Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy

Duboc, Denis; Meune, Christophe; Lerebours, Guy; Devaux, Jean-Yves; Vaksmann, Guy; Bécane, Henri Marc

doi:10.1016/j.jacc.2004.09.078

Cited by 364 publications

(194 citation statements)

References 11 publications

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“…We used an ACEI/ARB as the first drug of choice according to previous reports by Duboc et al [20,21]. Afterwards, we considered adding a BB for these patients.…”

Section: Discussionmentioning

confidence: 99%

Outcomes of Cardiac Involvement in Patients with Late-Stage Duchenne Muscular Dystrophy under Management in the Pulmonary Rehabilitation Center of a Tertiary Referral Hospital

Kwon

Kang²,

Kim

et al. 2012

Cardiology

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Objectives: The purpose of this study was to investigate the clinical outcome as well as the sequential changes of cardiac function in late-stage Duchenne muscular dystrophy (DMD) patients by 2-dimensional echocardiography. Methods: A total of 31 individuals (initial age: 21.6 ± 5.0 years, range: 15–35 years) with late-stage DMD (Swinyard-Deaver’s stage 7 or 8) were enrolled. All of these patients had respiratory insufficiency and were on ventilator support. Sequential echocardiographic data were collected over at least 3 years. Repeated measures analysis of variance was used to compare changes in left ventricular ejection fraction (LVEF) over time. Results: The sequential change in the mean LVEF showed no significant differences with initial, 1-, 2-, and 3-year follow-up LVEFs which were 42.2, 42.9, 43.8 and 42.6%, respectively (p = 0.320). In terms of the clinical outcome, all but 1 patient survived during the follow-up period of 46.5 ± 9.1 months. Conclusions: The cardiac function in late-stage DMD patients showed a stabilization of LVEF on adequate ventilatory support and optimal cardiac medication therapy until their mid-30s. In addition, considering the favorable clinical outcome in our study, the process of cardiac involvement in late-stage DMD may demonstrate that in some patients it is nonprogressive.

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“…We used an ACEI/ARB as the first drug of choice according to previous reports by Duboc et al [20,21]. Afterwards, we considered adding a BB for these patients.…”

Section: Discussionmentioning

confidence: 99%

Outcomes of Cardiac Involvement in Patients with Late-Stage Duchenne Muscular Dystrophy under Management in the Pulmonary Rehabilitation Center of a Tertiary Referral Hospital

Kwon

Kang²,

Kim

et al. 2012

Cardiology

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“…15 For BMD, only a small percentage of patients have clinical symptoms before 16 years of age, 13 increasing to ≈70% with symptomatic HF by age 40 years. 19 Given that therapeutic intervention before symptom onset has a greater impact, [125][126][127][128][129] the detection of abnormal LVEF affords the opportunity to act. 124 Carriers of DMD/BMD should undergo cardiac evaluation with any symptoms.…”

Section: Cardiac Evaluation In Dmd and Bmdmentioning

confidence: 99%

“…125 They prospectively randomized 57 boys 9.5 to 13 years old to receive perindopril (2-4 mg/day) or placebo. Baseline entry criteria included LVEF >55% by radionuclide imaging, systolic blood pressure ≥80 mm Hg sitting or >70 mm Hg supine, serum blood urea nitrogen ≤20 mg/dL, and ability to tolerate a 1-mg test dose of perindopril.…”

Section: Aceis and Angiotensin Receptor Blockersmentioning

confidence: 99%

“…However, only 1 participant assigned initially to receive ACEIs had an LVEF <45% versus 8 assigned to placebo (P=0.02). 125 The investigators continued the study to assess survival at 10 years, which was a prespecified secondary endpoint. Of the 28 participants initially randomized to ACEI, 93% were alive at 10 years versus 66% of those who were initially assigned to placebo for 3 years.…”

Section: Aceis and Angiotensin Receptor Blockersmentioning

confidence: 99%

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

Feingold¹,

Mahle²,

Auerbach³

et al. 2017

Circulation

223

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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“…The effect of ACEI on systolic dysfunction in monotherapy is controversial. ACEI alone have been shown to reduce mortality in DMD patients with normal left ventricular function at initiation of therapy [8,9]. However, systolic function in this study was only assessed by radionuclide ventriculography [10].…”

Section: Discussionmentioning

confidence: 99%

Recovery of Systolic Dysfunction in Duchenne Muscular Dystrophy due to the Point Mutation c.4213C>T

2010

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Objectives: So far, a beneficial effect of combined angiotensin-converting enzyme inhibitors (ACEI) and β-blocker therapy for systolic dysfunction in Duchenne muscular dystrophy (DMD) has been reported only in patients in whom DMD was due to deletions in the dystrophin gene. Case Report: In a 24-year-old male with DMD due to the point mutation c.4213C>T (p.Gln1405X) in exon 30 of the dystrophin gene, cardiologic examination at the age of 23 years revealed asymptomatic severely reduced systolic dysfunction with a fractional shortening of 14% in the absence of dilated cardiomyopathy. A combined therapy with enalapril (2.5 mg/day) and bisoprolol (1.25 mg/day) was initiated. After a slow increase in the dosage to 10 mg enalapril/day and 2.5 mg bisoprolol/day, systolic dysfunction resolved to a fractional shortening of 26% already after 7 months. Conclusions: This case shows that asymptomatic reduced systolic function also in patients with DMD due to a point mutation responds favourably to a combination therapy with ACEI and β-blockers.

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Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy

Abstract: Early treatment with perindopril delayed the onset and progression of prominent left ventricle dysfunction in children with DMD.

Cited by 364 publications

References 11 publications

Outcomes of Cardiac Involvement in Patients with Late-Stage Duchenne Muscular Dystrophy under Management in the Pulmonary Rehabilitation Center of a Tertiary Referral Hospital

Outcomes of Cardiac Involvement in Patients with Late-Stage Duchenne Muscular Dystrophy under Management in the Pulmonary Rehabilitation Center of a Tertiary Referral Hospital

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

Recovery of Systolic Dysfunction in Duchenne Muscular Dystrophy due to the Point Mutation c.4213C>T

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