad spectrum of diagnoses with overlapping but distinct phenotypes. Common to many NMDs is cardiac involvement. Although the past 3 decades have seen marked advances in our understanding of many NMDs, significant gaps in knowledge remain on how best to approach cardiac care in these patients. For example, survival in Duchenne muscular dystrophy (DMD) has been extended through the use of glucocorticoid use and respiratory support, yet cardiac complications remain a significant cause of morbidity and mortality.1-3 To achieve further gains in care, we will need to improve our understanding of the pathophysiologies driving cardiac involvement in NMDs and advance treatments aimed at preventing the progression of heart failure (HF) and sudden death in NMDs. The recently published findings of an expert working group on cardiac involvement in DMD, which outlined key gaps in knowledge and made specific recommendations aimed at improving diagnosis and management, are a step toward this goal. 4 In this statement, we include a comprehensive overview of the major categories of NMDs with cardiac involvement. For each, a brief background of the gene defect(s), common clinical manifestations (particularly cardiac findings), and current therapies is summarized. Gaps in knowledge are highlighted, and where possible, clinical treatment suggestions are made by the expert writing group appointed by the American Heart Association to review the available literature. Selection of the writing group was performed in accordance with the American Heart Association's conflict-of-interest management policy. Participants volunteered to write sections relevant to their expertise and experience. CLINICAL STATEMENTS AND GUIDELINESconducted a general search of the literature, restricted to human subjects research published between 1980 and 2016. Drafts of each section were written and sent to the chair of the writing group for incorporation into a single document, which was then edited. The edited document was discussed electron...

Background Ventricular Assist Devices (VADs) have come into increasing use in recent years for children. One year survival rates are now above 80% in multiple reports. This report describes adverse events experienced by children with durable ventricular assist devices, using a national-level registry (Pedimacs, a component of Intermacs) Methods Pedimacs is a national registry that contains clinical data on patients who are less than 19 years of age at the time of implantation with a VAD. Data collection concludes at the time of VAD explantation. All FDA-approved devices are included. Pedimacs was launched on September 1, 2012 and this report includes all data from launch until August 2014. Adverse events were coded with a uniform, pre-specified set of definitions. Results This report comprises data from 200 patients, with median age of 11 years (range 11 days-18 years), and total follow-up of 783 patient-months. The diagnoses were cardiomyopathy (n=146, 73%), myocarditis (17, 9%), congenital heart disease (35, 18%), and other (2, 1%). Pulsatile flow devices were used in 91 patients (45%), and continuous flow devices in 109 (55%). Actuarial survival was 86% at 6 months. There were 418 adverse events reported. The most frequent events were device malfunction (n=79), infection (78), neurological dysfunction (52), and bleeding (68). Together, these accounted for 277 events, 66% of the total. Although 38% of patients had no reported adverse event, 16% of patients had 5 or more adverse events. Adverse events occurred at all times following implantation but were most likely to occur in the first 30 days. For continuous flow devices, there were broad similarities in adverse event rates between this cohort, and historical rates from the Intermacs population. Conclusions In this cohort, the overall rate of early adverse events (within 90 days of implantation) was 86.3 events per 100 patient months, and of late adverse events, 20.4 events per 100 patient months. The most common adverse events in recipients of pulsatile VADs were device malfunction, neurological dysfunction, bleeding and infection. For continuous flow VADs, the most common adverse events were infection, bleeding, cardiac arrhythmia, neurological dysfunction and respiratory failure. Compared to an adult Intermacs cohort, the overall rate and distribution of adverse events appears similar.

For one-quarter of a century, major advances have occurred in mechanical support technology for children, thereby expanding the capability to bridge to HTx without compromising post-HTx outcomes. Significant challenges remain, especially for neonates and patients with CHD, but ongoing innovation portends improved methods of support during the next decade.

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