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Duchenne Dilated Cardiomyopathy: Cardiac Management from Prevention to Advanced Cardiovascular Therapies
Rachele Adorisio1, Erica Mencarelli1, Nicoletta Cantarutti2
1Heart Failure Clinic-Heart Failure, Heart Transplant, Mechanical Circulatory Support Unit, Department of Pediatric Cardiology and Cardiac Surgery, Heart and Lung Transplant, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy.
Insights
Duchenne muscular dystrophy cardiomyopathy requires regular cardiac care, with early ACEi use delaying disease. Current heart failure treatments are vital for managing this condition due to the lack of DMD-specific drugs.
Area of Science:
- Cardiology
- Genetics
- Neurology
Background:
- Duchenne muscular dystrophy (DMD) cardiomyopathy (DCM) presents as a progressive hypokinetic, dilated heart condition.
- Cardiac care is essential for managing DMD patients.
- Early intervention with angiotensin-converting enzyme inhibitors (ACEi) is key to delaying DMD-DCM onset.
Purpose of the Study:
- To review current heart failure (HF) strategies for identifying, characterizing, and treating DMD-DCM.
- To highlight the application of established HF treatments in the absence of DMD-specific therapies.
Main Methods:
- Review of current heart failure (HF) management guidelines.
- Analysis of pharmacological treatments for left ventricle (LV) systolic dysfunction.
- Focus on strategies applicable to DMD-DCM.
Main Results:
- Angiotensin-converting enzyme inhibitors (ACEi) are a primary strategy to delay DMD-DCM.
- Pharmacological treatments aim to improve symptoms and left ventricle (LV) systolic function.
- Established heart failure (HF) treatments are currently used for DMD-DCM.
Conclusions:
- Regular cardiac monitoring and early ACEi use are crucial for DMD patients.
- Current heart failure (HF) management strategies are the mainstay for treating DMD-DCM.
- Further research into DMD-specific therapies is warranted.
Abstract:
Duchenne muscular dystrophy (DMD) cardiomyopathy (DCM) is characterized by a hypokinetic, dilated phenotype progressively increasing with age. Regular cardiac care is crucial in DMD care. Early recognition and prophylactic use of angiotensin converting enzyme inhibitors (ACEi) are the main stay therapeutic strategy to delay incidence of DMD-DCM. Pharmacological treatment to improve symptoms and left ventricle (LV) systolic function, have been widely implemented in the past years. Because of lack of DMD specific drugs, actual indications for established DCM include current treatment for heart failure (HF). This review focuses on current HF strategies to identify, characterize, and treat DMD-DCM.
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