Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy

Bourke, John; Bueser, Teofila; Quinlivan, R.

doi:10.1002/14651858.cd009068.pub3

Cited by 30 publications

(41 citation statements)

References 113 publications

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“…The disease is invariably progressive and DMD boys are usually wheelchair-bound by age 10–13. Thanks to a steady improvement in palliative care, chiefly nocturnal assisted ventilation, life quality and expectancy have increased, but most patients eventually succumb due to respiratory/cardiac failure by the third decade [3].…”

Section: Introductionmentioning

confidence: 99%

Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy

Vitiello

Tibaudo

Pegoraro

et al. 2019

IJMS

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Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. The disease is caused by the lack of dystrophin, a structurally essential protein; hence, a definitive cure would necessarily have to pass through some form of gene and/or cell therapy. Cell- and genetic-based therapeutics for DMD have been explored since the 1990s; recently, two of the latter have been approved for clinical use, but their efficacy is still very low. In parallel, there have been great ongoing efforts aimed at targeting the downstream pathogenic effects of dystrophin deficiency using classical pharmacological approaches, with synthetic or biological molecules. However, as it is always the case with rare diseases, R&D costs for new drugs can represent a major hurdle for researchers and patients alike. This problem can be greatly alleviated by experimenting the use of molecules that had originally been developed for different conditions, a process known as drug repurposing or drug repositioning. In this review, we will describe the state of the art of such an approach for DMD, both in the context of clinical trials and pre-clinical studies.

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Section: Introductionmentioning

confidence: 99%

Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy

Vitiello

Tibaudo

Pegoraro

et al. 2019

IJMS

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“…Some DMD children with cardiac involvement were undertreated or not being treated with cardiac-specific medicines in clinical practice [ 4 ]. Although DMD cannot be cured, some medicines may modify the course of cardiomyopathy and be beneficial for delaying progression of heart failure [ 22 – 24 ]. The onset of symptoms and reduced LVEF were the important factors affecting the timing of medical treatment [ 5 , 6 ].…”

Section: Discussionmentioning

confidence: 99%

Usefulness of speckle-tracking echocardiography for early detection in children with Duchenne muscular dystrophy: a meta-analysis and trial sequential analysis

Song

Zhang

Wang

et al. 2020

Cardiovasc Ultrasound

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Background Duchenne muscular dystrophy (DMD) is the most common form of inherited muscle disease in children. The incidence of cardiomyopathy induced by DMD increases with age. Left ventricular ejection fraction usually fails to reflect the subclinical left ventricular dysfunction. Several studies have assessed this dysfunction using myocardial strain measured by speckle-tracking echocardiography (STE). However, the results were inconsistent and incomplete. Methods Several databases were searched from their inception to February 5, 2020. The summarized weighted mean difference (WMD) with 95% confidence intervals (CIs) were estimated for myocardial strain between DMD and healthy controls and a meta-analysis was conducted. Trial sequential analysis estimated whether the resulting evidence was sufficient. Results Eight studies with a total of 269 DMD children and 299 healthy participants were included. STE revealed that global longitudinal strain (GLS), global circumferential strain, average longitudinal strain (measured by two-dimensional STE at the apical four-chamber view), and average circumferential strain (measured by two-dimensional STE at the papillary muscle short-axis level) decreased (WMD = 4.17, 95% CI: 3.03–5.32; WMD = 3.98, 95% CI: 0.29–7.68; WMD = 4.18, 95% CI: 2.75–5.62; and WMD = 4.90, 95% CI: 2.38–7.43, respectively; all P < 0.05) compared with the controls and global radial strain was unchanged in the DMD group (WMD = − 4.33, 95% CI: − 9.53–0.87, P = 0.103). Trial sequential analysis indicated that available GLS samples were sufficient and confirmed that adequate evidence was accumulated. The credibility of other myocardial strains was questioned due to insufficiently involved studies. Conclusion GLS can be useful for early detection of left ventricle myocardial dysfunction in children with DMD.

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“…All patients need annual cardiac surveillance by specialists, which may involve ultrasound echocardiograms, cardiac magnetic resonance imaging, and 24 hour holter monitoring. Many patients take cardioprotective medications, such as ACE inhibitors and β-blockers 3233…”

Section: What Are the Key Complications And Their Recommended Managemmentioning

confidence: 99%

Duchenne muscular dystrophy

Fox

Millington²,

Mahabeer

et al. 2020

BMJ

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Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy

Abstract: Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy.

Cited by 30 publications

References 113 publications

Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy

Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy

Usefulness of speckle-tracking echocardiography for early detection in children with Duchenne muscular dystrophy: a meta-analysis and trial sequential analysis

Duchenne muscular dystrophy

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