Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy

Barber, Brent J.; Andrews, Jennifer; Lu, Zhenqiang; West, Nancy A.; Meaney, F. John; Price, Elinora; Gray, Ashley; Sheehan, Daniel W.; Pandya, Shree; Yang, Michele; Cunniff, Christopher

doi:10.1016/j.jpeds.2013.05.060

Cited by 115 publications

(113 citation statements)

References 30 publications

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“…7 A third study found a later age at onset of cardiomyopathy (%SF ,28) in treated boys (15.2 years, SD 3.4) than untreated boys (13.2 years, SD 4.8); for every year of corticosteroid treatment, cardiomyopathy onset was delayed by 4%. 17 A fourth study showed a greater odds of developing cardiomyopathy in untreated boys (4.4 times greater in 3-to 10-year-olds and 15.2 times greater in 11-to 21-year-olds). 30 The same authors later showed a hazard ratio of 0.15 for corticosteroid use in predicting cardiomyopathy in an age-adjusted model.…”

Section: Do Corticosteroids Have An Effect On Cardiac Function?mentioning

confidence: 94%

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Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

Gloss¹,

Moxley²,

et al. 2016

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Methods: We systematically reviewed the literature from January 2004 to July 2014 using the AAN classification scheme for therapeutic articles and predicated recommendations on the strength of the evidence.Results: Thirty-four studies met inclusion criteria.Recommendations: In children with DMD, prednisone should be offered for improving strength (Level B) and pulmonary function (Level B). Prednisone may be offered for improving timed motor function (Level C), reducing the need for scoliosis surgery (Level C), and delaying cardiomyopathy onset by 18 years of age (Level C). Deflazacort may be offered for improving strength and timed motor function and delaying age at loss of ambulation by 1.4-2.5 years (Level C). Deflazacort may be offered for improving pulmonary function, reducing the need for scoliosis surgery, delaying cardiomyopathy onset, and increasing survival at 5-15 years of follow-up (Level C for each). Deflazacort and prednisone may be equivalent in improving motor function (Level C). Prednisone may be associated with greater weight gain in the first years of treatment than deflazacort (Level C). Deflazacort may be associated with a greater risk of cataracts than prednisone (Level C). The preferred dosing regimen of prednisone is 0.75 mg/kg/d (Level B). Over 12 months, prednisone 10 mg/kg/weekend is equally effective (Level B), with no long-term data available. Prednisone 0.75 mg/kg/d is associated with significant risk of weight gain, hirsutism, and cushingoid appearance (Level B). This document summarizes extensive information provided in the complete guideline, available as a data supplement on the Neurology ® Web site at Neurology.org. Appendices e-1 through e-5, cited in this summary, are presented in the full guideline (data supplement). Tables e-1 through e-5 as well as references e1-e8, cited in this summary, are available at Neurology.org.A 2005 American Academy of Neurology (AAN) guideline on the use of corticosteroids in patients with Duchenne muscular dystrophy (DMD) recommended prednisone or deflazacort in the treatment of DMD for short-term benefit in muscle strength and function.1 There are currently variations in practice in corticosteroid use. 2 We address the following questions with regard to patients with DMD:1. What is the efficacy of corticosteroids, specifically their effect on survival, quality of life (QoL), motor function, scoliosis, pulmonary function, and cardiac function? 2. What are the side effects of corticosteroid treatment? 3. How do prednisone and deflazacort compare in efficacy or side effect profile?

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Section: Do Corticosteroids Have An Effect On Cardiac Function?mentioning

confidence: 94%

“…15 The average difference in mean age at loss of ambulation was between 1.4 and 2.5 years in 3 studies. Two studies using both prednisone and deflazacort showed an improvement in age at loss of ambulation 17 and functional motor score. 18 The other studies were not relevant to making conclusions since higher-level evidence was available.…”

mentioning

confidence: 99%

Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

Gloss¹,

Moxley²,

et al. 2016

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“…15,16 Recent studies have suggested that the average age for development of abnormal left ventricular ejection fraction (LVEF) is 14.3 years. 13,17 Because of limitations of movement caused by skeletal muscle disease, early signs of cardiac failure may not appear clinically. Decreased cardiac function or left ventricular (LV) dilation might only be detected by echocardiogram or other cardiac imaging.…”

Section: Dmd and Bmdmentioning

confidence: 99%

Management of Cardiac Involvement Associated With Neuromuscular Diseases: A Scientific Statement From the American Heart Association

Feingold¹,

Mahle²,

Auerbach³

et al. 2017

Circulation

211

195

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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...

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“…[21][22][23] However, most studies to date showed therapeutic effects only in chronic and milder phenotypic patients with DMD. It should be noted that a report by Nakamura, et al indicated that corticosteroids can be a treatment option even in the advanced refractory heart failure in BMD.…”

Section: Article P640mentioning

confidence: 99%