The Subclinical Window for Cardioprotection in Duchenne Muscular Dystrophy: A Systematic Review and Meta-analysis

Area of Science:

• Cardiology
• Genetics
• Pharmacology

Background:

• Duchenne muscular dystrophy (DMD) is a genetic disorder leading to progressive muscle degeneration.
• Cardiomyopathy is a primary cause of death in DMD patients.
• Myocardial injury in DMD begins during a subclinical phase with preserved left ventricular ejection fraction (LVEF).

Purpose of the Study:

• To evaluate the association between early cardioprotective pharmacotherapy and clinical outcomes in DMD.
• To determine if initiating treatment before overt ventricular dysfunction improves outcomes in DMD patients.

Main Methods:

• A systematic review and meta-analysis were conducted following PRISMA guidelines.
• Included randomized trials and observational studies assessing early cardioprotective therapy in DMD.
• Effect estimates were pooled using random-effects models.

Main Results:

• Fourteen studies with 1,788 patients were analyzed.
• Early cardioprotective therapy was linked to a significantly reduced risk of death (HR 0.39).
• Therapy also lowered the risk of cardiac progression (HR 0.36) and overt left ventricular dysfunction (HR 0.41).
• No significant short-term change in LVEF was observed.

Conclusions:

• Early cardioprotective therapy in DMD is associated with improved survival and reduced cardiomyopathy progression.
• Initiating treatment during the subclinical phase of Duchenne cardiomyopathy is supported.
• This strategy offers a potential therapeutic window for cardioprotection in DMD.