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. 2024 Aug;26(8):255-270.
doi: 10.1007/s11936-024-01046-2. Epub 2024 Jun 25.

Advanced Heart Failure Therapies in Neuromuscular Diseases

Arianne Clare Agdamag  1 , Phoo Pwint Nandar  2 , W H Wilson Tang  1
Affiliations

Advanced Heart Failure Therapies in Neuromuscular Diseases

Arianne Clare Agdamag et al. Curr Treat Options Cardiovasc Med. 2024 Aug.

Abstract

Purpose of review: The main objective of this review article is to discuss the prevalence, utilization, and outcomes associated with advanced heart failure therapies among patients with neuromuscular disorders.

Recent findings: Neuromuscular disorders often have multisystem involvement with a high prevalence of cardiovascular pathology. With the improvement in management of respiratory related complications, heart failure is now the leading cause of mortality in this patient population. Advanced heart failure therapies with durable left ventricular assist devices and heart transplantation have proven to be feasible and safe treatment options in selected patients.

Summary: Management of neuromuscular disease involves multidisciplinary team involvement given the systemic nature of the disease. Early recognition and close monitoring of these patients will allow for timely initiation of advanced heart failure therapies that can lead to successful outcomes.

Keywords: Arrhythmia; Cardiomyopathy; Heart failure; Heart transplant; Left ventricular assist device; Neuromuscular disorders.

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Conflict of interest statement

Competing Interests The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Proteins implicated in neuromuscular cardiomyopathy. LGMD, limb girdle muscular dystrophy; DMD, Duchenne muscular dystrophy; BMD Becker muscular dystrophy; FA, Friedrich ataxia; EDMD Emery-Dreifuss muscular dystrophy
Fig. 2
Fig. 2
Pathophysiology of abnormality in neuromuscular protein leading to heart failure
Fig. 3
Fig. 3
Proposed decision-making algorithm for advanced heart failure therapy in patients with neuromuscular dystrophy. LVAD – left ventricular assisted device, OHT – Orthotopic heart transplantation
Fig. 4
Fig. 4
Summary of mechanisms of different gene therapies. A-B Gene replacement can lead to loss of function or gain of function. C-D ASO and CRISPR Cas9 can lead to functional proteins or protein knockdown. E Small molecule therapeutics can lead to protein knockdown. miRNA-microRNA; mRNA -messenger RNA; RISC – RNA induced silencing complex; siRNA – small interfering RNA
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